LEHI If given a chance, who wouldnt want to spend a few bucks to find out if theyre at heightened risk for one day having to confront some life-changing or life-ending medical malady?

Thats the concept fueling an explosion in direct-to-consumer genetic testing and one thats also elevating ethical debates about how this most personal of information should be interpreted and protected.

Utah-born Ancestry.com is the latest entry into a growing list of companies offering health-focused genetic testing an industry expected to grow to $20 billion annually in the next few years.

While best known and an industry leader for its expertise in providing answers to the Where am I from? question, Ancestry will now expand its genetic testing resources to help people anticipate future health issues and help address, Whats going to make me sick?

Last week, the company that launched more than 30 years ago as a family history search service, later adding DNA testing to help customers identify their geographic ancestral roots, announced its new, health-focused genetic testing service.

AncestryHealth will offer two levels of genetic testing that the company said will deliver actionable insights that can empower people to take proactive steps in collaboration with their health care provider to address potential health risks identified in their genes and family health history.

Ancestry CEO Margo Georgiadis said the new genetic tests will help clients proactively manage their health care needs, armed with new insight on what conditions they may be predisposed for, based on genetic evidence.

Your genes dont need to be your destiny, Georgiadis said in a statement. Understanding your familial and inherited health risks can help you take action with your doctor to improve your chances of better health outcomes.

For more than three decades, Ancestry has empowered journeys of personal discovery to enrich lives. In the same way that knowledge of your family and ethnicity helps you understand your past to inspire your future, knowledge of your genetic health profile and any associated risks can help you be proactive in managing the future for you and your family.

The two testing products, according to the company, include AncestryHealth Core, which uses the companys current genotype genetic assessment technique to detect genetic differences and deliver personalized reports related to health conditions such as heart disease, hereditary cancers, blood-related disorders, and risks for carrier status of health conditions, such as Tay-Sachs disease. The one-time test costs $149 and also includes the companys family history report. Those who have already submitted a biologic sample to the company can get the new genetic report for $49.

While likely not available until sometime in 2020, the AncestryHealth Plus will use more current, genetic sequencing technology that will provide greater coverage of DNA differences for each condition and more risk categories such as those related to potentially developing heart disease, cancers, and disorders related to blood, the nervous system and connective tissues. The sequencing test will require a $199 activation fee, which the company said includes the first six months of membership and an additional $49 membership fee every six months. Existing Ancestry customers will be able to upgrade to AncestryHealth Plus for an initial payment of $49.

Ancestrys testing regimen will assess genetic samples and indicate predispositions for high cholesterol and cardiomyopathy, which can lead to heart disease; hereditary indicators for breast, ovarian, colon and uterine cancers; and blood disorders including abnormal clotting and iron overload. The testing can also determine if the sample donor is a gene carrier for cystic fibrosis, sickle-cell anemia or Tay-Sachs disease, a fatal nervous system disorder that most commonly occurs in children.

Unlike its competitor, 23andMe, which has earned U.S. Food and Drug Administration approval for providing genetic test results directly to customers without a physicians participation, Ancestrys genetic testing service requires a physicians order to conduct the tests and the company says it has contracted with a private network of independent physicians and genetic counselors who participate in the process. Ancestrys health testing service also connects customers to educational information, including access to genetic counseling resources and provides printable and consumer and physician-ready reports that provide guidance for next steps an individual and their health care provider can take together.

Lynn Jorde, chairman of the University of Utahs Department of Human Genetics and executive director of the Utah Genome Project, said while labs are now capable of sequencing the entirety of the human genome some 3 billion genetic basis pairs the microarray technique currently used by Ancestry evaluates a small window of genes that, if a variation is found, have a viable medical response.

What theyre looking at is specific changes in the DNA that we know about in specific instances ... and are often called actionable genes, Jorde explained. If you have a disease causing variant here, there is actually something we can do about it.

Jorde said while some genetic markers, like those for cystic fibrosis, indicate a high probability that you have or will develop that condition, many more are merely suggestive.

The predictive power of genetic testing is getting better and better, but it will never be perfect, Jorde said. For many of these conditions, there are nongenetic components that impact risk.

Jorde said things like environment, diet and exercise/activity level can play a significant role in an individuals risk of developing an illness or disease.

Teneille Brown is a professor at the University of Utahs S.J. Quinney College of Law and an expert in health law and medical ethics. In an interview, she noted direct-to-consumer genetic testing services, now being offered by dozens of companies according to the National Institute of Health, are occupying a space thats in between current regulatory boundaries aimed at protecting individuals privacy rights.

In the research realm, any federally funded projects are subject to stringent privacy rules, Brown said. That is also the case for health care institutions that handle genetic material, under (Health Insurance Portability and Accountability Act) rules.

But the big databases being built by testing companies are outside of the federal funding process and are not health care providers, so the HIPAA rules dont apply, Brown said.

Ancestry appears to underscore this by noting, in its user agreement, that it is not a covered entity under HIPAA rules.

Brown noted that in addition to unanswered questions about privacy protections, genetic test results can lead to deep emotional impacts for tested individuals, either through the discovery of gene markers that are suggestive of some future medical challenge or, less obviously, when a clean test is returned, which may provide an inaccurate suggestion that theres nothing to worry about.

Theres a huge problem when it comes to understanding what these risk scores mean, Brown said. The predictive values of these results is widely variable, including what is, or is not, implied by failure to find a specific marker.

Brown said genetic testing companies have wide-ranging policies regarding sharing an individuals genetic test results or stored biologic samples with third-party researchers. Ancestry, for example, says it will only share your information if youve given them specific permission to do so, through its informed consent agreement.

While the regulatory world is lagging behind the fast-moving development of genetic testing technology, Brown said she believes the bigger companies, including Ancestry, are working to create appropriate protections for their customers. And, she added, the growing body of knowledge being accumulated by this work could lead to groundbreaking advancements in treatments for serious diseases.

These companies might play a role in developing amazing drugs and therapies, Brown said. Collectively, they are adding all of this amazing content, providing pedigrees and information and incredibly powerful databases ... and a lot of good can come of it.

Its not at all sinister, but we need consumers to know what theyre submitting and being diligent about potential secondary uses of that data. More robust consent requirements for users and strict limitations for secondary uses are certainly in order.

See the rest here:
Genetic testing could keep you healthy. But what about personal info? - Deseret News

Recommendation and review posted by Bethany Smith

St. Elizabeth Healthcare

At St. Elizabeth Healthcare, we know that a breast cancer diagnosis can be shocking and overwhelming. We offer comprehensive care from the moment of your breast cancer diagnosis through your surgery, treatments, recovery and beyond.

Our breast cancer care protocol has recently expanded to include meeting with a genetic counselor shortly after your diagnosis.

New guidelines recommend multi-gene panel genetic testing be offered to all newly diagnosed breast cancer patients, regardless of age or family history.

Genetic information can be very impactful, says Justine Snyder, Licensed Genetic Counselor in the Hereditary Cancer Program at St. Elizabeth Healthcare. Our goal is to help keep patients and their family members healthy by making proactive decisions.

These proactive decisions could include earlier screening mammograms for family members if specific breast-cancer genes like BRCA1 and BRCA2 are identified, as they can increase the risk of ovarian or breast cancer. Genetic testing gives our patients and their families the information they need to properly and proactively manage their health moving forward.

At St. Elizabeth Healthcare, newly diagnosed breast cancer patients will meet with a genetic counselor during their initial breast surgeon appointment.

During your meeting with our genetic counselor, you will:

Once this information is reviewed and you decide to move forward with the genetic testing, a saliva sample will be collected. Our genetic counselors will also discuss the results timeline with you, which is typically two to four weeks, as well as how the results will be communicated to you. Additionally, GINA law (Genetic Information Nondiscrimination Act) and any insurance questions will be covered during this appointment.

Patients occasionally opt out of genetic testing after meeting with St. Elizabeth counselors and thats ok.

Genetic testing is a personal decision, says Justine. Patients are given the option to proceed with testing. If they decline to test, we provide our contact information and a brochure in case they decide to proceed at a later date.

Reasons for opting out include feeling too overwhelmed at the time, concern about out-of-pocket costs and family members not wanting to know the results of the testing.

No matter what your decision, our expert team of genetic counselors are here anytime to answer any questions or concerns you may have. Please call (859) 301-GENE (4363) to speak with a genetic counselor or to learn more about the Hereditary Cancer Program.

Link:
Healthy Headlines: Breast cancer genetic testing: What you need to know to make an informed decision - User-generated content

Recommendation and review posted by Bethany Smith

The morning Cuppa Coffee, with its caffeine is proving to be a simple therapy in the taming of a rare, involuntary movement disorder, that had defied treatment.

A 33-year-old patient from Bengaluru in October and an 11-year-old child from Paris in June, benefited from this fluke or chance treatment, according to scientists.

The efficacy of caffeine as a treatment of dyskinesia,caused by a mutation in the ADCY5 gene is causing waves in medical circles.

Dyskinesia refers to a family of disorders characterised by violent and involuntary muscle movements.

Caffeine is known to be against adenosine. Thus it prevents formation of excess cyclic AMP, which is responsible for the involuntary movements, by inhibiting its synthesis through blocking of adenosine receptors in the brain.

Scientists at the Centre for DNA Fingerprinting (CDFD), Hyderabad presented the case of 33-year-old Ramesh (name changed) who complained of involuntary movements of the limbs and face since the age of five.

This would occur 10 to 15 times per day and last for a few minutes. They were also affecting his daily routine. Such features are termed as 'paroxysmal dyskinesia'. Alterations in more than 12 genes are known to result in this phenotype.

Since many genes needed to be tested, an advance genetic testing method, called exome sequencing analysis was done by a team led by Aswin Dalal, Head, Diagnostics Division CDFD to identify genetic basis in this patient. The sequencing revealed a mosiaic mutation in ADCYS (adenylate cyclise) gene.

Ramesh had consulted neurologist Kuldeep Shetty and medical geneticist, SJ Patil at the Mazumdar Shaw Medical Centre, Narayana Hrudayalaya Hospitals, Bengaluru.

His condition is not very responsive to the currently available medication (like benzodiazepines, betablockers, tetrabenazine, anticholinergics etc).

The CDFD scientists, inspired by the case study in France decided to try out the coffee regimen. They found to their surprise a 90 per cent reduction in his involuntary movements within few days.

Although novel therapies are being developed for some genetic diseases, they are very expensive and and are not accessible to the public. "An accurate diagnosis of genetic disease is very important for proper management and prognosis," they said.

In June 2019, a group of scientists from France reported an unusual case. They found that in a family, the father and daughter were affected with a rare, involuntary movements. They were also having a mutation in the ADCYS gene. However, the consumption of coffee lead to a dramatic reduction of unwanted movements.

To cross check the results, they repeated the therapy on an 11-year-old boy with the similar symptoms. The boy was given three doses per day of coffee (in the form of espresso coffee).

To their utter surprise, they found that his had symptoms dropped from 30 bouts per day to one or two brief movements within few days of coffee treatment. Interestingly, the movements re-surfaced when the patient was given decaf coffee and disappeared again when caffeine containing coffee was given.

ADCY5-related dyskinesia is a roughly one-in-a-million disease, and there is no known cure. The gene in its normal state provides instructions for making an enzyme that helps to regulate muscle contraction. The mutation disrupts that process, and caffeine helps to restore.

See the original post:
Coffee therapy for rare genetic disease related to violent and involuntary muscle movements - BusinessLine

Recommendation and review posted by Bethany Smith

Editor's note: Seeking Alpha is proud to welcome Kerhan Woo as a new contributor. It's easy to become a Seeking Alpha contributor and earn money for your best investment ideas. Active contributors also get free access to SA PREMIUM. Click here to find out more

As a professional in the genomics community, I have been following Invitae (NYSE:NVTA) with great interest. However, I cannot understand why investors are so bullish on its long-term prospects when it continues to see such large losses. There are lots of ideas on how Invitae could become incredibly profitable in the future, but none of these seem validated for their particular circumstances. I wanted to write this article to question the mechanics of how Invitae will become profitable.

Invitae is democratizing germline genetic testing by making it affordable. With its low prices and execution at scale, it has grown revenues quickly. But it still keeps reporting more losses each year.

Source: Invitae 10-K data

This isn't unique to Invitae. Many of their competitors also make increasing losses, such as Fulgent Genetics and several private companies (private data). The exception is Myriad Genetics, which has diversified from the hereditary cancer testing market.

Myriad Genetics aside, Invitae's scale of revenues/losses are greater than many of its peers, as is its valuation. Investors consider its current losses as inevitable as Invitae invests extensively to maintain its growth trajectory. This is much like many other "tech" stocks; Invitae seems highly valued because fast growth in a nascent market might translate into market dominance, profitability and/or a high-valued acquisition. This figure shows how new tech markets develop over time with a winner-takes-all outcome, while the market becomes more highly valued. In the genomics industry, many of us suspect this could happen too for germline testing.

Source: PlayBigger

In addition, tech stocks often command higher multiples because their fixed costs are low, there is long-term value in the data that is being collected, and the marginal cost of adding new customers is low. Tech companies are given flexibility by their investors to sacrifice profitability at each transaction to prioritize growth as they plan to use the collected data as a revenue-generating asset.

There are reasons to be optimistic that this could happen for Invitae too. The germline testing market is indeed growing fast as reimbursements increase and expand, there is increasing awareness of genetic testing in the broader medical community, and Invitae has built a good brand and service globally.

However, as investors you may want to consider these risks.

Tech companies form monopolies because of some type of moat.

Companies like Genomic Health and Myriad Genetics also sell genomics tests, however their emphasis is on products that give them a competitive edge with discernible scientific differences. This could be through lab-developed-tests based on processes from discovery through to validation and reimbursement, and then applying a "blockbuster" financial model.

Invitae in comparison competes in the germline testing market, where there is limited differentiation between tests, creating a commodity market. These labs set up tests by assembling a pipeline using established methods that are shared across the community, free or otherwise. For example, these include mostly the same sequencing technology (Illumina), a bioinformatics pipeline often using similar tools (e.g., GATK), variant curation that uses publicly available databases to filter out irrelevant variants and identify suspect ones (e.g., ExAC, OMIM), and a scoring system for pathogenicity that is widely accepted (ACMG guidelines). The closest thing to a competitive advantage in this space is Myriad Genetics' database of BRCA variants, a large number of which are not shared publicly.

There is limited lasting advantage to be gained by Invitae in performing more testing than their competitors. Comparisons with Amazon's one-stop-shop model isn't appropriate. Amazon is a channel. Genetics is a small part of most physicians' day, so it is not likely to become a channel in the same way. If anything, the one-stop-shop needs to be a big pathology company (e.g., LabCorp or Quest), a channel that has integrated the genetic testing capabilities of Invitae.

Besides, in germline testing, loyalty is often attached to low prices. This has been shown by how easily Myriad Genetics' market has eroded once their customers were offered cheaper options.

Some labs may fine-tweak this better than others to improve efficiency. However, this does not manifest as a competitive advantage. Customers rarely do "bake-offs" to see which lab performs better for a range of difficult cases. Turnaround times are usually 2-6 weeks, with only limited demand to make this even quicker. At best, one could argue that it cuts down on costs and therefore prices, which has become Invitae's key differentiation.

Invitae's success is based on taking an established market and growing with it. Because of the lack of differentiation in this market since AMP vs. Myriad, market-leading low prices have helped grow Invitae's contracts with insurers and institutions. This is reflected in how Myriad Genetics' cancer revenues started to decrease after Invitae and others emerged with lower prices.

Source: Myriad Genetics 10-K data

An average COGS of $252/per sample compared with ASPs of $440 is too simplistic a calculation because some of their tests have multiple prices, and with the introduction of NIPT and whole exome sequencing, the cost basis for some for their tests varies. Some of their prices have been broken out in a previous Seeking Alpha article, which demonstrate how Invitae is selling below their cost for some of their tests and rely heavily on Medicare (and investors for that matter) to subsidize their price sensitive customers.

The most obvious example of selling below cost is their private-pay NIPT solution that is outsourced to Illumina at a cheaper price than what Illumina charges. I'm aware the typical prices for NIPT are higher, and Invitae also implied this in their earnings call explaining that their average COGS (currently $252) had actually increased in part due to NIPT.

