Nathan Anderson and two of his four sons, Griffin, 10, and Brennan, 4, have been diagnosed with a rare mutation on their Runx1 gene. Besides causing easy bruising, excessive bleeding and a tendency to develop hematomas, the mutation predisposes them to certain blood cancers. Wife and mother, Joy Anderson, has become an outspoken advocate for the condition and her family.

As Joy Anderson tucked her four boys into their beds one recent evening, she asked each of them to reflect on the difficult year the family has had.

Oldest son Griffin, 10, expressed anger and sadness, especially because he isnt allowed to play contact sports anymore.

Maxwell, 9, said hes worried about his siblings and parents.

Nolan, 8, said hes tried to be more compassionate, as you never know what someone is facing.

And then theres sweet-yet-rambunctious Brennan. At just 4 years old, he doesnt really understand whats been going on, only that hes had many doctors visits and needle pricks lately.

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A little over a year ago, the Anderson family received news that rocked their world: Brennan had tested positive for the Runx1 gene mutation.

It was the third such blow for the family, who hails from Arlington, a village in Hancock County outside of Findlay, in just a few short months. Griffin first received the same diagnosis in August 2018, followed by their father, Nathan, in November that year and now Brennan.

Maxwell and Nolan, fortunately, do not share the mutation.

"Its like a 50-50 chance of being passed down," Joy said. "In that case, I felt like we were a living statistic."

But their situation is anything but routine: Only 120 individuals in the world have been diagnosed with the mutation.

Visible symptoms of the disease, which causes platelet abnormalities, include easy bruising, excessive bleeding and a tendency to develop hematomas. But the most distressing part is its predisposition (40% to 70%) to certain blood cancers.

"I felt like I couldnt breathe, like I was having a panic attack," the 41-year-old mother said of the diagnoses. "I tried to Google everything to learn more about it, and there isnt really much out there."

Three people she loves dearly half her family now must be followed regularly by a hematologist and undergo bone marrow biopsies annually to monitor for cancer.

"Little did I know we would have such a domino effect after we all got tested," she said.

***

The Andersons had spent much of the first nine years of Griffins life trying to figure out what caused his bruising and the huge goose eggs that would form on his body.

Sure, he was an active boy, but with each small accident, his parents couldnt help notice the extreme results that sometimes landed him in the hospital to ensure his blood had clotted.

Doctors in Toledo offered a general diagnosis of low platelets and a blood disorder when Griffin was 3 and prescribed special medication to use during surgeries.

He began seeing various hematologists at Nationwide Childrens Hospital. However, the family still had few answers until a doctor suggested genetic testing in August 2018.

Thats when the Andersons met Elizabeth Varga, a genetic counselor at Childrens, who counseled them what results might yield.

Four weeks after Griffin had blood drawn for the genetic tests, Varga called to say they found something.

Runx1 is a gene involved in the making of blood cells. If there is an abnormality, it can impair the production of platelets, which help blood clot.

Patients are predisposed "to something called myelodysplastic syndrome, which is essentially a pre-leukemia state that can evolve and change to be a cancer of the blood," Varga told the family.

Typically, thats acute myeloid leukemia, which is most common in adults over 60, but the Runx1 mutation increases the risk for AML in all ages.

"So basically from the time of birth youre kind of set up to have that evolution," Varga said. "However, not all patients that have a Runx1 abnormality will ever get cancer right now we dont have a great way to gauge who will and who wont."

To deliver this news to Joy was very difficult, said Varga, who has three young sons.

Each phone call, Joy said, felt like a "sucker punch," as she struggled to learn what this would mean for their family.

***

In the days leading up to Griffin and Brennans first bone marrow biopsy in December 2018, the elder brother watched YouTube videos of the minor but uncomfortable procedure, which is the best way to monitor changes.

"I was interested in it," the fifth grader said. "I want to be an ER doctor when I grow up."

Joy and Nathan Anderson said its a bit of an odd blessing that two sons have the diagnosis as they dont have to be alone.

"I usually tell Brennan to be brave and we can do it," Griffin said, adding that his youngest brothers silly antics calm his nerves.

The mutation presents differently in Griffin and Brennan. Both bruise easily, but its much more pronounced in Griffin.

Brennan has dealt with a slew of pulmonology issues asthma, seven bouts with pneumonia that may be linked with the gene mutation.

Discovered only 20 years ago, very little research has been done on the mutation, making it difficult to discern what symptoms are caused by it, said Katrin Ericson, executive director of the Runx1 Research Program, a California-based nonprofit group that funds research and provides patient support.

Earlier this year, Joy Anderson was the first patient family representative to speak at the organizations annual conference. Shes become very active in increasing awareness of the Runx1 mutation, which is underdiagnosed, Ericson said.

Based on epidemiological estimates, between 2,000 and 18,000 people in the United States could be living with it.

Ericson said the organization is thrilled that, in May, the National Institutes of Health launched the first longitudinal, natural history study of Runx1. The Andersons are one of 25 or so families participating, and they traveled to Bethesda, Maryland, in June for testing.

"These patients have really been struggling with this most of their lives," Ericson said. "They had no idea they had this mutation. Maybe they were misdiagnosed at first."

There can be guilt felt by parents for passing it unknowingly to their children, and Joy said her husband feels this way.

Nathan his parents tested negative never experienced symptoms, he said. However, looking back, routine blood work often showed low platelet counts.

"Shortly after the diagnosis, I was at a charity event for work and I was chopping wood," Nathan said. "I noticed that I had bruises up and down my arm."