Through my experience working for instrument vendors and also the labs, through several off-the-record conversations with many of their competitors globally, the gossip is that Invitae is boldly selling below cost when it needs to. I've heard that one of their competitors have considered making an official complaint about this in the country they operate in. And when I asked one of Invitae's sales reps about this strategy at a conference, she smiled nervously and crossed her fingers.

You may also want to consider this: Invitae currently makes >93% of their revenue from US markets, which for now is great for them because outside of the US they tend to sell their tests at $250. However, about half of the global market for genetic testing is outside of the US (depending on the report you read), so if they need to continue expanding, their ASPs could drop further.

At the same time, how much R&D and sales & marketing costs can they strip away? Because of its valuation Invitae also needs to maintain its growth trajectory, cutting down on these areas creates risks to their long term growth. Contracts will give them some immunity to competition, but for how long if other competitors start to provide even lower prices?

Invitae has done an admirable job to grow as they have over the last few years. But how much more funds do they need to raise to keep this up? At what rate must they continue growing at to maintain your optimism, and for how much longer, before they demonstrate how they can convert their market dominance into sustainable profits?

I don't know the answer to that. But in echoing a recent tongue-in-cheek article from The New York Times, I do know that there has never been a better time for a patient without insurance coverage to get a genetic test.

The genomics market has grown at phenomenal rates regardless of which marker you want to use. 30% per year could be inferred from Illumina's data, which is much higher than pathology in general, which for example is in the single digits for Quest and LabCorp.

There is no doubt that some areas of testing will grow fast. But only a handful of Invitae's tests have actually progressed through to routine use. A market for a pathology test is created by meeting milestones, more or less in this order: analytical validity, clinical validity, clinical utility, professional guidelines, and finally reimbursement. In different geographic regions, different germline tests have been taken through this journey. As controversial as Myriad Genetics have been, we can thank them for helping to developing the acceptance of testing for hereditary breast and ovarian cancer.

Although Invitae is trying to help generate evidence to influence professional guidelines, it is not feasible to try moving the market towards reimbursement and regular usage for all their tests. Therefore their growth challenges aren't competition with other labs, but are (a) tests that aren't reimbursed and/or (b) physicians not ordering the test when they should.

To understand (a), we can use Myriad Genetics as a comparison. Myriad Genetics identifies clinical areas of interest and develops a specific test following the process described above, validating it extensively. This will help provide evidence to influence professional guidelines and eventually get some reimbursement approvals for their unique test. This model has its own risks - it relies on trials and is very slow - but to some extent, these market factors remain within their control.

On the other hand, "by leaning on well-scrubbed, publicly available data from studies of gene variants and rare disorders, Invitae doesn't need to run lots of expensive clinical trials on its own." This suggests Invitae relies on the various markets to mature on their own, without having to contribute extensively. Whether a test for say, dilated cardiomyopathy, is considered essential and not just investigational to justify reimbursement, is not in Invitae's area of influence.

To understand (b), one the biggest problem we have in germline diagnostics is tests not being ordered when they should be. Outside of the well-known areas like cancer, when a physician comes across appropriate patients they might not remember, or even want to use the test.

Invitae themselves are aware of this risk, stating that "clinicians in other areas of medicine may not adopt genetic testing for hereditary disease as readily as it has been adopted in hereditary cancer."

Not using a genetic test may not be detrimental for patients. Often, a genetic test is seen as supplementary to the diagnosis. A patient with dilated cardiomyopathy for example, can be diagnosed and managed without germline testing. Yes, a test would help, especially to identify which of their family members are at risk. But quite often, a cardiologist can make the diagnosis without genetics. It is not essential in most cases that they see. This isn't to say that cardiologists aren't ordering these tests, but it may take a while before the majority of cardiologists start to do the same. Genetic testing instead will be taken up where it dictates treatment such as for hereditary breast and ovarian cancer. But unfortunately, many germline tests are not paired to treatment outcomes.

There's a lot more cancer testing to be done, but Invitae's growth can't just be through cancer. Quoting Invitae again: "A lack of or delay in clinical acceptance of broad-based panels such as our tests would negatively impact sales and market acceptance of our tests and limit our revenue growth and potential profitability." For that to happen for other diseases, the rate of reimbursement and broad changes in physician practice may be the limiting step.

There are other ways to grow Invitae's markets, and the direct-to-consumer (DTC) market is being considered. However, expectations need to be managed. The DTC market has grown, but most of it is for ancestry uses. Color Genetics started out focusing on reaching women and offering BRCA testing at low prices, but has had to expand its markets from the DTC market to other ones, suggesting limits to their initial market. Sema4 have articulated their difficulties in this market too. Invitae may be able to learn from these companies and get a better shot, but it won't be without increasing competition from ancestry companies themselves expanding into healthcare services.

Tech companies have relatively low fixed costs. Frankly, they don't have to sequence each user's DNA and there is supplier competition.

I don't mean to state the obvious, but unlike traditional tech companies, Invitae and others in this industry have high costs to collect, sequence, and analyze each new "user." Health datasets are arguably more valuable than social media ones, but it costs a lot more.

Sequencing costs aren't coming down as quickly as it did previously. Over 2007-2015, the cost of sequencing plummeted, but this has since tailed off.

Source: National Human Genome Research Institute

Illumina in recent years does not feel the same competitive heat they did a decade ago. Although they still have competitors in their research markets, none are close to breaking in on their grip on the clinical market. This has been demonstrated many times but most recently with Qiagen ending their own sequencer's developments and instead partnering with Illumina. As a monopoly, Illumina gets to choose when to develop lower-cost sequencing, and have previously shared that they first need to investigate the elasticity of the clinical market. In the meantime, they can continue to be the arms suppliers for all parties in this price war.

There are other costs that can't be whittled away much further, such as library prep, collection costs, and staff time. As a result, there is a floor that probably isn't too far away.

If data is expensive to generate, what is this data that Invitae is getting?

It is not yet clear how much of the data Invitae has can be reused. Patients can opt out of having their data re-used, and in Europe patients have to opt in.

Consent for further use of data is taken more seriously in the medical community than in the DTC market. Physicians I've met don't sign their patients up for data exchanges, they simply want a test that is performed for the one question they are trying to answer at that point in time. This is important to understand. Even in a values-outcome healthcare system that we are moving into, pathology is still a question-then-answer model between a physician and the lab, not this multi-use and multi-direction relationship between several physicians, the lab, and their patients. This may limit the allowance provided to Invitae to re-use the data, even when de-identified.

Invitae uses a large sequencing panel to generate data for the patient being tested. This may have changed recently with more and more labs running all tests on whole exomes so that the data is uniform, but reading between the lines into Invitae's marketing materials and their COGS suggest that this isn't routine yet. This means that the data they are collecting is both lacking (by sequencing panels only and not the whole exome or more) and likely inconsistent, reducing the power of the dataset they are amassing. Genomics England in comparison sequences the entire genome for all their patients.

In addition, it is not clear how much clinical data Invitae is getting with each sample, which is crucial to bring meaning to the genomic data. Although physicians are encouraged to provide as much information as possible when a test is requested, my experience is that most of the time you just get a few relevant snippets of information. It would be specific to the clinical indication relevant to the test ordered, not across a whole range of medical information. For example, a patient may get tested for the breast cancer genes, so Invitae may collect information on their breast cancer diagnosis, personal, and family history. But would they get information on other medical conditions deemed irrelevant at the time? Would they get updated information after the test is performed and there is no more interaction with the lab? I doubt it, because Invitae's customers are in the business of diagnostics, not research.

The research arena is designed to generate genomic data paired with a rich treasure trove of clinical data that is also longitudinal. This is what "population genomics" is about, and the high water mark is the UK biobank where an extensive number of participants are documented extensively. Illumina is tracking about 50 other programs and many of these are undertaken by healthcare systems themselves because of the extent of data required and the research context.

Invitae and other labs are also getting into this business to support these projects. These include Color Genomics and Helix - both of whom had to expand upon their earlier business models to both grow revenue and collect more data. But I haven't seen evidence to suggest that Invitae has taken any sustainable leads here.

Finally, if data is their play, I cannot fathom why they got into the NIPT market given the genomic and clinical data they would get from each patient will be limited.

If Invitae sells lower-than-cost tests even when they operate on their scale, and without a moat they cannot just flip on a switch and raise their prices once they take a monopoly, they have to use this data for some future revenue stream. What are their options?

Sales of datasets to pharma:

Pharma companies benefit from these datasets to help them identify potential drug targets (e.g., what variants are correlated with a symptom that could be targeted) or to identify patients (e.g., which diagnosed patients with a relevant mutation they could trial a new drug on). One early example of this first model is Amgen purchasing deCode back in 2012 for its database, although I have been told that this has not led to much return on investment.

Invitae's datasets may not be fully consented for their research use, and may have patchy genomic and clinical information. 23andMe in comparison has built a product for this business model, collecting extensive non-genomic information from their patients. Although their genomic dataset is sparser, it is generally consistent.

From an R&D perspective, if pharma had to choose which was more important, they may prefer better clinical information linked to sparse genomic information over deep genomic information linked to sparse clinical information. This is because the clinical information helps them identify disease/symptoms/patients, while 23andMe's genomic information might provide sufficient "tags" that they can drill down on later. The genomic dataset from 23andme, although sparse, has the advantage of being genome-wide and the tool they use is designed for this purpose. I am not sure whether 23andme generates useful clinical information - I imagine it is hard through a DTC setting - but perhaps it is more harmonized between samples because they are in direct contact with their customers. Overall, this tells me that while Invitae could try selling to pharma companies, it remains to be seen what advantage they have and whether pharma will continue making these investments.

The alternative for pharma is to generate this data themselves, as Regeneron are doing.

Sales to pharma to help them generate data:

If pharma decides to generate their own dataset, they might outsource the collection, data generation and data analysis. This is what several of Invitae's competitors do too, including Fulgent Genetics (NASDAQ:FLGT) and Centogene (OTC:CNTG).

I've noticed in comments across SeekingAlpha articles that these two services to pharma are often confused. This second example has little to do with Invitae's dataset, but more to do with treating pharma as an institutional customer and providing "sequencing-as-a-service." This is a hotly contested market as non-clinical labs also compete in this space - these are the labs that hold the vast majority of Illumina's sequencers. If you thought germline testing was a race to the bottom, this market is probably already at the bottom and will bring down a lab's ASPs even further. This is not the market you growth investors are looking for.

Selling re-usable pathology tests to doctors:

I said that pathology is a transaction business model at present, rather than this multi-sided, multi-use model. The truth though, is that many doctors would love to see this new precision medicine future where pathology does not follow a use-and-dispose model.

This fits with Invitae's "Genome management" future, where the data is potentially re-usable but Invitae has not described in much detail how this will all work. This future will no doubt come in some shape or form, but the size of this change is monumental.

Source: Invitae Q2 2019 slides

It is beyond the scope of this article to describe on how a healthcare system might go through such a large, large, change. It requires changes in the way reimbursement works, the way EHRs work, and the way physicians are trained. It will likely need large numbers of trials and other data to validate this new approach, and cross-functional cooperation and funding on an enormous scale. There are studies being completed but who can say how long this future will take to be prepared? While this is the dream for medicine and a key reason I work in genomics, the system is not in place yet and there are no consensus road maps on how we reach this "genome management" future.

This is similar to the ideal future of all of us using fully driver-less cars one day. The technology for cars seems to be getting there, but this is not the only critical development required. There are factors like infrastructure, incentives, habits, etc, that also need change but aren't easy to. Illumina recognizes this too, and this may have played a factor as to why it hasn't prioritized dropping their prices.

By being the central testing lab for a range of genomic tests, Invitae will no doubt be a valuable company when this future arrives. But they need to be careful to avoid being a "Moses company." Moses featured in the Bible's Old Testament with a vision to bring his people to a new Promised Land. Although the vision was accomplished, he died before it came to pass, and instead his successor was the person who brought the people over. Invitae's vision is inspirational, but will they bring us to this Promised Land, or will they instead be laying the groundwork for other labs to do so?

I think that while monetizing data might be a useful trick to keep up its sleeve, without a validated monetization model that will work for their particular set of opportunities and challenges, Invitae might need a more mainstream business model to reach profitability to fall back upon.

Could it continue to grow as a pathology company? Invitae doesn't seem to want to compare itself to the Quests and LabCorps of the pathology industry. But if it wants to reach even more patients, what is the path forward - especially with its financial situation?

It may need to consider selling itself to one of these big pathology organizations. Genomics is an isolated pathology discipline and won't become mainstream until it is integrated with other pathology activities. For example, many pathology tests are ordered in groups by physicians and there is convenience of using the same vendor. These can be linked together so that physicians can "reflex" from one result to the next appropriate test to order. Genetic testing must be included in this pattern in order for it to become mainstream.

For this value to be realized, Invitae could be valuable to a large pathology company, because physicians are more likely to want the "Amazon of pathology," not the "Amazon of genetics." LabCorp and Quest Diagnostics (NYSE:DGX) have already purchased other germline testing labs for this purpose. It is not clear whether this has led to any synergies yet.

Invitae continues to grow its testing volumes quickly, and as a result so has its valuation. But at some point it needs to deliver profitability, either to make money off each transaction, and/or find a new business model. Invitae has grown fast primarily because of its low prices and ability to execute testing at scale. It needs to maintain its low prices because it is otherwise difficult to create a differentiator in this market.

If Invitae waits for reagent costs to go down, it has limited bargaining power, and the floor is near. If Invitae cuts into its fixed costs it runs the risk of slowing down its growth, which is risky given how it is valued. Invitae also needs the market to continue growing, much of which is not under its control.

If Invitae wants to find a new business model to capitalize on its stockpile of data, it needs to articulate this vision more specifically and validate it with the wider healthcare community. If Invitae wants to partner with a large pathology organization, it may need to validate how genetics is integrated with other areas of healthcare.

As we have found with WeWork and other recent news stories, we need to be careful not to value non-tech companies as tech companies. Even though Invitae will benefit from tech - such as its culture and ability to raise funds, it is still hampered by restrictions found in a traditional and slow moving pathology industry, regardless of whether genomics is a little quicker.

Invitae needs to validate how it really intends to make money. I write this humbly acknowledging that I am not in a position to take these questions to Invitae's senior management themselves. And I don't take pleasure in knocking Invitae. I work in their industry, have gained from their contribution, and am excited by their growth and success. However, if you are an investor who believes that Invitae's valuation will continue to rise because it is dominating a fast-growing market and collecting interesting data, make sure you ask hard questions about how this can be materialized into profits.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Invitae Needs To Validate How It Really Intends To Make Money - Seeking Alpha

Recommendation and review posted by Bethany Smith

SALT LAKE CITY, Oct. 28, 2019 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in molecular diagnostics and precision medicine, today announced that it will present results from seven studies at the 2019 National Society of Genetic Counselors (NSGC) annual meeting being held Nov. 58, 2019 in Salt Lake City.

"We are excited to present new data from seven studies at this years NSGC meeting," said Susan Manley, MS, CGC, MBA, senior vice president of Medical Services at Myriad Genetics. Our presentations highlight the companys commitment to advancing precision medicine in oncology and womens health.

A list of presentations at 2019 NSGC is below. Please visit Myriad Genetics at booth #711 to learn more about our leading portfolio of precision medicine products. Follow Myriad on Twitter via @myriadgenetics and follow meeting news by using the hashtag #NSGC19.

myRiskHereditaryCancer

ForesightCarrierScreen

AishwaryaArjunan

PrequelTMPrenatalScreen

About Myriad myRisk Hereditary CancerThe Myriad myRisk Hereditary Cancer test uses an extensive number of sophisticated technologies and proprietary algorithms to evaluate 35 clinically significant genes associated with eight hereditary cancer sites including: breast, colon, ovarian, endometrial, pancreatic, prostate and gastric cancers and melanoma.

AboutForesight Carrier ScreenThe Myriad Foresight Carrier Screen is designed to maximize detection of at-risk couples for serious, prevalent, and clinically-actionable conditions. Foresight has a rigorous disease selection that focuses on 175+ conditions that provides meaningful information to patients. Additionally, Foresight offers superior technology with unmatched detection rates for the vast majority of genes on the panel (>99% across ethnicities) which means patients can trust both positive and negative results.