During his first bone marrow biopsy at Ohio State Universitys Arthur G. James Cancer Hospital last December, doctors discovered he had myelodysplastic syndrome (MDS), or pre-leukemia. Hes unsure what this means for the future, except continued monitoring.

Nathan and his two sons will travel to the NIH in the summer for follow-up biopsies. (Griffin and Brennan had a second one in June with zero changes.)

The boys also have blood drawn every few months.

"Our hope is if we do more frequent surveillance for MDS, that we will hopefully be able to be preemptive," Varga said. "If we do see any progression, the only cure right now is a bone marrow transplant."

However, a bone marrow transplant which requires chemotherapy and a lifetime of immunity-suppressing drugs is a procedure that wouldnt be done without good reason, Varga said.

***

One of the biggest challenges of having a genetic disorder, Joy and Nathan agreed, is explaining it to others.

"Some people think were dying and some think were getting chemotherapy," said Nathan, who has taken up running to cope with the diagnosis and keep himself healthy. "Others think its not a big deal."

People have questioned the necessity of "putting our kids through all this," Joy said. Others have said that everyone would find something wrong if they did genetic testing, she said.

"Were trying to monitor and learn more," she said. "Im all about being proactive. I feel like what if we never even did anything about it, and then one day this has progressed to leukemia and its so far into it, we cant do anything to help."

That attitude, Varga said, represents a shift shes detected.

"Previous generations, there was much more of I dont want to know," Varga said. "There was a fear of stigma or discrimination, but younger generations are more embracing (of) knowledge and being powerful."

***

For now though, the Anderson boys will continue to climb on top of their swing set and run in the field behind their house. Theyll fish and ride bikes and wrestle.

"Theyre boys still, and we want them to live a normal life," Nathan said. "We dont stop."

Griffin has recently fallen in love with playing drums and though his parents dont always appreciate the noise, theyre thankful Griffin has found an outlet other than sports.

The family is thankful for the power the knowledge of this diagnosis ultimately brings them, and with how strong it has shown the six of them to be.

"Its a reminder of just how precious life is," Nathan said. "You might live a long life or you might run into complications. Its a constant reminder to live every day to the fullest."

award@dispatch.com

@AllisonAWard

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Ohio family faces uncertainty as 2 sons, husband diagnosed with rare genetic disorder - The Columbus Dispatch

Recommendation and review posted by Bethany Smith

Its been a banner year for First Opinion. We published nearly 500 essays written by more than 600 authors from industry, academia, government, and private life in the United States and beyond. They took on the mundane, like the implications of the Elijah E. Cummings Lower Drug Costs Now Act (aka H.R. 3), and the fanciful, like why we need a Public Domain Day to highlight when drugs go off patent.

Authors addressed the opioid crisis, patent thickets, the personal toll imposed by drug shortages, drug costs, deaths of despair, the oxymoron of having a waiting room in an emergency department, shoddy production of generic drugs by foreign manufacturers, the burgeoning use of artificial intelligence and machine learning in new drug research and development and health care, and much, much more.

Here are the five most widely read First Opinions of 2019. If you didnt get to read them when they first appeared, now is as good a time as any:

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1. 23andMe had devastating news about my health. I wish a person had delivered it Dorothy Pomerantz thought it would be fun to have her DNA analyzed, so she sent her spit to 23andMe. A link in an email took her to the companys website, where she learned she was at very high risk of developing breast and ovarian cancer. Im not the first person to get surprising and terrible news from an at-home genetic testing company. With the increasing popularity of 23andMe, and other companies like it, I wont be the last. But she wished she had gotten the news from a person.

2. Time in range: a new way for people with diabetes to monitor blood sugar More and more people with diabetes are using continuous glucose meters, which measure blood sugar every few minutes. That information offers a new way to evaluate how well someone is controlling his or her diabetes: time in range. The University of Washingtons Dr. Lorena Alarcon-Casas Wright explains how it works.

3. Our child received a devastating diagnosis before she was born. We decided to protect her Allison Chang learned that, at 15 weeks of gestation, her daughter had severe malformations due to trisomy 18, a deadly genetic condition. She and her husband could not protect our daughter from trisomy 18, but we could shield her from any pain or agony that would come with it.

4. I have spinal muscular atrophy. Critics of the $2 million new gene therapy are missing the point Nathan Yates has lived with spinal muscular atrophy for all 30 years of his life. He answers critics upset over the $2.1 million cost of Zolgensma, a new drug to treat the condition, and asks them to take into account its long-term benefits.

5. Ghost networks of psychiatrists make money for insurance companies but hinder patients access to care When Jack Turban started his training to become a psychiatrist, he went looking for a therapist of his own. What he found were ghost networks of mental health providers.

And heres a bonus: Published in July 2018, the essay Physicians arent burning out. Theyre suffering from moral injury was the third most widely read First Opinion of 2019.

On to 2020!

Read more here:
5 most widely read First Opinions of 2019 - STAT

Recommendation and review posted by Bethany Smith

This article, written byMichael Mackley, Dalhousie University, originally appeared on The Conversation and is republished here with permission:

Youve likely heard about direct-to-consumer DNA testing kits. In the past few years, at-home genetic testing has been featured in the lyrics of chart-topping songs, and has helped police solve decades-old cold cases, including identifying the Golden State Killer in California.

Even if you dont find a DNA testing kit under your own Christmas tree, theres a good chance someone you know will.

Whether youre motivated to learn about your health or where your ancestors came from, it is important to understand how these tests work before you spit in the tube.