About PrequelTM Prenatal ScreenThe Myriad Prequel Prenatal Screen is a noninvasive prenatal screen that uses cell-free DNA (cfDNA) to determine if a pregnancy is at an increased risk for chromosome abnormalities, such as Down syndrome. Prequel has been shown to be superior to screening methods that use maternal age, ultrasound and serum screening. Additionally, Prequel has a lower false-positive rate and false-negative rate than these other methods. The Prequel Prenatal Screen can be ordered with the Foresight Carrier Screen and offered to all women, including those with high body mass index, and ovum donor or a twin pregnancy.

About Myriad GeneticsMyriad Genetics Inc. is a leading precision medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on five critical success factors: building upon a solid hereditary cancer foundation, growing new product volume, expanding reimbursement coverage for new products, increasing RNA kit revenue internationally and improving profitability with Elevate 2020. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice HRD, EndoPredict, Vectra, GeneSight, riskScore, Prolaris, Foresight and Prequel are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to data being presented for its genetic tests at the 2019 National Society of Genetic Counselors Meeting being held Nov. 58, 2019 in Salt Lake City; and the Company's strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decision in the lawsuit brought against us by the Association for Molecular Pathology et al; risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2019, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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Myriad Genetics to Present Seven Studies at the 2019 National Society of Genetic Counselors Annual Meeting - BioSpace

Recommendation and review posted by Bethany Smith

Ninety-seven percent of potential new cancer drugs never make it to market, dropping out of clinical trials when they dont meet measures of safety or efficacy.

Why that is, we dont really know. But I think that this extremely high failure rate suggests that there are some fundamental issues in how new drug targets are studied and how new drugs are characterized, said molecular biologist Jason Sheltzer, PhD, an Independent Fellow at the Cold Spring Harbor Laboratory on Long Island, NY.

He decided to investigate, and uncovered the potential power of publishing negative evidence. The work fits in with Open Access week here at Public Library of Science.

CRISPR Improves Precision

The team reports inScience Translational Medicineon using the gene editing tool CRISPR-Cas9 to test whether 10 experimental cancer drugs work exactly how their developers predicted they would. And they found a tunnel vision in the way that drugs are targeted that might explain why certain patients do not respond as hoped.

Like an arrow that hits a tree rather than the bullseye, some cancer drugs may not actually reach their targets but many studies werent designed to reveal this. And so when results look promising, the drug candidate progresses through the FDA labyrinth.

Sheltzers strategy was straightforward: use CRISPR to remove the purported target, and if the drug still works, then the target isnt really the target. Perhaps preclinical research that identified a molecule as the drug target was halted, the scientists concluding success, when something fit the bill.

The researchers tested drugs that are either in clinical trials or once were, or are in preclinical studies (animals or human cells) not cancer drugs currently on the market. The experiments were done on standard cell lines from cancer patients.

The idea for many of these drugs is that they block the function of a certain protein in cancer cells. We showed that most of these drugs dont work by blocking the function of the protein that they were reported to block, Sheltzer explained.

Using CRISPR provided greater precision in interrogating potential drug targets than the older method, RNA interference. RNAi knocks down gene expression rather than snipping out a gene like CRISPR can.

Might a small molecule bind more than one type of target, like shooting arrows that hit trees and bushes as well as the bullseye? And sometimes what seems to be a valid drug target in vitro isnt exactly what happens in a body.

But a drug can make it to market without anyone knowing exactly how it works. Thats the case for selective serotonin reuptake inhibitor (SSRI) anti-depressants. The cartoons in ads depict neuromuscular junctions with the drug keeping serotonin in synapses longer by binding the reuptake proteins, presumably offsetting a deficit behind the symptoms. But googling SSRIsreturns the exact mechanism of actionof SSRIs is unknown.

From Slash-and-Burn to Hitting Targets

The new cancer drugs work in a few ways. Some of them zero in on molecules specific to cancer cells. These include:

These targeted drugs offer an alternative or adjunct to traditional drugs that broadly kill many types of rapidly-dividing cells, not just the cancer cells.

The targeted drugs began with Herceptinin 1998, its inventors recently honored with a Lasker award. The FDA approved another hugely successful targeted cancer drug, Gleevec, in just a few months in 2001. Today melodramatic ads pitch the new arsenal of cancer treatments: Zelboraf, Tafinlar, Keytruda, Opdivo.

But targeted drugs can fail if a new mutation alters the target or cancer cells find an alternate pathway that hikes cell division rate.

The researchers took a dual experimental approach based on logic:

Part of the confusion, I think, is semantic. Sometimes we deem a chemical interaction off-target if it doesnt do what we designed it to. Maybe our expectations were wrong. To be more unbiased, some researchers alter the language, calling the reliance of a cancer cell on a particular protein an addiction and investigating to seek druggable cancer dependencies.

The teams work indicates that what was deemed on-target may really be off-target, and vice versa. Perhaps its time to retire those terms.

The First Drug Tested

Earlier, Sheltzer investigated a protein called MELK, to which a company, OncoTherapy Science, is developing an inhibitor, called OTS167. Because MELK (maternal embryonic leucine zipper kinase) is abundant in many tumor types, it was presumed to be essential for their growth and therefore a drug target. But when CRISPR removed the gene that encodes MELK protein, nothing happened.

To our great surprise, when we eliminated these proteins from the cancer cells, they didnt die. The cancer cells continued to grow just fine, in spite of what had previously been published. They just didnt care about MELK, Sheltzer said.

The group published the findings on MELK in 2017, in eLIFE, raising the possibility that OTS167 is perhaps barking up the wrong tree. The drug candidate is in a phase 1(safety) trial for solid tumors and is recruiting for a phase 1trial for triple negative and metastatic breast cancer.

The MELK story inspired the group to use their genetic target-deconvolution strategy to see whether 10 other drugs were actually hitting their supposed targets. About a thousand cancer patients in total are taking one of these drugs in clinical trials.

Another Misguided Drug Reveals a Novel Target

In the new paper, the investigators question another drug, OTS964, being developed to treat certain lung and breast cancers. In the process, theyve discovered a new druggable cancer target.

RNAi had indicated that OTS964 targets a protein called PBK. But CRISPR told a different story cells with PBK gone still succumbed to the drug. It turns out that the interaction with PBK has nothing to do with how the drug actually kills cancer cells, Sheltzer said.

To find out how the PBK-targeting drug works, the researchers applied huge amounts of it to cancer cells and then gave the cells time to acquire mutations that would enable them to resist the drug. Cancer genomes are inherently unstable, mutating often. When a mutation renders a cell resistant to a drug, that cell then has an advantage and soon takes over the tumor.

Discovering how a cell circumvents a drug is priceless information.

The resistance experiments revealed that the cancer cell vulnerability that candidate drug OTS964 taps into isnt PBK after all, but a gene that encodes the protein CDK11. Its a cyclin-dependent kinase, an enzyme that is part of a pathway that leads to cell division.

The FDA has already approved CDK4/6 inhibitors, starting with Ibrance, in February 2015, to treat certain types of breast cancer. CDK11 is a brand new target. And thats potentially huge.

Whats Next?

At a news conference the researchers addressed concerns that their findings will affect people already taking targeted cancer drugs but they maintained that their work did not discover any approved drugs that were hitting trees instead of bullseyes.

But what about ongoing clinical trials for cancer drugs?

Sheltzer tried to alert folks running the trials. I filed a FOIA with the FDA to try to get additional information on the safety and efficacy of these drugs. The FDA declined to share that data, and said that it was a trade secret up until the point that these drugs received FDA approval.

He contacted companies sponsoring clinical trials too, but they wouldnt disclose any information either.

I think that the secrecy and the opacity in this drug development process really hurt scientific progress. A lot of drugs tested in cancer patients tragically dont help cancer patients. If this kind of evidence was routinely collected before drugs entered clinical trials, we might be able to do a better job assigning patients to therapies that are most likely to provide some benefit. With this knowledge, I believe we can better fulfill the promise of precision medicine, Sheltzer said.

The drug companies would do well to pay more attention to basic scientists who figure out how things work or dont work like Sheltzer. Using CRISPR can enable researchers to do a better job finding cancers central genes and a better job validating a drugs on-target mechanism of action. We think that that kind of preclinical foundation will help clinicians design better clinical trials to decrease the failure rate of new drugs, Sheltzer concluded.

Originally posted here:
When the Target Isn't Really the Target: One Way Cancer Drugs Fall Out of Clinical Trials | DNA Science Blog - PLoS Blogs

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass., Oct. 24, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report third quarter 2019 financial results after the Nasdaq Global Market closes on Thursday, November 7, 2019. Subsequently, at 4:30 p.m. E.T., the Company will host a conference call to discuss its third quarter 2019 financial results and to provide a corporate update.

The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 8998299. Please specify to the operator that you would like to join the "Sarepta Third Quarter 2019 Earnings Call." The conference call will be webcast live under the investor relations section of Sarepta's website at http://www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics to Announce Third Quarter 2019 Financial Results and Recent Corporate Developments on November 7, 2019 - BioSpace

Recommendation and review posted by Bethany Smith

Image: Breast cancer cell. Image credit:Anne Weston, Francis Crick Institute. Licence: CC-BY-NC-4.0.

Scientists in ourDivision for Breast Cancer Researchhave been involved in some of the most famous discoveries in the history of breast cancer research.

They discovered the BRCA2 gene, for example, and over the past 20 years have been pioneers in establishing the concept of synthetic lethality in cancer research and treatment. Their work underpinned and stimulated the development of olapariband other PARP inhibitors now standard treatments for genetic breast and platinum-sensitive ovarian cancers.

In the past five years, results of a clinical trial co-led by Professor Andrew Tutt, Head of our Division of Breast Cancer, have also established carboplatin a drug discovered at the ICR as a better treatment forBRCA1 andBRCA2mutated forms of advanced breast cancer than the usual breast cancer standard.

Many of our successes in breast cancer research have come from The Breast Cancer Now Toby Robins Research Centre at the ICR.

The integrated teams at the ICR, together with colleagues at our partner hospital The Royal Marsden NHS Foundation Trustand through Breast Cancer Nowprogramme integration with Kings College Londonand Guys Cancer Centreat Guys and St Thomas NHS Foundation Trust, have committed to define what they term the evolving unmet need for breast cancer research by 2025 with funding from Breast Cancer Now underpinning these ambitious plans.

The Division of Breast Cancer Research, which incorporates theBreast Cancer Now Research Centre, contains over 100 scientists and clinicians working in 12 teams.

Find out more

Researchers at the Centre lead important programmes to understand the genetic and environmental causes of breast cancer, and how a tumours molecular biology and surrounding microenvironment affect its behaviour and response to treatment.

They are also working on identifying new therapeutic approaches to breast cancer, and understanding the mechanisms tumours use in becoming resistant to treatment.

In a recent review of the Centres funding by Breast Cancer Now, Professor Tutt and colleagues across the centre set out a raft of successes over the past five years including 398 research manuscripts, two new drug discovery programmes, the development of a new drug candidate, and 13 breast cancer clinical trials.

The Centre is clear in its aims to keep the fundamental science taking place in its laboratories relevant to real patient problems. This includes close collaboration with hospital colleagues, a focus on rarer but biologically distinct forms of breast cancers, the study of tumour evolution during treatment in clinical trials, and extending the impact of findings to broader patient groups.

First, a thank you to Professor Mitch Dowsett, Head of the joint Academic Department of Biochemistry at The Royal Marsden and the ICR. Professor Dowsetts work on the role of hormones in breast cancer has led to the development of both the most effective endocrine therapy treatments used for patients today and the biomarkers that identify those who may benefit most from them helping patients to avoid lengthy treatments and chemotherapy. Professor Dowsett's work on endocrine therapy resistance has been led with Dr Lesley-Ann Martin.

This work will sadly not be continuing in the next five-year round of funding from Breast Cancer Now as Professor Dowsett scales down his work. We are hugely proud of the team's myriad achievements to date, and the centre will continue research into hormones and breast cancer with Professor Stephen Johnston at The Royal Marsden and new appointments to ICR Faculty including Professor Cathrin Brisken.

The approval of the innovative breast cancer drug palbociclibis another example ofthe Centres success in achieving meaningful improvements for patients in the past five years.

Palbociclib is known as a first-in-class drug, because its the first drug to work in the way that it does blocking two proteins, called CDK4 and CDK6 that help cancer cells divide and spread.

Professor Nicholas Turner, Professor in Molecular Oncologyat the ICR and Consultant Oncologist at The Royal Marsden led a key clinical trial of palbociclib, showing that it could slow the progression of advanced breast cancer in conjunction with standard treatments, and substantially extend the lives of patients.

This innovation is already changing lives more than 90,000 patients have been prescribed the drug.

Professor Turner, along with Professor Spiros Linardopoulos, Head of Drug Discovery at the Centre and other colleagues, continue to work on how resistance develops and how it may be targeted.

ICR patient advocate Christine was initially diagnosed with breast cancer in 2012 but, nearly five years after surgery and chemotherapy left her seemingly cancer free, she found the disease had returned, and spread to her brain and bones.

Following radiotherapy, she needed a targeted treatment to keep her cancer at bay. Fortunately, palbociclib had been approved for use on the NHS just months before, thanks to clinical trials that showed how palbociclib could benefit patients.

Christine is doing well on palbociclib: Im on the 21st cycle and my cancer is currently stable, she says. Im able to work part-time, and I can keep up my cycling, which would have been more difficult on conventional therapy.

Professor Linardopoulos leads the Centres work to search for gene targets for which new anticancer drugs can be developed.

A recent success for his team helped to progress work on a new drug candidate effective against fast-dividing cells, including triple-negative breast cancers. The target for the drug is a protein known to play a key part in controlling cell division monopolar spindle 1 (MSP1). The first clinical trial of the new treatment is under way at The Royal Marsden and will shortly expand to other NHS Hospital partners like Guys Hospital.

Professor Spiros Linardopoulos said: This exciting cancer treatment uses cancer's rapid growth against it, by forcing cells through cell division so quickly that they accumulate fatal errors.

With the drug now entering clinical trials, we can see how our translational approach at the ICR to get drugs from the lab to the clinic really has an impact for patients.

Professor Tutt explains that: together with other Team Leaders in the Breast Cancer Now centre at the ICR, there is a focus on driving progress to address niche, harder problems which are biologically distinctive but often not an initial focus for industry.

These are some examples of programmes of work currently taking place across the centre:

Unpicking the functional genetics of breast cancer risk

Dr Olivia Fletcher, Team Leader in Functional Genetic Epidemiologyleads a team of genetic epidemiologists and molecular biologists working in partnership with the Breast Cancer Now Generations Study, the British Breast Cancer Study and other population-based studies.

Population based studies allow us important insights into who genetic variants are associated with breast cancer risk specifically, variants that map to non-coding DNA, and which are as a result difficult to identify. Olivias team are understanding how some of these variants act to drive risks in the hope of identify groups for early detection or prevention strategies.

Targeting neighbouring healthy cells

Professor Clare Isacke, Team Leader in Molecular Cell Biology, is leading work to identify the processes by which tumour cells recruit and activate non-cancerous cells during metastasis, the process of cancers growth and spread from its original site.

The team is identifying strategies to effectively target these neighbouring heathy cells, due to the important part they play in modulating the response of tumour cells to treatment and as a result, tumour progression and resistance.

Mobilising the bodys immune system

Mobilising the bodys immune system after cell death is one major area of focus and innovation for the centre. Professor Pascal Meier, Team Leader in Cell Death and Immunity, is leading a team to explore the complex relationship between cell death, immunity and tumour growth and survival.

In particular, the team is focussing on the role of cell death and inflammation in adaptation to tissue stress, treatment resistance and tumour surveillance.

The team hope that new combination treatments they are trialling will allow doctors to manipulate the types of cell death which occur after treatment with current standard-of-care drugs and emerging targeted therapies, to more effectively mobilise the bodys natural immune response and enable better treatment outcomes.

Exploiting vulnerabilities in different subsets of breast cancer

Biomarkers are biological products like proteins or genes that can be measured in patient samples to detect disease. Biomarker development plays an important role in allowing researchers the ability to identify different tumour subsets with great accuracy, and in turn with a greater understanding of these subtypes for the biomarkers to help predict disease outcome.

Dr Rachael Natrajan, Team Leader in Functional Genomics, is researching the ways in which different subtypes of breast cancer can be characterised through genetic analysis of the tumour, to understand how different subgroups of a cancer and subpopulations of cells or clones become treatment resistant and can cooperate in doing so to drive relapse.

Other teams are leading work to understand the different dependencies and potential new drug targets within different subpopulations of a tumour.

Professor Chris Lord, Deputy Head of the Division of Breast Cancer Research, approaches this as an expert in the field of synthetic lethality. The term refers to when scientists exploit the fatal effects of disruption of the activity of two genes even though either disruption alone would not be fatal for the cells. Combining the effect of mutations in the tumour and use of targeted drugs can allow potent and tumour specific therapeutic effects. His team use tumour cell models and CRISPR-CAS9 gene perturbation techniques to help build their understanding of models of resistance in tumour cells.

Professor Tutt and Professor Lords laboratories are also working together to understand and target DNA repair deficient and genetically unstable breast cancers. Professor Tutts group have a special focus on basal-like Triple Negative breast cancers and create tumour organoid models, using tumours from patients treated with drugs that target DNA damage response and DNA replication stress.

To date, breast cancer research at the ICR has had a huge impact in helping patients assess their risks of developing breast cancer, choose the right treatment options and live longer.

Find out more

Ultimately, the end-goal for everything taking place in our Division of Breast Cancer Research is to improve the outcome and quality of life of women with or at risk of breast cancer.

Professors Tutt and Turner, as practicing oncologists and trial leaders, work with many others in the Centre to translate findings through to clinical trials using an integrated and patient-focussed strategic approach.

The centre also works closely with the wider national Breast Cancer Now network of research centres, and the international breast cancer research community. In partnership with The Royal Marsden, the Centre acts as a beacon for discovering therapy approaches and developing proof of concept early phase and international practice changing late phase trials.

October is Breast Cancer Awareness Month a good time to reflect on the Centres work ahead, as 2020 draws close and the next five-year phase for the Centres strategy begin in earnest. We know the work is in good hands.

See the original post here:
How ICR researchers are leading the way in breast cancer research - The Institute of Cancer Research

Recommendation and review posted by Bethany Smith

The Viral Vector Manufacturing Market research report 2019 has been estimated considering the application and regional segments, market share, and size, while the forecast for each product type and application segment has been provided for the global and regional markets. Viral Vector Manufacturing report offers detailed profiles of the key players to bring out a clear view of the competitive landscape of the Viral Vector Manufacturing outlook. It also comprehends market new product analysis, financial overview, strategies and marketing trends.

In Viral Vector Manufacturing Market Report, Following Companies Are Covered:

Get a Sample Copy of the Report https://www.industryresearch.co/enquiry/request-sample/14099017

Market Overview:

Scope of the Report:

The Research Document Will Answer Following Questions Such as:

For More Information or Query or Customization Before Buying, Visit at https://www.industryresearch.co/enquiry/pre-order-enquiry/14099017

Key Market Trends:

The Cancer Sub-segment is Expected to Grow Faster in the Disease Segment

In the field of oncology, viral vector-based gene therapy has demonstrated steady progress. A multitude of viral vectors has been engineered for both therapeutic and preventive applications, in cancers. A critical development in viral vector-based cancer therapy has been the application of engineered and naturally occurring oncolytic viral vectors. These vectors are programmed to specifically replicate inside the cancer cells and induce toxic effects, which ultimately results in apoptosis. The attractive features of viral vectors relate to their capability to provide high levels of transgene expression, in a broad range of host cells.

The high demand for effective therapeutics for the management of cancers, the presence of fast track approval process, and the prospects of novel drugs to turn into blockbuster products are primary reasons responsible for the significant R&D investments in the field of viral vector-based cancer therapeutics, which, in turn, is driving the markets growth.

North America Dominates the Market and is Expected to Follow the Same Trend in the Future As Well

North America currently dominates the market for viral vector manufacturing and is expected to continue its stronghold for a few more years. The United States holds the largest market share in the North American region owing to factors, such as the high adoption rate of new therapies and high awareness of the general population. The increasing prevalence of genetic and chronic disorders, such as cancer, an aging population, growing demand for targeted and personalized medicine, and favorable government initiatives are the factors responsible for the significant market size in the United States.

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Detailed TOC of Viral Vector Manufacturing Market Report 2019-2024:

1 INTRODUCTION1.1 Study Deliverables1.2 Study Assumptions1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS4.1 Market Overview4.2 Market Drivers4.2.1 Rising Prevalence of Genetic Disorders, Cancer, and Infectious Diseases4.2.2 Increasing Number of Clinical Studies And Availability of Funding For Gene Therapy Development4.2.3 Potential Applications in Novel Drug Delivery Approaches4.3 Market Restraints4.3.1 High Cost of Gene Therapies4.3.2 Challenges in Viral Vector-manufacturing Capacity4.4 Porters Five Forces Analysis4.4.1 Threat of New Entrants4.4.2 Bargaining Power of Buyers/Consumers4.4.3 Bargaining Power of Suppliers4.4.4 Threat of Substitute Products4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION5.1 By Type5.1.1 Adenoviral Vectors5.1.2 Adeno-associated Viral Vectors5.1.3 Lentiviral Vectors5.1.4 Retroviral Vectors5.1.5 Other Types5.2 By Disease5.2.1 Cancer5.2.2 Genetic Disorders5.2.3 Infectious Diseases5.2.4 Other Diseases5.3 By Application5.3.1 Gene therapy5.3.2 Vaccinology5.4 Geography5.4.1 North America5.4.1.1 US5.4.1.2 Canada5.4.1.3 Mexico5.4.2 Europe5.4.2.1 Germany5.4.2.2 UK5.4.2.3 France5.4.2.4 Italy5.4.2.5 Spain5.4.2.6 Rest of Europe5.4.3 Asia-Pacific5.4.3.1 China5.4.3.2 Japan5.4.3.3 India5.4.3.4 Australia5.4.3.5 South Korea5.4.3.6 Rest of Asia-Pacific5.4.4 Middle East & Africa5.4.4.1 GCC5.4.4.2 South Africa5.4.4.3 Rest of Middle East & Africa5.4.5 South America5.4.5.1 Brazil5.4.5.2 Argentina5.4.5.3 Rest of South America

6 COMPETITIVE LANDSCAPE6.1 Company Profiles6.1.1 Cobra Biologics6.1.2 Finvector Oy6.1.3 Fujifilm Diosynth Biotechnologies U.S.A., Inc.6.1.4 Kaneka Corporation (Eurogentec)6.1.5 Merck KGaA6.1.6 Novasep Inc.6.1.7 Oxford BioMedica Plc.6.1.8 Sanofi SA6.1.9 Spark Therapeutics Inc.6.1.10 Thermo Fisher Scientific Inc.

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

Contact Us:

Name: Ajay More

Phone: US +14242530807/ UK +44 20 3239 8187

Email: [emailprotected]

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Brandon Thoene, left, and Morgan, right, sit with their son Levi sit up during a visit to his doctor at Children's Hospital on Friday, October 18, 2019. Levi, who has spinal muscular atrophy, a month ago received a new gene therapy called Zolgensma and has been recently able to sit up on his own for the first time.

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Dr. Geetanjali Rathore examines Levi Thoene just before his second birthday at Children's Hospital on Friday, October 18, 2019. Thoene has spinal muscular atrophy and recently received a new gene therapy called Zolgensma.

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Brandon Thoene, left, fist-bumps his son Levi during a visit to his doctor at Children's Hospital on Friday, October 18, 2019. Levi, who has spinal muscular atrophy, a month ago received a new gene therapy called Zolgensma and has been recently able to sit up on his own for the first time.

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Morgan Thoene, left, comforts her son Levi during a visit to his doctor at Children's Hospital on Friday, October 18, 2019. Levi, who has spinal muscular atrophy, a month ago received a new gene therapy called Zolgensma and has been recently able to sit up on his own for the first time.

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Boy, 2, first in Nebraska to receive $2.1 million therapy for rare disorder. 'He's doing fantastic' - Omaha World-Herald

Recommendation and review posted by Bethany Smith

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I never imagined every woman in my family would get breast cancer. It started in 1998 when my aunt was diagnosed at 58-years-old. In 2010, my mom was diagnosed at 65. Two years later, my cousin (my aunts daughter) was diagnosed at 42 with Stage 1 Triple Negative breast cancer, the most aggressive and fastest-growing type. Had it been discovered just a year later, it would have grown to Stage 4. I joined the club in 2014, just two weeks shy of my 35th birthday. All four of us tested negative for the BRCA gene mutation.

In the simplest terms, thats code for the BReast CAncer gene and is split into two categories: BRCA1 and BRCA2. Though we all have BRCA genes, they are believed to increase a persons chances of developing the disease when mutated. Dr. Sunil Hingorani, a family friend and pancreatic cancer specialist, once told me It doesnt mean there isnt a gene link, it just means they havent found the gene yet. Eek. Then I thought, Oh, maybe theyll name it after us. Cool. Wait. Nope, not cool.

After being told we werent BRCA gene mutation carriers, I morphed into a boob spy named Erin Boobivich to investigate exactly what the culprit was. My cousin believes it has something to do with the water in Connecticut. Not only had each of us lived there for at least 30 years prior to our diagnosis (Boobivich knows her number-crunching); its also home to some of the countrys highest breast cancer rates. My brother thinks the microwave we grew up using is to blame. My mom thinks my aunt got it from eating too much barbecue. Im convinced it has something to do with potato chips. (What?! Theyre a carcinogen! I read an article once and now I dont eat potato chips). Okay, thats the extent of my research, but it could be all or any of those things, plus a bag of genes.

Reasoning aside, whats really worth sharing goes beyond statistics. Ultimately, breast cancer completely changed our lives collectively and individually. And while its important to share the commonalities weaved throughout, our individual experiences carry lessons that should be amplified, too.

My aunt, who left India for America in the 70s, was diagnosed with HER2 neu positive, a very aggressive type of breast cancer. She had a lumpectomy (aka breast-conserving surgery that removes abnormal tissue) and more than 20 lymph nodes removed to determine if it had spread throughout her body. This was followed by debilitating chemotherapy that left her extremely ill for months. From the outside, you could never tell she was in pain because her sense of humor made cancer seem fun. She was always upbeat and cracking super inappropriate jokes, like the one where she called me in 2003 and joked her cancer was back, then cackled loudly and said she was kidding. Ha. Ha.

During chemo, she chose not to wear a wig, and instead wore headwraps and proudly flew bald. She just didnt seem to care. Her hair never really grew back. If you ask my aunt to take a picture with you today, she will refuse, claiming she hates pictures. The truth is cancer changes how you feel about your body. Her hair didnt grow back to its former glory, but thankfully, neither did her cancer. She just hit her 21 year clear MRI on October 8, 2019.

My moms cancer was hormone-based. She was told by a doctor that she would need a lumpectomy, radiation and chemotherapy. Our family friend Dr. Hingorani insisted she go to Dana Farber, a renowned cancer institute in Boston, for a second opinion. My mom balked at it, but my father and Hingorani insisted. Its a good thing she listened. The doctors there confirmed chemotherapy wouldnt be beneficial. At all. Had she gone through with it, she would have lost her hair and who knows what else. for No. Benefit. At. All. With that being said, get second opinions. Get thirds. Make sure you have all the info you need.

By the way, after her lumpectomy and radiation, Mom was put on Arimidex, a drug specifically for post-menopausal women to reduce the risk of cancer coming back. She just hit 9 years clear and was told on October 9, 2019 that she no longer needs to take it.

My cousin was diagnosed in November 2012 in early November and went through at least 5 biopsies well into 2013. Because her cancer was especially aggressive, a combo of lumpectomy, radiation, and chemotherapy was the only choice. At the time her children were 11, 9 and 7 years old, respectively.

At our Christmas dinner, a month before she began chemotherapy, I remember asking if I could get a picture of her, my aunt and the kids with my new camera. Priya responded, Sure, since itll be the last time I have hair like this. And she was right. Her hair has never returned to what it was that day.

She endured 8 rounds of chemo within 4 months and each infusion took 4 to 6 hours. We took turns accompanying her to Boston. Her husband, Douglas, did the first round. But when he tried to unplug the chemo machine to charge his Blackberry, lets just say he wasnt invited to return.

Priyas hair fell out, her nails turned blue, and her eyebrows disappeared. My cousin is a very strong and stoic individual to a fault. She never wanted to admit she was in pain or needed help. I understood. With three young children, she didnt want them to feel unsafe or think that their mother was dying. One morning she couldnt protect them from what was going on, and while they were having breakfast, she fainted in the pantry. Thankfully, Douglas wasnt charging his Blackberry, ran to the pantry, and moved the kids out of the kitchen so he could help her. He was scared. So were the kids.

She was told that chemotherapy would only improve her chance of no recurrence by 3-5%. It seems like nothing right? But with 3 kids, she said she would do whatever was necessary. Today, shes 7 years clear. But in addition to her hair thinning, chemo also affected her brainspecifically her attention span and memory. It took her two years to be able to get through a long book again. And like a lot of women, chemo threw her into early menopause at just 43.

My mother and I got the exact same type of cancer. Same boob. Same exact spot. Like mother, like daughter. The only difference was age; my diagnosis came much earlier in life (30 years before Mom, to be exact.) It sucked. I always thought Id have children of my own. Unfortunately, this diagnosis changed that.

Prior to getting the bad news, everyone in my family begged me to get screened; specifically, after my cousins diagnosis. I finally did a year later. I thought there was no way I could to get cancer at such a young age. Thats what we all thought.

But then they saw something on my right boob. After two mammograms, 1 ultrasound and a biopsy, I got an all-clear. Phew. Relief. No cancer. But then something strange happened. In my right underarm, I got a swelling that was extremely painful. So on Christmas day, my uncle drove me around to see if we could find an urgent care center. The doctor who had previously ordered my biopsy, asked for an MRI. It revealed everything was fine on the right breastand a tumor on the left. The latter was missed on two mammograms and an ultrasound.

My mom said when I called her on March 4, 2014 to break the news, it was one of the most shocking moments in her life. I remember bursting into tears and Im not a crier. That doctor told me, So listen, you can get a lumpectomy, radiation, maybe chemo. Or you can just get a mastectomy and not have to worry about any of that. Wow. (That was the most glossed-over statement that was ever said to me). My cousin called Dr. Alexandra Heerdt, her breast surgeon at Memorial Sloan Kettering Cancer Center, who later told me a mastectomy wasnt something shed recommend.

By the time my lumpectomy was done on April 9, 2014, I had been through 2 mammograms, 2 ultrasounds, and 3 biopsies. Two days after my surgery, I developed cording, despite only having 3 sentinel nodes removed. Its a traumatic reaction your body has when the muscles and nerves in that same area wrap around each other. It was one of the most painful experiences of my life and took 5 months of physical therapy to treat.

Radiation nukes everything. The doctors told me if I ever had kids, I wouldnt be able to breastfeed on my left side. It also charred my boob and literally turned it black. Thankfully, my cancer had not spread, so chemotherapy was eliminated. However, I had to go on a drug (like Mom) to inhibit the hormones that caused my cancer in the first place.

My doctor initially recommended shutting down my ovaries for 5 years. What the ever loving?! Yes, thats correct. He went over the side effects, which include but arent limited to hair loss, decreased libido, and joint pain. No thanks. So I talked to Dr. Rachel Freedman, my other doctor at Dana Farber (who also happens to be my cousins oncologist). She said the research wasnt there to confirm ovarian shutdown as the best option. Instead, she recommended I start with Tamoxifen and if a new study was done on ovarian shutdown, I could switch. A few weeks later, a new study became available and my main oncologists recommendation aligned with Dr. Freedmans.

He also said pregnancy is a hormone storm for your body. You cant for at least 5 years. That was probably the hardest thing to hear. I chose to not freeze my eggs, because quite frankly, getting rid of cancer and going through treatment was enough to deal with at the time. I worry that I will regret this decision.

This year, I hit 5 years clear. I will have to take Tamoxifen for a total of 10 years, or until Im 46. Ill probably enter menopause shortly before or after that. It sucks. Thankfully, Tamoxifen hasnt thrown me into early menopause; at least not yet. For this reason, I actually get excited for the period I used to curse each month. As long as Im still getting it, maybe theres still a chance I can have kids.

My cousin Priya has two daughters, Bella, age 16 and Emma, age 14. About 2 months ago, we were out to lunch with my Aunt Veena, their grandmother. The topic of breast cancer came up, and Emma, Bella and I made some jokes as our family is apt to do. My aunt looked horrified, and Emma lightly said, What Nani? We know were probably going to get it.