While exciting, there are things that these genetic testing kits cannot tell users and important personal implications that consumers should consider.

Health, traits and ancestry kits

My main area of research is around clinical genome sequencing, where we look through all of a persons DNA to help diagnose diseases. With a PhD in genetics, I often get questions from friends and family about which direct-to-consumer genetic test they should buy, or requests to discuss results. Most questions are about two types of products: ancestry and health kits.

The most popular ancestry kit is from AncestryDNA. These kits are aimed at giving users insight into where their ancestors might be from. They can also connect users with family members who have used the service and have opted into having their information shared. Another option is Living DNA, which has a smaller dataset but provides more precise information on the U.K. and Ireland.

The most popular health kit is from 23andMe. Depending on the users preference, results include information on predispositions for diseases such as diabetes and Alzheimers, as well as on the likelihood of having certain traits such as hair colour and taste. This company also offers ancestry analysis, as well as ancestry and trait-only kits that dont provide health information. The kit offered by the newer MyHeritage DNA also provides a combined ancestry and health option.

There are other kits out there claiming to evaluate everything from athletic potential to relationship compatibility. But gift-buyers beware: for most of these, in contrast to those above, the evidence is seriously lacking.

How these tests work

For all of these tests, customers receive a kit in the mail. The kits contain instructions for collecting a saliva sample, which you mail back to the company for analysis.

During this analysis, these popular tests do not look at the entire genome. Instead, they employ single nucleotide polymorphism (SNP) genotyping. As humans we all share 99.9 per cent of our DNA. SNPs are essentially what is left: all of the points at which we can differ from our neighbour, making us unique. SNP genotyping looks at a subset of these sites to survey the users genome.

These SNPs are then compared to reference datasets of individuals with known conditions or ancestry. Most results are based on the SNPs shared with a given group. For example, if your results say that you are 42 per cent Southeast Asian, its because 42 per cent of your SNPs were most likely to have come from a group in the reference dataset labelled Southeast Asian. The same goes for traits and health conditions.

How they differ from clinical tests

Direct-to-consumer genetic tests are not a substitute for clinical assessment. The methods used differ dramatically from what is done to diagnose genetic diseases.

In a clinical setting, when suspicion of a genetic condition is high, entire genes are often analyzed. These are genes where we understand how changes in the DNA cause cellular changes that can cause the disease. Furthermore, clinical assessment includes genetic counselling that is often key to understanding results.

In contrast, findings from direct-to-consumer genetic tests are often just statistical links; there is commonly no direct disease-causing effect from the SNPs.

Users may interpret a result as positive, when the risk increase is only minimal, or entirely false. These tests can also give false reassurance because they do not sequence genes in their entirety and can miss potentially harmful variants.

Before you spit in a tube, stop and think

These tests are exciting: they introduce new audiences to genetics and get people thinking about their health. Theyre also helping to build vast genetic databases from which medical research will be conducted.

But for individual users, there are important caveats to consider. Recent reports have questioned the accuracy of these tests: identical twins can receive different results. Furthermore, a lack of diversity in the reference data has caused particular concern regarding accuracy of results for ethnic minorities.

There are also concerns about the way these tests emphasize racial categories that science considers to be social constructs and biologically meaningless.

A recent paper in the British Medical Journal suggests four helpful questions for users to consider. First, users should ask themselves why they want the test. If it is to answer a medical question, then they should speak with their doctor. Users should also think about how they might feel when they receive results containing information they would rather not know.

Users should also consider issues around security and privacy. It is important to read the fine print of the service youre using, and determine whether youre comfortable sharing personal information, now and in the future.

In Canada, policies around genetics have not always kept up with the science. At present, direct-to-consumer genetic testing is unregulated. And, although Canadians have legislative protections against genetic discrimination, those laws are being challenged in the courts, and could change.

Finally, it may also be worth discussing DNA testing with relatives. We share half of our genome with our immediate family members, and smaller fractions with more distant relatives. Genetic results not only affect us, but our family.

Bottom line: Its all for fun

Some users may feel they learn more about themselves. For others, results may bring people closer together not a bad outcome for the holiday season.

At the end of the day, these genetic testing kits are for entertainment: they should not be used to assess health risk in any meaningful way.

If you have any questions related to your health or a genetic disease, discuss these with your family doctor or a suitable health-care professional.

Michael Mackley, Junior Fellow, MacEachen Institute for Public Policy and Governance; Medical Student, Dalhousie University

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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DNA tests might be a fun holiday gift, but beware of the hype - HalifaxToday.ca

Recommendation and review posted by Bethany Smith

In this last full stock trading week of 2019, let's tip our collective hat to the markets. It was quite a year for equities, with major indexes rising to fresh all-time highs on a frequent basis and some of our favorite top stocks reaching the same milestone.

That said, we're seeing a few late-in-the-game stumbles from certain companies listed on the various exchanges. Here's a pair that were nursing a Santa hangover on the day after Christmas.

Image source: Getty Images

Shares of Qiagen(NYSE:QGEN) were eviscerated by almost 21% on Boxing Day, following news that a hoped-for takeover is not going to happen.

The Germany-based company announced the day before Christmas that it has opted to go it alone rather than sell itself to an outside entity. This isn't the gift many investors were hoping for from the genetic testing specialist -- in November it temptingly announced that it had been the target of "several conditional, non-binding indications of interest for a full acquisition." The stock promptly took off like a rocket.

So Thursday's fall to Earth is not surprising. It also brings back concerns investors had before the takeover speculation. After all, Qiagen's latest reported quarter indicated some bumps in the road, with a second consecutive reduction of full fiscal year sales guidance (blamed on difficulties in the Chinese market) and concerns about leadership following the departure of longtime CEO Peer Schatz.

Although the company recently signed up for a potentially promising, long-term collaboration with Illuminain next-generation sequencing diagnostics, we have no pertinent details on the arrangement. Given that, plus uncertainties over Qiagen's other business areas and the pesky matter of the CEO job, it might be best to leave the stock alone just now.

Planet Earth wasn't the only home of downer stocks on Thursday. Far up in space, Virgin Galactic Holdings (NYSE:SPCE) saw a downward trajectory of more than 4% across the day.

It's very possible that a deal announced on Thursday by SpaceX, Virgin Galactic's rival in otherworldly exploration, was the culprit. Privately held SpaceX won a contract from NASA to supply parts for exploratory vehicles.

This isn't a planet-sized deal, as it only totals about $7.5 million. Besides, although Virgin Galactic's ambitions overlap those of SpaceX, the former company is not a maker of components. However, the deal does indicate that there is revenue to be made in the private space sector, and Virgin Galactic still isn't making much (or any, if you don't count deposits for potential space flights by well-to-do tourists).

Somewhat surprisingly, for a company that could be a poster boy for speculative early-stage investments, Virgin Galactic has quite a few admirers in the investment community. Earlier this month, it racked up its third buy recommendation from a noted investment bank (Morgan Stanley, to be exact), which feels it has a lot of upside if it can manage to become the first viable space tourism operator.

That's an awfully long stretch to wait, though, given the monster costs and unproven model of this particular business. I think Virgin Galactic will be a fun stock to watch, but I personally am not about to invest in it.

Read more here:
2 Stocks That Tumbled on Thursday - The Motley Fool

Recommendation and review posted by Bethany Smith

According to news articles published in early December, Veritas Genetics, a Massachusetts-based company that hoped to lower the cost of whole-genome sequencing, is suspending its U.S. operations because of a lack of investment. Articles theorize that the decreased funding was driven mainly by new CFIUS regulations and heightened CFIUS scrutiny.

Early in December 2019, Veritas announced that its adverse financing situation had forced the suspension of its U.S. business. Veritas has stated that it is assessing potential paths forward, and there are rumors that one such path is the sale of the company. Veritas will no longer sell its tests, which include genetic testing for diseases and cancers (such as the BRCA test), in the United States. Veritas will continue to operate and sell its tests outside the United States.

Veritas first launched in 2014, and since 2015 it had raised $50 million in financing. Major investors included Chinese companies, such as Lilly Asia Ventures, which invested $10 million into the company, and Simcere Pharmaceutical. However, there has been increased scrutiny in the past two years for transactions that involve Chinese investors, especially when sensitive personal information, such as genetic information, is at stake. This year, for example, CFIUS forced iCarbonX, the Chinese, majority owner of U.S. company PatientsLikeMe, to divest its stake in the U.S. company.

According to news reports, recent CFIUS activity may have scared away not only Chinese investors but also non-Chinese investors reluctant to invest in a company with Chinese ownership. Non-Chinese investors may fear that Veritass Chinese ownership will lead to increased CFIUS scrutiny of any investment into Veritas, regardless of the investors nationality. Investors may also worry that CFIUS scrutiny could delay their return on investment if their firms are forced to stall business to address CFIUSs concerns.

No doubt the proposed CFIUS regulations from September also concern foreign investors: the proposed regulations explicitly target U.S. companies that maintain or collect sensitive personal data of U.S. citizens. While most sensitive personal data only triggers the proposed regulations if the U.S. business maintains or collects such data on greater than one million individuals, companies with genetic data are considered to be covered businesses no matter how many individuals are involved. Thus, companies like Veritas will always fall under CFIUS jurisdiction if a foreign person would acquire certain rights in the company. These rights include:

Several genetic and biopharmaceutical companies expressed concern in public comments to the regulations that the proposed regulations, specifically including all genetic data in the definition of sensitive personal data, would stymie foreign investment in these companies. Several companies argued that the Department of the Treasury should revise the proposed CFIUS regulations to require that genetic data be identifiable. Companies often are in possession of anonymized genetic information, which these companies argued does not pose a risk to national security. We await publication of the final regulations and whether CFIUS will make any changes to the definition of sensitive personal data, particularly as it pertains to genetic information. It is to be seen whether U.S. companies in other industries will face similar funding obstacles as foreign investors grow more wary of CFIUS.

Originally posted here:
Veritas, a US Genetic Sequencing Company, Suspends US Operations Due to Decreased Funding; CFIUS Thought to be Leading Cause - Lexology

Recommendation and review posted by Bethany Smith

Check out the companies making headlines before the bell:

Boeing Boeing remains on watch, after new documents reviewed by a congressional panel revealed what's being called "very disturbing" revelations regarding the grounded 737 Max jet, according to a congressional aide quoted by Reuters. Boeing issued a statement saying it had proactively brought the documents to the FAA and Congress, and said the tone and content do not reflect "the company we are and need to be".

Amazon.com Amazon said the holiday shopping season broke all prior records, with "billions" of items ordered worldwide and "tens of millions" Amazon devices purchased.

Qiagen Qiagen said it decided against a sale of the company following a review. The Netherlands-based genetic testing firm said it determined that operating as a stand-alone business is its best option. Qiagen said it had gotten several indications of interest, with reports saying one of those potential bids came from medical device maker Thermo Fisher Scientific.