I hope they never join this club. And with research and progress over the next few years, maybe they wont. Today, all of us are diligently scanned and have annual MRIs and mammograms. I have blood tests every 3 months. And when we get clear tests, we text our family group chat to report the news because were all scared on some level. The fear that its going to come back never goes away. Sometimes, the further I get from it, the more I fear I experience.

So cancer does change things. A lot. But with a good boob squad friends, family, and doctors it will be ok. My friends saved me when I was going through all of this. In fact, this post-it my friend Marisa found on her desk from 2014 says it all. (Seriously, if you get cancer, you can get your friends to do stuff for you). And remember, every day they are discovering new genes and new treatments. So get your mamms, maams.

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Every Woman in My Family Had Breast Cancer and None of Us Have the BRCA Gene - STYLECASTER

Recommendation and review posted by Bethany Smith

A majorityof cystic fibrosis patients who took part in an Instagram poll by BioNews Servicessaid they would be interested in moving from current treatments to Trikafta (elexacaftor,tezacaftor, andivacaftor), the newly approved and next-generation combinationtherapy by Vertex that is expected to treat 90% of all with CF.

Among those now using Symdeko (tezacaftor/ivacaftor, ivacaftor) orOrkambi (lumacaftor/ivacaftor),twoearlier Vertex treatments,93% of the 129 people who responded to this question said they would like to switch, largely because the benefits they felt from their current therapies were, at best, mixed.

My doctor told me that the new med blows Symdeko out of the water! said Christina Khoury of Pennsylvania, who was diagnosed at 3 weeks old.

Others, including Wendy Caroline of Wisconsin and Elizabeth Rogers of California, both diagnosed before age 2, simply noted that Orkambi and Symdeko hadnt work as hoped, and were a disappointment.

Drew Hanley of Florida, diagnosed at 13, was a bit more succinct: In medical lingo Symdeko and Orkambi were never considered highly effective. Trikafta is.

The informal, one-day poll,conducted Oct. 2223, asked seven questions largely to patients who follow Cystic Fibrosis News Today (a BioNews publication).

It also found thatmost were not surprised by the U.S. Food and Drug Administrations quick approval a little more thab two months since a request for approval was filed and given an accelerated review. Perhaps that is because of the positive results reported from clinical trials.

A slight majority, 54% of the 212 people who responded to this question, thought the FDAs speedy review was reasonable.

But a stronger majority 83% of 193 people responding were disappointed in Trikaftas expected $311,500 yearly list price in the U.S., equivalent to $23,896 for a 28-day supply, according to financial reports. That puts Trikaftas list price up with Kalydeco (ivacaftor), Vertexs most effective CF therapy to date, but a potentiator that treats only about 6% of all patients, as the most expensive of all of the companys CF medications.

Symdeko carries a yearly list price of about $292,000, and Orkambi of about $272,000. Financial analysts in the U.S. had speculated that Trikafta would enter the market at a list price similar to Symdeko. That it appears unlikely to do so has some patients worried.

FOR SURE, said respondent Bethany Schulberg of the United States, diagnosed at 18 months.The cost of this will make it difficult for people who have certain insurance access.

Insurance coverage and its quality could be key.

Caroline of Wisconsin, diagnosed at 6 weeks old, considered herself fortunate: Im lucky to have good insurance, so it shouldnt be an issue for me personally.

Keisha Hummel, also of the U.S., said shes a [h]ighly concerned adult with CF & married. Insurance is always a big issue for me. Always fighting.

Those from other countries expected a long wait, calling Trikaftas likely arrival at their home a dream. (Respondentswho could not be reached later to confirm use of their statements are not quoted.)

Trikafta, whose U.S. approval was based on two Phase 3 clinical trials in the AURORA program, is expected to be available soon for patients ages 12 and older with one F508del and one minimal function mutation in the CFTRgene, or with two F508del mutations, reflecting the patient groups evaluated in each trial NCT03525444for one F508del mutation, and NCT03525548for two.

An F508del defect in CFTR gene the most common CF mutation leads to faulty instructions being given for the creation of the CFTR protein. This protein regulates the production of mucus needed to lubricate organs, especially the lungs and digestive system, by controlling the transport of charged substances (chloride and sodium, especially) across cell membranes. Its failure results in a buildup of thick mucus that is difficult to clear and traps viruses and bacteria, promoting infections that weaken patients and damage their organs.

Previously approved Vertex therapies Kalydeco, Orkambi, and Symdeko were able to treat some people with one or two F508del mutations in CFTR, but not those with what are called minimal function or nonsense mutations. More than 1,700 mutations are known to exist in the CFTRgene, and nonsense mutations are those that prematurely stop the CFTR protein from forming.

Because this triple combination covers people with both the common F508del mutation and those with another minimal function mutation, it is expected to treat up to 90% of all CF.

Data fromboth AURORA studiesshowed significant gains in lung health in people on Trikafta, the primary goal of each trial, as assessed by the percent predicted forced expiratory volume in one second (ppFEV1, a widely used measure of lung function). ppFEV1 increased by an average of 13.8 percentage points in AURORA F/MF, which enrolled people with one F508del and one nonsense mutation,and by 10 percentage points in AURORA F/F, in which patients had two F508del mutations.

Trikafta-treated patients in AURORA F/MF also showed improvements in all secondary study goals, including a lower annual rate of pulmonary flares and diminishedsweat chloride levels.

Todays landmark approval is a testament to making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options, and giving others in the cystic fibrosis community access to an additional effective therapy, Ned Sharpless, MD, the FDAs acting commissioner, said in an agency releaseannouncing Trikaftas Oct. 21 approval.

The importance of this new option is evident: In this one-day poll on Instagram, where responses were limited by the nature of this social media platform, 47% of the 165 respondents a near majority said Trikafta would be a first CF treatment available to them.

Curiously, a follow-up question asking how they felt about having a first disease-modifying treatment was the only question to elicit no response.

Grace Frank worked as a copy editor, city editor, reporter and news designer for leading American newspapers, including The New York Times and The International Herald Tribune, for many years. She has won numerous journalism awards, and was nominated for a Pulitzer Prize for an investigative series into eye surgeries wrongly conducted outside a clinical trial.

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CF Patients Voice Hopes and Expectations for Trikafta in Instagram Poll - Cystic Fibrosis News Today

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Miriam didnt start out seeing herself as someone who could beat MCI. It didnt even feel like a possibility.

But early one day in September, she logged into a support group on Facebook to share the good news.

Heres exactly how she put it:

Miriams outcome, which took more than four years of hard work to achieve, isnt the most common for someone with mild cognitive impairment.

But it isnt the rarest either.

A recent study shows that over roughly that period of time, about half of the people diagnosed with MCI are likely to see their memory loss stabilize, and about a third will return to cognitively normal as Miriam did or at least fluctuate between MCI and cognitively normal.

So Miriam is not an outlier. Rather, shes an inspiration. She shows us that MCI can indeed be reversed. More importantly, she shows us how.

Thats why I tracked Miriam down after I saw her post on Facebook, and why Im glad she was willing to talk to me. I wanted to understand what worked for her how she did it.

Not because shes found some secret formula that anyone else can follow and get exactly the same result she did. But because Miriam took so many of the fundamental things people with MCI are encouraged to do, and threw herself into them.

For her, they worked. And shes convinced they can work for others, too.

When I started on this journey of recovery, my goal was to keep myself from getting worse; thats all I wanted, Miriam says.

But what happened was, I got better and it reversed, and I wasnt expecting that. I want people to know if that happened to me, it could happen to you.

For Miriam, the odyssey back to cognitively normal involved long, vigorous walks with her husband. It required her to radically improve her eating habits, giving up meat and eventually moving to an entirely plant-based diet. She had to learn ways to manage stress, and practice them faithfully.

So make no mistake about Miriams journey. It was a long, arduous one. And it began with memory problems that sent her stress level shooting off the charts, and a frightening diagnosis involving a condition she knew nothing about.

Miriam was 50 years old, married with three adult children and working as an accountant in the southwestern U.S. when her memory began to slip.

I was working a very stressful job, she says. The thing I remember most is that I would have to run meetings and when someone else was talking, I couldnt write down what people were saying because I couldnt remember what they said.

She wasnt the only one to notice. At one point, my manager said, Miriam, they just told you that, she recalls. But I couldnt write it down. It was gone. I thought, You didnt used to do this. Something is wrong. I started to get really stressed out.

Miriam immediately assumed the worst. Alzheimers runs strongly in her family, on both sides. But it really threw me, because I thought it was happening too soon, she says. It didnt hit my family until they were in their 70s, and I was 50.

She talked to her doctor, who sent her for an MRI and referred her to a neuropsychologist for cognitive testing. The following month, the results came in and she received her diagnosis mild cognitive impairment due to vascular disease and depression.

It wasnt the news she was hoping for. Even though I thought something was wrong, I had hoped it wasnt that bad, Miriam says. I hoped it was just me being too worried. I guess I was a little shocked, even though I wasnt completely shocked.

Based on her family history, Miriam knew all about Alzheimers. But MCI? Shed never heard of it.

I had no clue what MCI is, she says. I started reading everything I could find about it on the Internet. I think probably at that time I saw something about the percentage of people with MCI who went on to Alzheimers, and I knew I was at high risk. I thought, So this is how it starts.

If the concept of MCI was vague and confusing, the reality of living with it hit her in ways she has no trouble describing.

I was tired. I was fatigued, Miriam says. I tried to exercise but it felt like there was a weight on me. I couldnt focus properly. I couldnt think properly. I was scared. I had to write everything down, because I couldnt remember. I could not come home and cook dinner. I would come home and sit and watch TV. At that particular point, it was strictly that I wasnt able to thrive.

The company Miriam worked for at the time was a high-tech startup, and it was struggling. Her manager got laid off. Soon after that, she was laid off too.

During the time when I was let go, I was feeling so much anxiety, Miriam says. I decided I didnt want to start another job yet. I wanted to get some of this anxiety down. I started searching to see if I could find a doctor, someone who I could trust, who knew something about dementia. I knew there really was no cure, but I thought if I could find someone who was really into it, they would be on the front line and be aware of something if it did come up.

She was fortunate to find a memory specialist who was well-versed in MCI and dementia, and that helped put her on a better course. Miriam began to understand that even if theres no medicine approved for the treatment of MCI, theres ample evidence that lifestyle changes have the potential to slow or halt it.

She took up an exercise program, and coupled that with dramatic changes to her diet.

I started walking with my husband, Miriam says. We would walk three times during the week, three or four miles. On weekend days, wed walk four to eight miles each day. It was hard. I felt like I had weights on my legs.

But she persevered. Those regular walks turned into a healthy habit she continues today, and that fatigue has been replaced by a feeling of fitness. I still do about that much walking, but I walk a lot faster now, she says. Im not dragging. I have pep. I have energy. I feel a lot younger.

For Miriam, an even bigger turning point came when a physician assistant who worked for her memory specialist recommended a primarily plant-based diet, with no meat, but fish two or three times a week. She gave Miriam the book The Perfect Gene Diet, by Pamela McDonald, and told her to follow the diet recommended for people with the APOE4 gene. That gene is associated with a higher risk of Alzheimers.

Four months after being on The Perfect Gene Diet and I followed it strictly I was no longer on an anti-depressant, Miriam says. The diet cleared up my depression, so I continued doing the diet. I followed the diet, except no meat whatsoever. I only did fish.

While exercise and diet were beginning to benefit Miriam, that wasnt the whole picture. She was working a new job now, and she continued to struggle with work-related stress and the emotional toll of everything she was dealing with in her life. She realized that she had to address those things if she was going to get better.

Around mid-2016, I started going to a therapist, she says. I did individual therapy for two and a half years, and I also did about a year of group therapy. That helped me in this whole process of growing and changing things in my life, and moving forward and getting well.

She also learned a couple of stress-reduction techniques, and began to use them regularly.

One was a visualization technique that taught her how to leave her stress at work. I was bringing things home from work mentally, she says. So I would visualize putting whatever that concern was in a jar, putting the lid on the jar and setting that jar on the desk at my office before I left work.

She began using a very simple breathing technique as well. Breathe in, breathe out slowly, and letting go, just relaxing, she says.

All of these things were making a difference, though Miriam still had a ways to go. I felt a little bit better, my mind was a little clearer, she say, but I was still having some trouble.

The final turning point came when Miriam found a new neurologist. The memory specialist she worked with previously decided to stop taking patients and focus full-time on research. So Mariam had gone a while without seeing a specialist, and her general practitioner wanted her to go back to a neurologist again. I hadnt been able to find anyone else, Miriam says, so she recommended this nice young neurologist, right out of school.

Miriam found her new neurologist to be energetic and full of fresh approaches. About the same time, she discovered the teachings of Dr. Dale Bredesen, author of The End of Alzheimers. She read the book, and a lot of what Bredesen teaches made sense to her, so she asked her neurologist about his protocol. She had heard about it, and she said it was very expensive, but she said she believed in it, Miriam says.

Based on the expense and other considerations, Miriam choose not to seek out a medical specialist trained in the Bredesen protocol. Still, she felt there were parts of it she could incorporate into her life without that help. I decided I would just have to see if I could figure it out on my own, she says.

By then, Miriam had fully committed herself to a healthier lifestyle, and she had a lot of support and encouragement along the way. While she was dealing with MCI, her husband was diagnosed with a blood disorder, so they motivated each other to stick with the improvements in exercise and diet that both of them were making.

She had other family support as well. My half-brother, who is nine months older than me, was diagnosed with early onset Alzheimers, she says. He became another inspiration for me. Hes an electrical engineer, and by changing his diet, and exercise and all the extra things he does, he was still able to work, so he encouraged me. He was always reaching out to me to make sure I was following the protocol.

Yet even at this point, there still was one piece of the puzzle missing for Miriam. And the new neurologist helped her find it.

After I started seeing her, she began running all these different test, Miriam says. She told me, I have some blood work I want to do on you. Its called a dementia profile. She did my blood work in April. Because I was eating fish all the time, she wanted to be sure my mercury levels and my lead levels were OK.

The test results came back, and showed there indeed was a problem. Miriam had high levels of mercury, not to mention arsenic. Her neurologist moved immediately to address the problem. She told me, Stop eating that fish, Miriam says.

Her neurologist predicted it would take six months to a year for Miriam to flush the mercury from her body. But a month later, she says, my brain was clear.

And Miriam says once the mercury was completely out of her system, it was like a curtain being lifted. I noticed I was getting better and better, she says.

After that, all the indications began to turn positive for her.

Shed been working with a speech therapist on issues related to cognition, memory and speech, but her therapist told her that she didnt need further help with those. She said, You are not having any problems, Miriam remembers.

Miriam also had changed jobs, and found the new position to be less stressful. Now I was in a job thats perfect for me, and it was going well, Miriam says. I was having hour-long meetings, and I could talk about everything. I couldnt do that before. I am able to do that now.

The most encouraging indication, though, came this summer, when Miriams former memory specialist reached out to her. The specialist had found a study that she thought Miriam would be a good candidate for, and wanted to screen Miriam for it.

Miriam had always been open to participating in a clinical trial, but most studies are looking for people age 65 or older, so she hadnt been eligible. This one was open to people her age, who either had mild cognitive impairment or in some cases hadnt even received that diagnosis yet.

So Miriam volunteered to be screened for the study. But it turns out she didnt qualify for the best possible reason. They ran me through some of the testing, she says, and then they told me, Were sorry, but your memory is too good for our research.

I could tell even when I was being asked the questions, I was getting more answers right, she says. I was like, Wow, Im remembering more. I knew I was doing better, so I wasnt surprised.

I asked Miriam what it felt like to experience that, and she was giddy in her reply. She described one day at work, when she was so happy and doing so well, she found herself skipping along the corridor like a schoolgirl.

I am thrilled. Im just thrilled, she says. I knew how well I was doing. I was skipping down the hall because I felt so well.

I also invited Miriam to reflect back on all the hard work she had put in over those four-plus years, and all the ups and downs, and the fears and the frustrations of facing MCI. I asked her what she thought had made the difference for her.

The diet was a big piece of it a big, big, big piece of it, she says.

Asked to describe how she eats now, she says: Im on a whole-food, plant-based vegan diet. I strongly limit processed foods. I dont eat meat, fish, dairy, eggs, sugar or oil. I eat all fresh fruit, all vegetables and whole grains such as rice, oats, quinoa, bulgar, and beans, nuts and seeds. I limit bread and pasta.