PayPal PayPal will continue to pursue potential takeover targets in 2020, according to Chief Financial Officer John Rainey. He told the Wall Street Journal there are many acquisition opportunities in the payments sector, with PayPal targeting transactions in the $1 billion to $3 billion range.

Exxon Mobil, Chevron These and other oil stocks could get a boost as oil prices touch their highest in more than 3 months, boosted by a report showing lower U.S. crude inventories.

TiVo The digital video recorder maker said it would pay a termination fee of $50.8 million to technology licensing company Xperi under certain circumstances, if their planned all-stock merger does not take place. There are other termination scenarios, according to an SEC filing, in which Xperi would pay TiVo $44 million.

KKR The private equity firm is buying digital content platform Overdrive from Rakuten for an undisclosed amount. The Japanese e-commerce company purchased Overdrive in 2015 for $410 million.

Spectrum Pharmaceuticals The drug maker said its experimental treatment for non-small cell lung cancer missed its primary goal in a mid-stage trial.

Read this article:
Stocks making the biggest moves premarket: Boeing, Amazon, PayPal & more - CNBC

Recommendation and review posted by Bethany Smith

A research team from the University of Houston has found a way to use the stem cells found in fat and guide it to become a pacemaker-like cell, according to a new study.

We are reprogramming the cardiac progenitor cell and guiding it to become a conducting cell of the heart to conduct electrical current, said study co-author Bradley McConnell, associate professor of pharmacology, in a press release

The team, publishing the study in the Journal of Molecular and Cellular Cardiology, worked on converting adipogenic mesenchymal stem cells, which reside within fat cells, into cardia progenitor cells. The ensuing cardiac progenitor cells can be programmed to aid heartbeats as a sinoatrial node (SAN), which is part of the electrical cardiac conduction system.

The researchers used what they called a standard screening strategy to test for reprogramming factors for converting human cardiac progenitor cells into pacemaker-like cells. According to their study results, the authors observed expressions of many pacemaker-specific genes, including CX30.2, KCNN4, HCN4, HCN3, HCN1, and SCN3b. The authors wrote that SHOX2, HCN2, and TBX5 (SHT5) combinations of transcription factors were much better candidate(s) in driving cardiac progenitor cells into pacemaker-like cells than other combinations and single transcription factors.

Results of this study show that the SHT5 combination of transcription factors can reprogram CPCs into Pacemaker-like cells, they wrote in their conclusion. SHT5 may be used as a potential stem cell therapy for sick sinus syndrome (SSS) and for other cardiac conduction diseases.

Original post:
The Next Generation of Biologic Pacemakers? New Discovery in Stem Cells from Fat Creates Another Alternative Treatment - DocWire News

Recommendation and review posted by Bethany Smith

University of Houston's C.T. Bauer College of Business has received its second largest donation to benefit its entrepreneurship program.

The Cyvia and Melvyn Wolff Center for Entrepreneurship, which was recently ranked the top undergraduate entrepreneurship program in the country, received the $13 million gift from its namesake foundation The Cyvia and Melvyn Wolff Family Foundation and the state of Texas is expected to match an additional $2 million, bringing the total impact to $15 million.

"Our family is deeply committed to the ideals of entrepreneurship," says Cyvia Wolff in a news release. "Our business personified everything that it means to be an entrepreneur. The skills, the thinking, the mindset are fundamental to success for business leaders today and in the future. On behalf of my late husband, we are truly honored to ensure the entrepreneurial legacy not only endures but remains accessible for students. We are truly honored to be part of this program and university."

The money will be used to create three endowments for the program. The Dave Cook Leadership Endowment, named for the center's director, Dave Cook, will be created and funded with $7 million of the donation to support leadership within the organization. For $4 million, the center will create the Wolff Legacy Endowment, which aims to increase students involved in the center, as well as the companies coming out of the program. The last $2 million will be used to create the Cyvia and Melvyn Wolff Endowed Chair(s)/Professorship(s) in Entrepreneurship. This initiative will support research and community outreach.

"We are passionate about entrepreneurship and how it can forever change students' lives," says Bauer Dean Paul A. Pavlou in the release. "We seek to further promote entrepreneurship as a university-wide, even citywide effort, by collaborating within and across the university in a multitude of areas, such as technology, health care, arts and sports."

The program was created in the mid '90s and was later renamed after Cyvia and Melvyn Wolff in 2007, and has seen great success over the past decade. In that time, Wolff students have created 1,270 businesses, with identified funding of just over $268 million. According to the release, the program has been ranked in the top two spots of the Princeton Review's top undergraduate entrepreneurship programs for nine of the past 12 years.

"Entrepreneurship is crucial for the future of our country, as well as our city and state," says UH President Renu Khator in the release. "We are proud to be at the forefront of work around entrepreneurial training and research. The uniqueness of our program has and continues to make it the model program. This extraordinary gift ensures our leadership in this space will continue and will support the creation of businesses, change communities and impact our students' lives."

At UH, 2,500 students take at least one entrepreneurship course a year, and more than 700 students complete certificate programs.

"What we are doing is transformative in the lives of students, mentors and stakeholders in a way that elevates everyone towards excellence," Cook, who was named the director of the program in 2017, says in the release. "The impact of this gift allows us to remain the leader and to move forward with confidence, purpose and permanence."

Go here to read the rest:
3 innovative research projects coming out of the University of Houston - InnovationMap

Recommendation and review posted by Bethany Smith

The report covers the forecast and analysis of the gene therapy market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the gene therapy market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the gene therapy market on a global level.

In order to give the users of this report a comprehensive view of the gene therapy market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

Download Sample of This Strategic Report:https://www.kennethresearch.com/sample-request-10170423

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the gene therapy market by segmenting the market based on the type, vector type, therapy area, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

Gene therapy is utilized for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. Moreover, gene therapy also finds lucrative applications in precision medicine. In addition to this, a rise in the occurrence of cancer is prompting the demand to treat the disease through gene therapy.

Based on the type, the market can be segregated into Germ Line Gene Therapy and Somatic Gene Therapy. In terms of vector type, the gene therapy industry can be divided into Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome. On the basis of therapy area, the market for gene therapy can be classified into Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others.

Request For Full Report:https://www.kennethresearch.com/sample-request-10170423

The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

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Gene Therapy Market 2019-2027 / Trends, Growth, Opportunities And Top Key - Market Research Sheets

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Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market,By Applications (Eurodegenerative Disorders, Autoimmune Diseases, Cancer & Tumors, Cardiovascular Diseases), By Product (Blood Pressure (BP) Monitoring Devices, Pulmonary Pressure Monitoring Devices, Intracranial Pressure (ICP) Monitoring Devices), By End User (Hospitals And Ambulatory Surgical Center), By Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2025

The Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market is expected to reach USD113.04 billion by 2025, from USD 87.59 billion in 2017 growing at a CAGR of 3.7% during the forecast period of 2018 to 2025. The upcoming market report contains data for historic years 2015 & 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025.

For In depth Information Get Sample Copy of this Report @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market

In autologous stem-cell transplantation persons own undifferentiated cells or stem cells are collected and transplanted back to the person after intensive therapy. These therapies are performed by means of hematopoietic stem cells, in some of the cases cardiac cells are used to fix the damages caused due to heart attacks. The autologous stem cell and non-stem cell based therapies are used in the treatment of various diseases such as neurodegenerative diseases, cardiovascular diseases, cancer and autoimmune diseases, infectious disease. According to World Health Organization (WHO), cardiovascular disease (CVD) causes more than half of all deaths across the European Region. The disease leads to death or frequently it is caused by AIDS, tuberculosis and malaria combined in Europe. With the prevalence of cancer and diabetes in all age groups globally the need of steam cell based therapies is increasing, according to article published by the US National Library of Medicine National Institutes of Health, it was reported that around 382 million people had diabetes in 2013 and the number is growing at alarming rate which has increased the need to improve treatment and therapies regarding the diseases.

Major Market Drivers and Restraints:

Introduction of novel autologous stem cell based therapies in regenerative medicine

Reduction in transplant associated risks

Prevalence of cancer and diabetes in all age groups

High cost of autologous cellular therapies

Lack of skilled professionals

Browse Detailed TOC, Tables, Figures, Charts and Companies @https://www.databridgemarketresearch.com/toc/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market

The global autologous stem cell and non-stem cell based therapies market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of autologous stem cell and non-stem cell based therapies market for global, Europe, North America, Asia Pacific and South America.

Some of the major players operating in the global autologous stem cell and non-stem cell based therapies market are Antria (Cro), Bioheart, Brainstorm Cell Therapeutics, Cytori, Dendreon Corporation, Fibrocell, Genesis Biopharma, Georgia Health Sciences University, Neostem, Opexa Therapeutics, Orgenesis, Regenexx, Regeneus, Tengion, Tigenix, Virxsys and many more.

Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analysed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report. To know more pleaseRequest an Analyst Callor can drop down your inquiry.

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Speak to Author of the report @ https://www.databridgemarketresearch.com/speak-to-analyst/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market

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Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Industry Trends and Forecast to 2025 - News Distribute

Recommendation and review posted by Bethany Smith

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States. Meredith Rizzo/NPR hide caption

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

When Victoria Gray was just 3 months old, her family discovered something was terribly wrong.

"My grandma was giving me a bath, and I was crying. So they took me to the emergency room to get me checked out," Gray says. "That's when they found out that I was having my first crisis."

It was Gray's first sickle cell crisis. These episodes are one of the worst things about sickle cell disease, a common and often devastating genetic blood disorder. People with the condition regularly suffer sudden, excruciating bouts of pain.

"Sometimes it feels like lightning strikes in my chest and real sharp pains all over. And it's a deep pain. I can't touch it and make it better," says Gray. "Sometimes, I will be just balled up and crying, not able to do anything for myself.

Gray is now 34 and lives in Forest, Miss. She volunteered to become the first patient in the United States with a genetic disease to get treated with the revolutionary gene-editing technique known as CRISPR.

NPR got exclusive access to chronicle Gray's journey through this medical experiment, which is being watched closely for some of the first hints that changing a person's genes with CRISPR could provide a powerful new way to treat many diseases.

"This is both enormously exciting for sickle cell disease and for all those other conditions that are next in line," says Dr. Francis Collins, director of the National Institutes of Health.

"To be able to take this new technology and give people a chance for a new life is a dream come true," Collins says. "And here we are."

Doctors removed bone marrow cells from Gray's body, edited a gene inside them with CRISPR and infused the modified cells back into her system this summer. And it appears the cells are doing what scientists hoped producing a protein that could alleviate the worst complications of sickle cell.

"We are very, very excited," says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating Gray.

Frangoul and others stress that it's far too soon to reach any definitive conclusions. Gray and many other patients will have to be treated and followed for much longer to know whether the gene-edited cells are helping.

"We have to be cautious. It's too early to celebrate," Frangoul says. "But we are very encouraged so far."