And of course, there was a bigger picture than that, in terms of how she fought to defend her cognition.

It wasnt just the diet, she says. It also was the exercise and the help that I got with stress and overcoming the anxiety and just dealing with my whole thought process.

To put Miriams experience in perspective, its important to understand that no one can promise you the results she got. But remember, reversing MCI wasnt even her goal in the first place.

She was just hoping to slow it down or keep it from getting worse. And for people with MCI, its not an unrealistic goal. That is actually the more frequent outcome.

Earlier this year, researchers at the University of Pittsburgh published a study showing that across a broad general population of adults with mild cognitive impairment, relatively few of them went on to develop dementia over a period of five years.

Most people with MCI do not progress to dementia in the near term, but rather remain stable with MCI or revert to normal cognition, the study says.

In fact, according to the study, of nearly 900 adults with mild cognitive impairment:

There was a time when MCI was routinely referred to as a precursor to dementia, or an intermediate stage leading to dementia. We know better now.

For people with mild cognitive impairment, the odds of slowing, halting or reversing it are actually pretty good. And you can improve those odds even more by adopting healthy habits that promote brain health.

One of the quotes I like to share as often as I can comes from Dr. Joel Salinas, a neurologist at Massachusetts General Hospital. He told the Harvard Health Letter that only about 15 percent of people over the age of 65 who have MCI will progress to dementia.

Salinas says hes seen plenty of patients stay in the MCI stage for many years, even when we presume it was a neurodegenerative disease, and by that, he means even in cases where the doctors assumed the person had Alzheimers.

Salinas goes on to add: The people who spend the most time cognitively stable are often the ones who stick to lifestyle recommendations.

In other words, people like Miriam.

Again, its important to understand her experience is not a prescription for how to beat MCI. Its more of an allegory.

Its an example of what becomes possible when someone struggling with cognitive loss:

I had no earthly anticipation that I would be back to where I was before I began having this difficulty and getting this diagnosis, Miriam says.

I just want people to know that if you have mild cognitive impairment, theres something you can do and you dont have to spend a lot of money to do it, she says. I hope someone hears my story, and it inspires them to make the changes that are necessary. I hope it helps someone else. I really do.

Continued here:
She reversed her MCI, and here's how she did it - GoCogno.com

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My daughter teaches at a school where a major theme for the students this year is doing acts of kindness.

This prompted me to wonder about the health ramifications of being kind. Its not unusual for some of my older patients to feel withdrawn, and I speculate that if they could engage in sustainable acts of kindness (such as volunteering), maybe their mental health and physical health would improve.

When we hold an elevator door open for someone, hand a dollar to a homeless person or let someone go ahead of us in a line, we notice that we tend to feel good. Aside from the benefit to the recipient when were kind, might there be health benefits to the giver, as well?

In order to answer this question, first we have to look at what happens in the brain when a good deed is performed.

An act of kindness results in the release of several brain chemicals: oxytocin, dopamine, serotonin and endorphins.

Oxytocin is known as the love hormone. It plays a role in social bonding and helps mothers when giving birth and breastfeeding. It also causes the release of nitric oxide in the body, which dilates blood vessels. This results in lowering blood pressure, which, in turn, leads to lower risk of heart attack and stroke.

Dopamine is the neuro-transmitter that is most credited with causing the helpers high. It has a major role in the brains pleasure and reward system.

Serotonin is the neuro-transmitter that regulates mood and happiness. Most prescription anti-depressants work by increasing serotonin levels in the brain.

Endorphins, another kind of neuro-transmitter, interact with opiate receptors to reduce the perception of pain and can lead to feelings of euphoria. Prolonged exercise and consuming chocolate are familiar ways of increasing endorphins.

Performing acts of kindness regularly provides other health benefits. It reduces cortisol, the stress hormone, thereby lowering anxiety.

In a study of 3,000 patients aged 57 to 85, volunteering was the activity most strongly linked to lowering inflammation in the body as measured by C-reactive protein (CRP), a substance made in the liver. High CRP can increase the risk of stroke, heart attack and mortality. In particular, active volunteers aged 70 to 85 had lower CRP levels than those aged 58 to 69 who were not active as volunteers. If thats not enough, there have been several studies that show volunteering leads to longer lifespan.

A study from Pennsylvania State University published in November 2015 in the American Journal of Public Health studied 753 kindergartners to determine the link between social competence and future wellness. Each child was scored on a scale that included items like cooperates with peers without prompting and is helpful to others.

Follow-up was done when these students were approximately 25 years old. The children who scored the highest in social competency in kindergarten predictably had significantly higher college graduation rates and stable employment, and fewer crime, binge-drinking and mental-health problems as adults. By fostering kind behavior in children, we are giving them a greater chance for success later in life.

So how much kindness do you need to do to acquire long-lasting emotional and health benefits?

A lot.

A single act of kindness results in maybe a two-minute high, so to obtain real benefits, performing acts of kindness needs to be repetitive or sustained like by volunteering. Performing a single act of kindness would be like saying you did your exercise quota for the entire day by walking half a block.

An example: After a colleague of mine lost his wife, I was surprised to see him back at work the next day. Being a physician afforded him many opportunities to demonstrate kindness, a natural way for him to cope with his despair. I imagine social-service professionals and those who work as caregivers, educators and clergy have similar chances.

There are many acts of kindness that can become habits. These may include things like tutoring, making charitable donations, expressing gratitude to others, or reading to children or grandchildren.

Caring about one another can lead us to act compassionately. And when acts of kindness become a ritual, the positive effect on our own health can become a surprising side benefit. What will be your next act of kindness?

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Acts of kindness are good for your health - The Jewish News of Northern California

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LOS ANGELES (Reuters) - One toke for the road could end up being a total bummer for drivers who smoke pot, with several companies in the United States preparing to market cannabis breathalyzers as legalized marijuana spreads across the country.

Law enforcement agencies will require breathalyzers to detect marijuana as they are faced with the necessity of stopping more and more motor vehicles being operated under the influence of THC, said Brett Meade, a retired police chief and a senior program manager for Washington-based non-profit group the Police Foundation.

Nearly a dozen U.S. states allow recreational marijuana consumption and 33 states permit pot for medical use. But all states prohibit driving under the influence of marijuana.

Oakland, California-based Hound Labs is one of the companies developing a breathalyzer to detect THC - the component in marijuana that gets people high - and plans to market it in 2020.

Construction companies could be a big part of its market, said Hound Labs Chief Executive Officer Mike Lynn.

Nobody wants a crane operator 50 stories up to be smoking a joint, he told Reuters.

Lynn, a physician, said pregnancy tests, which can detect minute quantities of hormone, inspired him to tackle the challenge of measuring THC on users breath.

Separately, Cannabix Technologies Inc based in the Vancouver suburb of Burnaby is testing a pair of devices at different price points.

Its THC Breath Analyzer could be cheap enough at a few hundred dollars per unit to potentially allow parents interested in testing their teenager before turning over the keys to the family car, said Cannabix CEO Rav Mlait.

The U.S. court system would need to consider how to treat evidence from THC breathalyzers.

Assuming a motorist who tested positive with a THC breathalyzer was impaired behind the wheel could be problematic, said Stanford University law professor Robert MacCoun.

Unlike with alcohol, scientific research has not yet established firm correlations between the amount of marijuana people consume and how impaired they become, MacCoun said in an email.

Paul Armentano, deputy director of the National Organization for the Reform of Marijuana Laws, expressed similar concerns.

But he welcomed breathalyzers as an improvement over existing tests used by police and employers, such as urine analysis that is unable to determine whether marijuana was used recently with the potential for impairment, or days or weeks in the past. Breathalyzers are likely to only detect a user who consumed cannabis within the last few hours.

A test like that would frankly make sense, Armentano said. Just like we wouldnt allow employees to have a couple drinks and show up to work.

Additional reporting by Jane Ross in Newark, California; editing by Bill Tarrant and Bill Berkrot

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Jerusalem Linda Gradstein has a strong family history of breast cancer. Her mother survived breast cancer in her 60s but died a decade later from leukemia. Her maternal grandmother died of the disease in her 50s and her fathers father died of it in his 60s.

So when Gradsteins physician suggested she get tested for the BRCA1 & 2 mutations that greatly raise the risk of developing breast and ovarian cancers especially in Ashkenazi women, the 57-year-old Jerusalem resident readily agreed.

When the test came back negative I felt quite relieved, as if I didnt have to deal with this now, Gradstein, a journalist, told The Jewish Week.

Even so, Gradstein is aware that she may harbor another type of breast cancer-inducing gene mutation.

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Its scary because I feel I have no control over it. I try not to think about it too much, she acknowledged.

Hoping to lower her risk, Gradstein eats healthily and exercises regularly, especially because being overweight is a risk factor for breast cancer. She also continues to get mammograms, may retest for the BRCA1 & 2 mutations (the test has improved over time), and is considering undergoing testing for other gene mutations.

Gradstein is one of the many women who have tested negative for the BRCA1 & 2 mutations but are still considered to be at higher than average risk of developing breast cancer.

About her family history of breast cancer, Linda Gradstein says, Its scary because I have no control over it. Courtesy of Linda Gradstein

Just 5 to 10 percent of breast cancers are thought to be hereditary, caused by mutated genes inherited from a parent. But these low odds are of little comfort to families where multiple relatives have been diagnosed, especially prior to the onset of menopause.

BRCA1 and 2 mutations account for an estimated 20 to 25 percent of hereditary breast cancers and about 5 to 10 percent of all breast cancers.

Li-Fraumeni and Cowden syndromes raise the risk of developing several types of cancer, including breast cancer. Additionally, more than 150 other genetic mutations have been associated with a slightly higher breast cancer risk, according to a report by the American Cancer Society.

A mutated gene can be inherited from a persons father or mother.

Physicians advise high-risk BRCA-negative women to undergo frequent breast exams that combine a physical exam by a breast specialist with mammograms and/or ultrasounds. Some also advise an MRI, in between mammograms.

Otherwise, you have almost a year with no imaging, said Dr. Tal Hadar, a breast surgeon at Shaare Zedek Medical Center, who advises many of her patients at risk for hereditary breast cancer to alternate mammograms with MRIs every six months, along with a clinical exam.

A trained breast specialist may be able to detect breast lumps and spot irregular discharge from the nipples that imaging technology can miss.

Professor Tamar Peretz, who heads Hadassahs Sharett Institute of Oncology, Center for Malignant Breast Diseases, emphasized that not every woman who has a mutated gene will develop breast cancer.

We cannot say or define exactly why one woman with a mutated gene develops cancer and her sister with the same genomic profile does not. This is part of our research, Peretz said.

Hadassahs Professor Tamar Peretz says a healthy lifestyle can reduce the likelihood of developing breast cancer. Hadassahinternational.org

But there is data suggesting that a healthy lifestyle can reduce the likelihood of developing breast cancer.

Factors such as hormone exposure, environmental exposures, diet, exercise and other genes can affect cancer risk, both in people with inherited genetic mutations that increase cancer risk and people who do not have a mutation.

Based on a womans estimated risk and her personal preferences, her physician may recommend comprehensive panel testing (testing for a number of gene mutations) to determine whether she has a non-BRCA mutation that could raise her breast cancer risk.

Although limited testing for just a handful of so-called Ashkenazi mutations, including BRCA1 & 2, was once the norm, some cancer specialists now advise their patients to opt for full sequence testing, with searches for those mutations plus many others.

We know if a woman is Ashkenazi and tested [negative] only for the mutations most common in Ashkenazi Jews, there is still a 4-to-5 percent probability she is carrying another mutation in the BRCA gene, Peretz said. And among the BRCA-negatives, there are several gene mutations in other genes that have already been identified.

Before opting for genetic testing a woman should sit with a genetic counselor not only to gauge her risk based on everything from family history to whether she breastfed her children, but to discuss the implications of discovering a non-BRCA mutation.

While the risks of having certain mutations (such as PALB2, CHEK2, ATM, BARD1, PTEN, TP53, NF1, CDH1, NBN, and STK11) are well-established, there are many other mutations whose level of risk is not currently known.

There are instances when the tests come back with variants of uncertain significance, which means that there is a mutation but its potential impact on health is unknown at this time, said Dr. Ephrat Levy-Lahad, the director of the Medical Genetics Institute at Shaare Zedek Medical Center in Jerusalem.

While scientists may one day learn more about the mutations implications, right now geneticists cannot say how much risk the mutation carries. Not everyone is comfortable living with that uncertainty.

When a mutations implications are known, knowing you are carrying that variation may lead to more positive outcomes, according to Hadar.

Speaking at a medical gathering, Hadar noted that patients at Shaare Zedeks Noga clinic for women who are BRCA1 & 2 positive are usually diagnosed earlier [than patients who are not BRCA-positive] because they are aware of the danger and have a better prognosis. They have more frequent screenings and may decide to have a preventative double mastectomy to prevent cancer from developing.

Being BRCA1 & 2 negative has only boosted Gradsteins desire to learn as much as possible about her cancer risk.

I feel like if we have the opportunity to have more information, why wouldnt we want to have it, in the hopes of early detection? she said.

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BRCA-negative Women Explore Their Options - Jewish Week

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For years, speed skater Katherine Adamek struggled with chronic professional self-doubt, despite having won two Olympic medals. The anxiety and insecurity were bleeding over from the ice into her personal life. A hip injury forced her into retirement in 2013.

After three years, though, Adamek was craving a comeback, but I wanted to do things differently this time, she says, building my confidence and learning how to be present for my teammates, how to enjoy my successes and learn from my mistakes instead ruminating and judging myself for them. She reached out to an Olympic sports psychologist with whom shed previously trained, who agreed to help, with one caveat: Adamek needed to start using a meditation app.

Between grueling workouts, Adamek interspersed three-minute guided meditations, building to 10- and 20-minute sessions. Within a few weeks, negative emotions passed more easily. I noticed changes in my relationships with my family, friends and teammates. I could enter a strength training session feeling confident, even if my last workout wasnt great. Adamek set a new American record at her first World Cup Circuit back from retirement in 2016.

Related: The Best Free Meditation Appsand One You Should Pay For

In the early 90s, when Phil Jackson (Nickname: The Zen Master) introduced mindfulness to the Chicago Bulls, the concept of athletes using meditation to tune into their bodies and minds and live in the moment was novel. But as meditation has moved mainstream among the American publicmore than tripling from 2012 to 2017, per the U.S. Centers for Disease Control and Preventionmore and more athletes have embraced the technique.

Kobe Bryant, who learned to meditate when Jackson began coaching the Los Angeles Lakers, has said 15 minutes of daily morning meditation act like an anchor for his day, leaving him calm, set and ready for whatever may come my way. Pete Carroll, head coach of the Seattle Seahawks and an ardent believer in mindfulness, hired a sport psychologist to teach meditation, deep breathing and visualization to his athletes as a means of calming their minds during stressful plays and helping them enter the zonethe mental state of peak performance where an athlete can seemingly do no wrong. And Nike has partnered with the Headspace meditation app, offering subscribers features like mindful guided runs as part of a holistic athlete experience.

Orthopedic surgeon Mark Schickendantz, MD, director of Cleveland Clinic Sports Health and head team physician of the Cleveland Indians Major League Baseball team (yes, they employ a mindfulness coach), says meditation doesnt make players run faster or hit the ball with more force. It allows athletes to grow more grounded and centered, he explains and that mental stillness translates into them being able to tune out noise from the crowd, or not let a strike out wreck their performance for the rest of the game.

Schickendantz says it can do the same for everyday folks watching from the bleachers. Think about the stressors that come into our lives and catch us off guard; we tend to react mindlessly, yelling at the person who cut us off in traffic, for example. With practice, meditation and other mindfulness techniques help you respond to stress in a calmer, more present manner.

Practice gratitude. It can reduce depression and anxiety, lower your risk of disease and flood the brain with feel-good chemicals like serotonin. Work gratitude into your daily routine by reaching out to say thanks to someone who means a lot to you.

You dont need a candlelit yoga studio or an NBA locker room; just find a quiet, comfortable place where you can sit upright and be comfortable. Choose something to focus on; your breath, a mantra, an object. Feel your feet on the floor, connecting to the earth, and take three deep cleansing breaths, inhaling through your nose and out through your mouth, Schickendantz says.

Outside thoughts, like your looming To Do list, are bound to arise. Not only is that perfectly OK its the point of meditating. Mind-wandering isnt bad, he assures. Dont judge it. Just recognize it, label itWhoops, my mind is wandering and bring yourself back to the meditation. This continual re-centering is what builds mindfulness muscles, helping you better filter out stress and distractions and respond to life in a calmer, less-judgmental manner.

Creating a formal daily practice can maximize your benefits, but Journal of Clinical Psychology research found that meditating for five minutes a day, five days a week was enough to slash stress, elevate happiness and enhance the sense of connection subjects felt with others. Try grabbing it throughout the day, encourages Schickendantz, who practices while stuck in traffic. Do it while standing in line at the coffee shopinstead of automatically grabbling your phone [and mindlessly scrolling,] just stand there and be with yourself. (These are sometimes called micro-meditations.)

Related:The Four Sacred SecretsAuthors Share Their Favorite 6-Step Meditation for Beginners

Thanks to modern-day addictions like smartphones and social media, along with the tendency to over-analyze our words and actions, many of us neglect to tune into our thoughts and emotions, causing us to miss out on everyday moments, big or small, and disconnecting us from our bodies, says ChristianSlomka, Community Manager for the Calm meditation app. For athletes, losing focus can mean the difference between a huge win or loss; for you, the stakes range from sad (your little one feels ignored when you appear to choose your tablet over her) to sick (constant rumination triggers the bodys stress response, which is linked with an exhaustive laundry list of illnesses) to devastating (a texting-while-driving car accident).

Schickendantz says relief comes by treating pain just like an unwelcome thought: You identify it, label itTheres that pain againand let it go, accepting it as something that just happens to be a part of your life right now. Its not easy, he acknowledges but it can work. In a recent study by the Hospital for Special Surgery in New York, meditation and mindful breathing helped patients manage chronic pain, sometimes lessening the need for opioid medication. Other research showed a single 10-minute meditation session could feasibly replace painkillers, boosting pain tolerance and reducing pain-related anxiety.

Whether youre an injured Olympian coping with an injury or a parent attempting to keep cool during a toddler temper tantrum, meditation can help you respond to life in a calm, mindful, less judgmental way. The practice also elicits multiple calm-inducing health effects, such as decreased heart rate and blood pressure, and reduced stress hormone production.

Celebrity interviews, recipes and health tips delivered to yourinbox.

The National Sleep Foundation says people who meditate fall asleep faster, sleep longer and catch higher quality zzzs.

Pre-meditation, Id bring work home with me and it would affect my interactions with my husband, Adamek described. After six weeks of using the Vision Pursue meditation app (cofounded by 10-year NFL veteran Jon McGraw), I brought less stress home with me and could be present, asking him about his dayand really listening. Back on the ice post-retirement, her new mindful skills helped her tune into teammates having bad days, encouraging them to open up. Today, as owner of a coaching company called Fix Your Mindset, Adamek helps athletes and organizations reach their next level via mental toughness skills including, of course, meditation.

Try a mealtime meditation with this easy exercise.

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5 Benefits of Meditation: Why Athletes Meditate and You Should, Too - Parade

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During the week, online newspaper, TheCable published an expose on the rot and other shady deals that take place at police stations.

Although they zeroed in on a particular police station in Lagos, however, what they exposed is what is happing at the various other police stations dotted across the length and breadth of this country.

In the story, captioned: UNDERCOVER INVESTIGATION (I): Bribery, bail for sale Lagos police station where innocent civilians are held and criminals are recycled, TheCable wrote in their intro to the piece: Investigative journalist FISAYO SOYOMBO spent two weeks in detention five days in a Police cell and eight as an inmate in Ikoyi Prison to track corruption in Nigerias criminal justice system, beginning from the moment of arrest by the Police to the point of release from prison.

To experience the workings of the system in its raw state, Soyombo adopting the pseudonym Ojo Olajumoke feigned an offence for which he was arrested and detained in police custody; arraigned in court and eventually remanded in Prisonhe uncovers how the Police pervert the course of justice in their quest for ill-gotten money.

TheCable then goes on to give a vivid account of what transpires at the Pedro Police Station and even Ikoyi Prison through the first-hand experience of their undercover reporter.

In one instance, Ojo Olajumoke wrote: The complainant was already registering the case with a policewoman by the time we returned, and soon after they were haggling over the fees. Chigozie Odo, the policewoman, had rejected his offer of N500. After some five minutes of talking, he handed her a N1, 000 note.

Odo stripped me of my shirt, singlet, belt, wristwatch, shoes and cash. Look at his hair; na you gangan be Ruggedy Baba, she said as she unlocked the cell and bundled me in.

This report was only published during the week, but sadly, I can point out that this has been the pattern for decades; because I have also personally experienced it.

In my own case, it was not an attempt to expose the rot through fine investigative reporting, but rather I was a forced participant because a then military governor wanted to deal with my then medium, The Guardian.

I have actually written the story before in a piece I titled How Champions League landed me in Kirikiri Prison, which I wrote to commemorate the 25th year that I and three other colleagues, Bayo Oguntimehin, Taiwo Akerele and Ben Akparanta (now late) were hounded into detention for about 16 days, first at Alausa Police Station and then Motor Traffic Division (MTD), both in Ikeja, Lagos, before ending up in Kirikiri Medium Prisons, just because the then governor Col, Raji Rasaki (rtd) had some bones to pick with the flagship over The Guardians critical position on some of his actions as governor of Lagos.

Our ordeal began on May 29, 1991 when the then governor made his move leading to the closure of The Guardian by the state government.

At both Alausa and MTD, we witnessed first-hand how the police who claim to be our friends interact with those who come to the station to either lodge complaints or are brought in for allegedly committing and offence.

They (police officers) were mostly unfriendly towards such people and more often than not were ready to either bend the law or dish out favours following the receipt of some form of gratification.

For instance, on the first night of our detention after our management had spoken to the officers on duty, we were not immediately shoved into the detention cells with other suspects.

Instead, we were kept outside until about midnight before we were told we had to be put into the cell because it was against the law to allow suspects spend a whole night outside the holding bay.

But of course, before sending us into the cell, they (police) had spoken to the president to ensure that we (journalists) were not given the traditional welcoming which is often a severe beating.

And just like Ojo Olajumoke wrote, I still recall having to remove my belt, watch and other valuables on me and when I jokingly asked what would happen should my trousers not stay up without a belt, I was told if that be the case, then I would have to hold my sokoto because I could not enter the cell with a belt so that I dont use it to commit suicide or as a weapon!

Those in the holding cells were a sorry sight to behold with many of them complaining that they had been thrown in on trumped up charges and would only regain their freedoms as soon as they settled.

Many of them were brought in for wandering, which was then an offence that the police exploited very well in apprehending people.

Although it is often said lightening does not strike twice, however, I have been an exception; and six years after my Kirikiri trip, I was again a forced guest of the police, after I and my two other co-tenants were arrested for armed robbery and gun running.

Incidentally, I had just arrived in the country from Egypt, where I had gone to cover the FIFA U17 World Cup when this fresh incident occurred.

This time we were lodged at the Adeniji Adele Police Station, while we battled to extradite ourselves from the case, which if not properly handled could see me face the death penalty.

Sadly, during the course of our investigation, we were told by our fellow detainees, that we had actually been setup by one of our fellow tenants, who was the only one not with us in detention.

We were told that he (the tenant) had come to the police that he could make money for them by writing a petition that he knew of some armed robbery suspects who once caught would be ready to cough up money in order to free themselves and it was this money that they (the tenant and police) would share.

Unfortunately in the end, the police doubled crossed him on the grounds that the money they made was not as much as he had claimed they would make.

Both stories were subsequently written, but here more than two decades after, we are reading the same story all over a clear indication that absolutely nothing had changed!

And this sadly is one of the biggest problems we face in this country failure to tackle issues head on in order to improve as a nation.

Instead we will all complain about poor governance and yet still vote for the same people that are the hindrance to the nation having decent governance.

At the end of the day, the path to a decent society lies with us as a people, who must then decide once and for all that enough is enough and we are now ready for real change and not just mouth change.

Unless this happens, I can bet that two decades down the line another Ojo Olajumoke will write another expose on virtually the same issue.

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When the hair falls off - New Telegraph Newspaper

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Although Lo Vauclare is just 3 years old, his kisses are known around the world.

A Franco-American living in Paris with his parents and little sister, Lo is the boy behind Bisous for Leo, a nonprofit organization named with the French word for kisses.

He loves swimming in the pool, listening to music and laughing. His big eyes light up behind a pair of large, round spectacles every he time he smiles which is often.

But he has trouble eating. He can no longer hold himself upright without support.

When Lo was born, he developed like any other child crawling, cruising and then finally taking his first steps. But his mother, Deborah Vauclare, said he never got past that. He would fall every few paces.

At about 15 months, Los parents took him to the doctor, who assured them that some children take a few more months to learn how to walk.

But when the toddler continued to falter, his parents worries grew. Extensive testing and therapy followed. Then they pursued genetic testing.

The results showed their son had Infantile Neuroaxonal Dystrophy or INAD, an extremely rare neurodegenerative disease that robs children of their fledgling abilities. It can weaken their muscles, taking away their ability to walk, hold up their own heads or control their eye movements, according to the INADcure Foundation.

Vauclare called the disease a mixture of Alzheimers and Parkinsons that impacts children.

Antoine, Deborah and Lo Vauclare are the family behind Bisous for Lo, a nonprofit organization that raises funds for research on INAD, a neurodegenerative disease that affects Lo.

(Courtesy of Deborah Vauclare)

The Vauclares had just received the devastating news last June when their close family friend, Emily Rogath Steckler, visited Paris with her daughter, Chloe.

The Stecklers had scarcely arrived at the Vauclares apartment when the two little friends perched together on the windowsill.

Chloe planted a kiss on Lo.

And from there, the nonprofit organization was born.

I literally couldnt get it out of my head, said Steckler, who co-founded Bisous for Lo with Vauclare. I just knew everybody needed to be embraced and to have the kiss.

Bisous for Lo has since raised more than $200,000 for gene therapy research at Washington University in St. Louis, Vauclare said.

The organization funnels funds through the INADcure Foundation to researchers addressing INAD and similar diseases. The organizations Facebook page collects posts tagged with kisses and stories from other children with INAD worldwide.

Recently, the group collaborated nationwide with Martin Lawrence Galleries, which is owned by Stecklers parents, David and Leslee Rogath. A portion of sales from reprints of artist Robert Deybers piece, Seal with a Kiss, will benefit Bisous for Lo.

Deyber is known for his artistic plays on literal phrases. His works include Love Sick, depicting a polar bear spewing hearts out of its mouth, and The Right to Arm Bears showing a polar bear standing beside a cannon.

Seal with a Kiss, a painting of a proud pinniped with a big red kiss on its cheek preening atop a rock, was the perfect fit for Bisous for Lo.

I think it may have popped up in a conversation or something. ... It will hit me, and Ill be like, Oh my God, seal! Deyber said with a laugh. Its gotten to be where its taken over my life at this point.

Funds from the lithograph sales of artist Robert Deybers piece Seal with a Kiss will be donated to the nonprofti organization Bisous for Lo.

(Courtesy of the Martin Lawrence Galleries)

Funds from the lithograph sales will support research on gene therapy and treatment taking place in the United States and Europe, Vauclare said.

The challenge, Vauclare added, is to convince researchers studying Alzheimers and Parkinsons to pay attention to INAD a disease that Vauclare said impacts only about 150 children worldwide.

Vauclare recently resigned from her job at an architectural firm so she can spend more time working on the nonprofit and supporting her son.

The second my kids go to bed at night, Im on the computer basically until my eyes shut, every single night, she said.

Lo, now 3, is participating in a clinical trial that will last until June. Despite trips between the United States and Paris for treatments, Vauclare said her son remains cheerful.

I definitely think as the disease progresses, we see a little bit less, she said. But even though we see less of it, its very pleasant in the morning when he wakes up and gives you a big smile. It takes him longer to connect with you.

With every social media post and painting sold, the Vauclare family hopes to kiss the disease goodbye.

Donations can be made at BisousForLeo.org and lithographs may be purchased at martinlawrence.com/pages/every-kiss-counts.

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Sealed with a kiss: how one little boy in Paris is stealing hearts in Costa Mesa - Los Angeles Times

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The National Institutes of Health (NIH) announced a partnership with the Bill and Melinda Gates Foundation on Wednesday to fund the development of targeted cures for HIV and sickle cell disease with a view to helping people in developing countries using gene therapy. With most of the populations affected by each disease residing in sub-Saharan Africa, treatments are being sought with regional conditions in mind.

The NIH and the Gates Foundation are investing $100 million in the initiative to develop low-cost gene therapies. The announcement follows President Donald Trump's pledge in his 2019 State of the Union address that the United States would eradicate HIV within the next decade. The Trump administration has also tried to draw more attention to sickle cell disease (SCD) in the past few years, according to a press release from NIH.

Sickle cell disease is a blood disorder that can cause anything from mild pain to heart failure. Human immunodeficiency virus (HIV) is a communicable disease that, if left untreated, wipes out the immune system. People with SCD inherit the disease from their parents, whereas HIV is acquired through blood contamination with certain bodily fluids of an infected person. While the mechanisms of transmission are different, both diseases are carried in the genome of infected individuals. Globally, both diseases also disproportionately impact individuals in lower-income communitiesand scientists believe that both could be combatted with gene-based treatments.

The past few years have seen unprecedented strides toward cures for these two diseases using gene therapy, which the NIH defines as experimental technique wherein doctors insert genes into a patient's cells so their body can more effectively resist a disease. It can include inserting a healthy variant of a gene to replace the unhealthy copy that causes a disease, or placing an entirely new gene in the body to fight the disease.

"Dramatic advances in genetics over the last decade have made effective gene-based treatments a reality... Yet these breakthroughs are largely inaccessible to most of the world by virtue of the complexity and cost of treatment requirements, which currently limit their administration to hospitals in wealthy countries," the press release states. The new initiative will focus on developing treatments that can be delivered in "low-resource settings."

Speaking on the initiative's viability, Dr. Ronald Mitsuyasu, a professor of medicine in hematology-oncology at the University of California, Los Angeles with more than 25 years of experience in HIV clinical trials research, told Newsweek that this sort of solution has been attempted in the past, but gene therapy hasn't yet proved successful in treating HIV.

"There have been several attempts to use gene therapy for HIV by either incorporating genes that suppress HIV genes, producing decoys for various viruses required processes needed for viral replication, or substituting inactive genes for functional genes of HIV," he said.

But those living in developing countries have not had as many chances to benefit from these solutions as those living in places like the U.S., according to the press release.

"SCD and HIV are major burdens on health in low-resource communities around the world," the press release read. "Approximately 95% of the 38 million people living with HIV globally are in the developing world, with 67% in sub-Saharan Africa, half of whom are living untreated. Fifteen million babies will be born with SCD globally over the next 30 years, with about 75% of those births occurring in sub-Saharan Africa."

Further, the prediction indicates that three-quarters of those infants will be born into low-income countries and communities. Between 50 and 90 percent of babies born with the disease in sub-Saharan African countries will die before the age of five, according to the release.

So, the NIH and the Gates Foundation's initiative aims to identify potential cures for both diseases as well as partner with groups in Africa to identify candidates on whom these new cures can be tested.

We are losing too much of Africa's future to sickle cell disease and HIV. Beating these diseases will take new thinking and long-term commitment. I'm very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africa's greatest public health challenges." Matshidiso Rebecca Moeti, M.B.B.S., the World Health Organization's regional director for Africa said of the initiative.

Mitsuyasu said he agreed that continued investigation into gene-based cures would eventually yield worthwhile results. "I personally believe that it should be possible to ultimately develop a gene therapy approach to overcome ... HIV," Mitsuyasu said. "Continued scientific developments in the field of gene therapy will eventually allow for the conquest of most genetic and viral gene integrated diseases."

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Gates Foundation, NIH Bet on Gene Therapy To Bring Cheap HIV and Sickle Cell Cures to Sub-Saharan Africa - Newsweek

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NEW YORK and BASEL, Switzerland, Oct. 23, 2019 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (AXGT), a clinical-stage company developing innovative gene therapies, today announced preliminary data from an expanded access study administering investigational AXO-AAV-GM2 gene therapy in two patients with infantile Tay-Sachs disease (TSD) at the 27th Annual Congress of the European Society of Gene and Cell Therapy. Infantile TSD, a rapidly progressive and fatal pediatric neurodegenerative genetic disorder, has a median life expectancy of approximately 3-4 years. This data indicates the potential to modify the rate of disease progression in children with infantile TSD.