Collins agrees.

"That first person is an absolute groundbreaker. She's out on the frontier," Collins says. "Victoria deserves a lot of credit for her courage in being that person. All of us are watching with great anticipation."

This is the story of Gray's journey through the landmark attempt to use the most sophisticated genetic technology in what could be the dawn of a new era in medicine.

The study took place at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe. Meredith Rizzo/NPR hide caption

The study took place at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Life filled with pain

When I first meet her, Gray is in a bed at the TriStar Centennial Medical Center in Nashville wearing a hospital gown, big gold hoop hearings and her signature glittery eye shadow.

It's July 22, 2019, and Gray has been in the hospital for almost two months. She is still recovering from the procedure, parts of which were grueling.

Nevertheless, Gray sits up as visitors enter her room.

"Nice to meet y'all," she says.

Gray is just days away from her birthday, which she'll be celebrating far from her husband, her four children and the rest of her family. Only her father is with her in Nashville.

"It's the right time to get healed," says Gray.

Gray describes what life has been like with sickle cell, which afflicts millions of people around the world, including about 100,000 in the United States. Many are African American.

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company. Meredith Rizzo/NPR hide caption

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

"It's horrible," Gray says. "When you can't walk or, you know, lift up a spoon to feed yourself, it gets real hard."

The disease is caused by a genetic defect that turns healthy, plump and pliable red blood cells into deformed, sickle-shaped cells. The defective cells don't carry oxygen well, are hard and sticky and tend to clog up the bloodstream. The blockages and lack of oxygen wreak havoc in the body, damaging vital organs and other parts of the body.

Growing up, Victoria never got to play like other kids. Her sickle cells made her weak and prone to infections. She spent a lot of time in the hospital, recovering, getting blood transfusions all the while trying to keep up with school.

"I didn't feel normal. I couldn't do the regular things that every other kid could do. So I had to be labeled as the sick one."

Gray made it to college. But she eventually had to drop out and give up her dream of becoming a nurse. She got a job selling makeup instead but had to quit that too.

The sickle-shaped cells eventually damaged Gray's heart and other parts of her body. Gray knows that many patients with sickle cell don't live beyond middle age.

"It's horrible knowing that I could have a stroke or a heart attack at any time because I have these cells in me that are misshapen," she says. "Who wouldn't worry?"

Gray says she understands the risks involved in the treatment. "This gives me hope if it gives me nothing else," she says. Meredith Rizzo/NPR hide caption

Gray says she understands the risks involved in the treatment. "This gives me hope if it gives me nothing else," she says.

Gray married and had children. But she hasn't been able to do a lot of things most parents can, like jump on a trampoline or take her kids to sporting events. She has often had to leave them in the middle of the night to rush to the hospital for help.

"It's scary. And it affected my oldest son, you know, because he's older. So he understands. He started acting out in school. And his teacher told me, 'I believe Jemarius is acting out because he really believes you're going to die,' " Gray says, choking back tears.

Some patients can get help from drugs, and some undergo bone marrow transplants. But that procedure is risky; there's no cure for most patients.

"It was just my religion that kind of kept me going," Gray says.

An eager volunteer

Gray had been exploring the possibility of getting a bone marrow transplant when Frangoul told her about a plan to study gene editing with CRISPR to try to treat sickle cell for the first time. She jumped at the chance to volunteer.

"I was excited," Gray says.

CRISPR enables scientists to edit genes much more easily than ever before. Doctors hope it will give them a powerful new way to fight cancer, AIDS, heart disease and a long list of genetic afflictions.

"CRISPR technology has a lot of potential use in the future," Frangoul says.

To try to treat Gray's sickle cell, doctors started by removing bone marrow cells from her blood last spring.

Next, scientists used CRISPR to edit a gene in the cells to turn on the production of fetal hemoglobin. It's a protein that fetuses make in the womb to get oxygen from their mothers' blood.

"Once a baby is born, a switch will flip on. It's a gene that tells the ... bone marrow cells that produce red cells to stop making fetal hemoglobin," says Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's TriStar Centennial Medical Center.

The hope is that restoring production of fetal hemoglobin will compensate for the defective adult-hemoglobin sickle cells that patients produce.

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells don't carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain. Ed Reschke/Getty Images hide caption

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells don't carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

"We are trying to introduce enough ... fetal hemoglobin into the red blood cell to make the red blood cell go back to being happy and squishy and not sticky and hard, so it can go deliver oxygen where it's supposed to," Frangoul says.

Then on July 2, after extracting Gray's cells and sending them to a lab to get edited, Frangoul infused more than 2 billion of the edited cells into her body.

"They had the cells in a big syringe. And when it went in, my heart rate shot up real high. And it kind of made it hard to breath," Gray says. "So that was a little scary, tough moment for me."

After that moment passed, Gray says, she cried. But her tears were "happy tears," she adds.

"It was amazing and just kind of overwhelming," she says, "after all that I had went through, to finally get what I came for."

The cells won't cure sickle cell. But the hope is that the fetal hemoglobin will prevent many of the disease's complications.

"This opens the door for many patients to potentially be treated and to have their disease modified to become mild," Frangoul says.

The procedure was not easy. It involved going through many of the same steps as a standard bone marrow transplant, including getting chemotherapy to make room in the bone marrow for the gene-edited cells. The chemotherapy left Gray weak and struggling with complications, including painful mouth sores that made it difficult to eat and drink.

But Gray says the ordeal will have been worth it if the treatment works.