Todays exciting clinical results from the AXO-AAV-GM2 program are the first reported evidence for potential disease modification in Tay-Sachs disease, and suggest an opportunity for gene replacement therapy to improve outcomes for children with this devastating condition, said Dr. Gavin Corcoran, chief R&D officer at Axovant. Myelination is an important component of healthy brain development in infants and is often abnormal in children with Tay-Sachs disease. We were encouraged to see MRI evidence of preserved brain architecture and improved myelination in the early symptomatic child treated at 10 months of age, coupled with stability of neuromuscular function as measured on the CHOP INTEND scale. We look forward to Dr. Terry Flottes presentation of this data at the ESGCT conference where he will describe these two clinical cases in detail.

Key findings from this first-in-human study in patients treated with AXO-AAV-GM2, an investigational gene therapy designed to restore -Hexosaminidase A enzyme activity in the central nervous system, include:

*CHOP INTEND is a 16-item scale of motor function that has been validated in infants with neuromuscular disorders. Items of motor function are graded from 0 to 4 for each item, where zero equals no response, and 4 equals a complete response. Change from baseline in total score of 4 points or a total score sustained > 40 points has been associated with a clinically meaningful benefit.

Dr. Terence Flotte, Professor of Pediatrics and Dean at the School of Medicine, University of Massachusetts Medical School, said, Bilateral intrathalamic and intrathecal delivery of rAAV gene therapy may surmount the obstacle of providing widespread distribution of therapeutic enzyme throughout the brain and CNS. This innovative delivery could overcome one of the primary challenges for developing treatments for Tay-Sachs, Sandhoff and many other severe pediatric genetic disorders, providing much needed hope for these families.

Dr. Flotte will present this data on AXO-AAV-GM2 in the First-in-Human gene therapy session on October 23, 2019 at 5:30 PM Central European Time (CET).

About AXO-AAV-GM2

AXO-AAV-GM2 is an investigational gene therapy for Tay-Sachs and Sandhoff disease, which rare and fatal pediatric neurodegenerative genetic disorders within the GM2 gangliosidosis family, caused by defects in theHEXA(leading to Tay-Sachs disease) orHEXB(leading to Sandhoff disease) genes that encode the two subunits of the -hexosaminidase A (HexA) enzyme. Both forms of GM2 gangliosidosis are caused by overwhelming storage of GM2 ganglioside within neurons throughout the central nervous system), which is normally degraded in the lysosome by the isozyme HexA. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA levels by introducing a functional copy of theHEXAandHEXBgenes via delivery of two co-administered AAVrh8 vectors.

In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

About Axovant Gene Therapies

Axovant Gene Therapies, part of the Roivant family of companies, is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinsons disease.

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Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visitwww.axovant.com.

About Roivant

Roivantaims to improve health by rapidly delivering innovative medicines and technologies to patients.Roivantdoes this by buildingVants nimble, entrepreneurial biotech and healthcare companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. Roivant today is comprised of a central technology-enabled platform and 20 Vants with over 45 investigational medicines in clinical and preclinical development and multiple healthcare technologies. For more information, please visitwww.roivant.com.

About the University of Massachusetts Medical School

The mission of the University of Massachusetts Medical School is to advance the health and well-being of the people of the commonwealth and the world through pioneering education, research, public service and health care delivery.

Research into potential therapies for lysosomal storage diseases such as Tay-Sachs, Sandhoff disease and GM1 gangliosidosis at UMass Medical School and Auburn University has led to significant advances in the field. Miguel Sena-Esteves, PhD, associate professor of neurology at UMass Medical School; Heather Gray-Edwards, PhD, DVM, formerly of Auburn and currently assistant professor of radiology at UMass Medical School; and Douglas Martin, PhD, professor of anatomy, physiology and pharmacology in the College of Veterinary Medicine and the Scott-Ritchey Research Center at Auburn University, have worked collaboratively for more than a decade on animal models and therapeutic approaches for these and similar disorders. For more information, visit http://www.umassmed.edu.

Forward Looking Statements and Information

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, believe, estimate, and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the initiation, timing, progress, and reporting of results of its preclinical programs, clinical trials, and research and development programs; cash to be used in operating activities; its ability to advance its gene therapy product candidates into and successfully initiate, enroll, and complete clinical trials; the potential clinical utility of its product candidates; its ability to continue to develop its gene therapy platforms; its ability to develop and manufacture its products and successfully transition manufacturing processes; its ability to perform under existing collaborations with, among others, Oxford Biomedica, and theUniversity of Massachusetts Medical School, and to add new programs to its pipeline; its ability to enter into new partnerships or collaborations; its ability to retain and successfully integrate its leadership and personnel; and the timing or likelihood of its regulatory filings and approvals are forward-looking. All forward-looking statements are based on estimates and assumptions by Axovants management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected.Such risks and uncertainties include, among others, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the expectations for regulatory submissions and approvals; the continued development of its small molecule and gene therapy product candidates and platforms; Axovants scientific approach and general development progress; and the availability or commercial potential of Axovants product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovants most recent Quarterly Report on Form 10-Q filed with theSecurities and Exchange CommissiononAugust 9, 2019, as updated by its subsequent filings with theSecurities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made.Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts:

Media and Investors

Parag MeswaniAxovant Gene Therapies (212) 547-2523investors@axovant.commedia@axovant.com

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Axovant Presents First Evidence of Clinical Stabilization in Tay-Sachs Disease at the European Society of Gene and Cell Therapy 27th Annual Congress -...

Recommendation and review posted by Bethany Smith

Here we go again.

This week, investors may have seen that a new gene editing technique will soon be available to scientists attempting to engineer DNA for human health applications. Dubbed "prime editing," some have called it a game-changer due to its inherent advantages over more commonly used CRISPR techniques. It has the potential to correct up to 89% of disease-causing mutations and is engineered to make more precise edits than other gene editing techniques. And it all belongs to a new start-up: Prime Medicine.

But despite all of the media hype, prime editing has one enormous problem that may make it impossible to commercialize: The molecular components are way too big to be delivered into cells using current methods. It's as if an automaker designed a car with an amazing interior, but forgot to add wheels.

The "big" obstacle facing prime editing doesn't necessarily mean CRISPR Therapeutics (NASDAQ:CRSP), Editas Medicine (NASDAQ:EDIT), and Intellia Therapeutics (NASDAQ:NTLA) will have smooth sailing ahead, but it does highlight an important facet of gene editing technologies that investors can't overlook: delivery.

Cutting both strands of DNA is bad. Image source: Getty Images.

Healthcare investors are probably familiar with the basics of how CRISPR gene editing works by now, but a little refresher can't hurt.

The payload comprises a guide RNA and a cutting enzyme such as Cas9. The guide RNA helps the payload locate the target sequence of DNA that will be edited. Once located, the payload binds to the target sequence of DNA, and the cutting enzyme makes a double-stranded break (meaning it cuts through both strands of DNA) to remove or insert DNA.

But there are problems. The guide RNA and cutting enzyme aren't always 100% accurate, and can sometimes edit the genome relatively far from the target site. That can introduce changes to the genome, which can be harmless -- or potentially harmful.

An even larger problem is that traditional CRISPR gene editing techniques make double-stranded DNA breaks that require one of two natural DNA repair mechanisms to patch up. Unfortunately, there can be significant consequences to this, such as allowing a dormant cancer cell to proliferate and reduced editing efficiency. This problem isn't alleviated by using different cutting enzymes, such as switching from Cas9 to Cas12.

While CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics have gone all-in on CRISPR-Cas systems, many scientists aren't confident that the technique will deliver on its promise in a clinical setting. But the learning experiences in the lab have spurred a search for new gene editing techniques that don't make as many off-target edits and don't make double-stranded breaks. Prime editing is the latest tool.

Cutting one strand of DNA is better. Image source: Getty Images.

Prime editing makes some notable modifications to the traditional CRISPR-Cas setup. The cutting enzyme, Cas9, has been modified to only cut one strand of DNA. The guide RNA deployed also contains the instructions for the desired edit. And a second enzyme, called reverse transcriptase, has been added to the payload to write the desired edits into the exposed strand of target DNA.

The changes allow prime editing to change any DNA base -- A, T, C, or G -- into any other. Prime editing has also been used to insert up to 44 base letters and delete up to 80 base letters from target DNA in limited studies. It may not seem like much considering the human genome contains over 3 billion base pairs, but the characteristics described above could allow the new technique to correct up to 89% of disease-causing mutations in humans.

The versatility is pretty amazing, but the problem is that prime editing payloads -- comprising two enzymes and a longer guide RNA -- are too large to be delivered with today's delivery vehicles, such as adeno-associated viruses (AAV); after all, enzymes are big. The field of gene therapy only recently overcame the hurdle of delivery, and gene editing is still trying to figure it out.

CRISPR Therapeutics has (perhaps wisely) kicked that can down the road by focusing initial efforts on ex vivo editing, or editing cells in a controlled environment outside of the human body, where delivery is somewhat less complicated. Editas Medicine has taken up the mantle of being the first to try in vivo editing, or editing cells directly in the human body. It's using AAV delivery vehicles initially.

Meanwhile, Intellia Therapeutics has fallen out of favor with investors because its slow-and-steady approach won't let it enter clinical trials until 2020 or 2021. But investors may be missing the point. Rather than plowing ahead with AAV delivery, the company is working to develop new and novel delivery systems based on lipid nanoparticle (LNP) tech licensed from Novartis. It could end up being the main differentiator in the CRISPR gene editing space, and the move is entirely aimed at solving the tricky problem of delivery.

There's also Precision BioSciences (NASDAQ:DTIL), which has a proprietary gene editing system called ARCUS. The tech is completely shielded from CRISPR patent disputes, doesn't make double-stranded DNA cuts, is ahead of CRISPR Therapeutics in an important clinical area, and can diversify its revenue by forging into agriculture or industrial biotech. It relies on AAV delivery vehicles.

Simply put, at a time when gene editing techniques are attempting to figure out the best solution to overcome delivery obstacles, prime editing went all-in on optimizing other characteristics of the tool, while betting the delivery problem will solve itself. That's a risky bet.

Image source: Getty Images.

Luckily for scientists and investors, the swift rise of CRISPR gene editing in research settings has led to the development of robust testing tools. That should shorten the learning curve for researchers wanting to know whether or not prime editing is going to live up to the hype -- or overcome its big delivery problem.

Nonetheless, the media hype highlights that individual investors need to be more selective in their approach to gene editing stocks. Tools that make double-stranded breaks (read: CRISPR) are likely to have a high rate of failure, but ex vivo applications in engineering immune cells could yield success. That would bode well for CRISPR Therapeutics and Precision BioSciences.

The delivery problem will make in vivo editing much more difficult, which could be a problem for Editas Medicine's lead drug candidates and future areas of focus of CRISPR Therapeutics. Intellia Therapeutics is hopeful its LNP approach could solve the problem, but it's too soon to know.

That said, there's one gene editing technique that could strike a balance between traditional CRISPR-Cas systems and prime editing: base editing. It was developed by the same lab that pioneered prime editing and belongs to a start-up called Beam Therapeutics, and Editas Medicine may already have one foot in the doorto licensing the tech. Then again, the gene editing space is very young and rapidly evolving, so investors cannot dismiss the inherent risks of owning gene editing stocks.

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There's Yet Another Gene Editing Technique, but It Has 1 Glaring Problem - Motley Fool

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Newswise October 25, 2019 Major burns lead to changes in the gastrointestinal tract bacteria, and these alterations of the gut microbiome are influenced by resuscitation with intravenous (IV) fluids, according to animal studies reported inSHOCK: Injury, Inflammation, and Sepsis: Laboratory and Clinical Approaches,Official Journal of theShockSociety. The journal is published in the Lippincott portfolio byWolters Kluwer.

Potentially disease-causing gut bacteria are increased after a major burn, while large volumes of IV fluids promote recovery of beneficial bacteria, suggest the experiments by David Burmeister,PhD, of the US Army Institute of Surgical Research and colleagues. They write, "This study is the first to show that the gut microbiome is altered following a large burn injury in pigs and that the gut microbiome may be influenced by the resuscitation strategy used."

Gut Microbiome Changes May Affect Outcomes After Major Burns

Burnsdon'tjust cause skin damage they result in physiological stress and inflammation that may lead to infections and other serious complications. While a growing body of research has shown that the gut microbiome has important effectsforhealth and disease, little is known about how burns may alter the microbiome.

The researchers examined changes in the gut bacteria after burns and how those alterations are affected by fluid resuscitation. Major burns, covering 40 percent of body surface area, were induced in anesthetized swine. Because their skin and gastrointestinal tract are similar to those of humans, pigs are an important model for studying burns and their treatment.

The animals then received low- or high-volume fluid resuscitationor no IV fluids. Effects on gut microbiome diversity the proportions of different types of bacteria were analyzed by isolating and analyzing bacterial DNA.

The results showed significant changes in the gut microbiome after burns, with disruption of the normal balance between different groups of bacteria. Findings included a "hyperacute spike" in one major group called Proteobacteria, which includes many potentially pathogenic (disease-causing) organisms.

In contrast, high volumes of resuscitation fluids seemed to promote recovery of potentially beneficial microbes, such asBacteroidesbacteria. SinceBacteroidesplays a role in converting sugar into energy, this shift might help in coping with the increased metabolic demands on the body after burns. Fluid resuscitation also prevented increases in certain opportunistic bacteria, which might protect against the risk of sepsis and other serious infections.

Resuscitation fluids also seemed to affect some functional proteins in the gut for example, heat shock proteins that promote resistance to stress-induced cell damage. That provides a clue to understanding how fluid resuscitation helps the body recover from inflammation and other metabolic changes after burns.

Despite increasing evidence on the role of the gut microbiome, there has been little information on how bacterial diversity in the gut is affected by burns and their treatment. While early, aggressive fluid resuscitation is a key part treatment for burns,debatecontinues over the resuscitation strategies used.

Dr. Burmeister and colleagues call for further studies to clarify shifts in microbiome diversity after burns, and how these changes affect the outcomes of burns and responses to fluid resuscitation. Future research might lead to new treatments targeting the microbiome for example, using fecal transplant or antibiotics to promote recovery after burns. The researchers add, "Future clinical trials of resuscitation strategies should include gut microbiome analysis."

Click here to read "The Effect ofBurnResuscitationVolumes on theGutMicrobiomein aSwineModel"

DOI:10.1097/SHK.0000000000001462

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AboutSHOCK

SHOCK:Injury, Inflammation, and Sepsis: Laboratory and Clinical Approachesincludes studies of novel therapeutic approaches, such as immunomodulation, gene therapy, nutrition, and others. The mission of the Journal is to foster and promote multidisciplinary studies, both experimental and clinical in nature, that critically examine the etiology, mechanisms and novel therapeutics of shock-related pathophysiological conditions.SHOCK is the Official Journal of theShock Society, the European Shock Society, the Indonesian Shock Society, the International Federation of Shock Societies, and the Official and International Journal of the Japan Shock Society

About Wolters Kluwer

Wolters Kluwer is a global leader in professional information, software solutions, and services for the health, tax & accounting, finance, risk & compliance, and legal sectors. We help our customers make critical decisions every day by providing expert solutions that combine deep domain knowledge with specialized technology and services.

Wolters Kluwer reported 2018 annual revenues of 4.3 billion. The group serves customers in over 180 countries, maintains operations in over 40 countries, and employs approximately 18,600 people worldwide. The company is headquartered in AlphenaandenRijn, the Netherlands.

Wolters Kluwer provides trusted clinical technology and evidence-based solutions that engage clinicians, patients, researchers and students with advanced clinical decision support, learning and research and clinical intelligence. For more information about our solutions, visithttp://healthclarity.wolterskluwer.comand follow us onLinkedInand Twitter@WKHealth.

For more information, visitwww.wolterskluwer.com, follow us onTwitter,Facebook,LinkedIn, andYouTube.

Link:
Gut Microbiome Is Altered by Burns, Affected by Fluid Resuscitation: New Evidence in SHOCK - Newswise

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Global Gene Therapy for CNS Disorders Market 2019 - Gilead (Kite Pharma), Amgen (BioVex), Novartis, Roche (Spark Therapeutics), Bluebird Bio -...

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