She calls her new gene-edited cells her "supercells."

"They gotta be super to do great things in my body and to help me be better and help me have more time with my kids and my family," she says.

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer. Meredith Rizzo/NPR hide caption

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

Concerns about risk

Other doctors and scientists are excited about the research. But they're cautious too.

"This is an exciting moment in medicine," says Laurie Zoloth, a bioethicist at the University of Chicago. "Everyone agrees with that. CRISPR promises the capacity to alter the human genome and to begin to directly address genetic diseases."

Still, Zoloth worries that the latest wave of genetic studies, including the CRISPR sickle cell study, may not have gotten enough scrutiny by objective experts.

"This a brand-new technology. It seems to work really well in animals and really well in culture dishes," she says. "It's completely unknown how it works in actual human beings. So there are a lot of unknowns. It might make you sicker."

Zoloth is especially concerned because the research involves African Americans, who have been mistreated in past medical studies.

Frangoul acknowledges that there are risks with experimental treatments. But he says the research is going very slowly with close oversight by the Food and Drug Administration and others.

"We are very cautious about how we do this trial in a very systematic way to monitor the patients carefully for any complications related to the therapy," Frangoul says.

Gray says she understands the risks of being the first patient and that the study could be just a first step that benefits only other patients, years from now. But she can't help but hope it works for her.

Dr. Haydar Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, is leading the study in Nashville. Meredith Rizzo/NPR hide caption

Dr. Haydar Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, is leading the study in Nashville.

She imagines a day when she may "wake up and not be in pain" and "be tired because I've done something not just tired for no reason." Perhaps she could play more with her kids, she says, and look forward to watching them grow up.

"That means the world to me," Gray says.

It could be many weeks or even months before the first clues emerge about whether the edited cells are safe and might be working.

"This gives me hope if it gives me nothing else," she says in July.

Heading home at last

About two months later, Gray has recovered enough to leave the hospital. She has been living in a nearby apartment for several weeks.

Enough time has passed since Gray received the cells for any concerns about immediate side effects from the cells to have largely passed. And her gene-edited cells have started working well enough for her immune system to have resumed functioning.

So Gray is packing. She will finally go home to see her children in Mississippi for the first time in months. Gray's husband is there to drive her home.

"I'm excited," she says. "I know it's going to be emotional for me. I miss the hugs and the kisses and just everything."

After living for months in Nashville, where the study was taking place, Gray packs her bags to finally go home to her kids and family in Forest, Miss. Meredith Rizzo/NPR hide caption

After living for months in Nashville, where the study was taking place, Gray packs her bags to finally go home to her kids and family in Forest, Miss.

Gray is wearing bright red glittery eye shadow. It matches her red tank top, which repeats "I am important" across the front.

She unzips a suitcase and starts pulling clothes from the closet.

"My goodness. Did I really bring all this?" she says with a laugh.

Before Gray can finish packing and depart, she has to stop by the hospital again.

"Are you excited about seeing the kids?" Frangoul says as he greets her. "Are they going to have a big welcome sign for you in Mississippi?"

Turns out that Gray has decided to make her homecoming a surprise.

"I'm just going to show up tomorrow. Like, 'Mama's home,' " she says, and laughs.

After examining Gray, Frangoul tells her that she will need to come back to Nashville once a month for checkups and blood tests to see if her genetically modified cells are producing fetal hemoglobin and giving her healthier red blood cells.

"We are very hopeful that this will work for Victoria, but we don't know that yet," Frangoul says.

Gray will also keep detailed diaries about her health, including how much pain she's experiencing, how much pain medication she needs and whether she needs any blood transfusions.

"Victoria is a pioneer in this. And we are very excited. This is a big moment for Victoria and for this pivotal trial," Frangoul says. "If we can show that this therapy is safe and effective, it can potentially change the lives of many patients."

Gray hopes so too.

See more here:
Sickle Cell Therapy With CRISPR Gene Editing Shows Promise : Shots - Health News - NPR

Recommendation and review posted by Bethany Smith

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

Picture: Supplied.

WASHINGTON, United States - In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta-thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

CURES

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumour-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face gruelling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in the hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

**BIOTERRORISM **

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his ex-communication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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2019: The year gene therapy came of age - Eyewitness News

Recommendation and review posted by Bethany Smith

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

Meredith Rizzo/NPR

When Victoria Gray was just 3 months old, her family discovered something was terribly wrong.

My grandma was giving me a bath, and I was crying. So they took me to the emergency room to get me checked out, Gray says. Thats when they found out that I was having my first crisis.

It was Grays first sickle cell crisis. These episodes are one of the worst things about sickle cell disease, a common and often devastating genetic blood disorder. People with the condition regularly suffer sudden, excruciating bouts of pain.

Sometimes it feels like lightning strikes in my chest and real sharp pains all over. And its a deep pain. I cant touch it and make it better, says Gray. Sometimes, I will be just balled up and crying, not able to do anything for myself.

Gray is now 34 and lives in Forest, Miss. She volunteered to become the first patient in the United States with a genetic disease to get treated with the revolutionary gene-editing technique known as CRISPR.

NPR got exclusive access to chronicle Grays journey through this medical experiment, which is being watched closely for some of the first hints that changing a persons genes with CRISPR could provide a powerful new way to treat many diseases.

This is both enormously exciting for sickle cell disease and for all those other conditions that are next in line, says Dr. Francis Collins, director of the National Institutes of Health.

To be able to take this new technology and give people a chance for a new life is a dream come true, Collins says. And here we are.

Doctors removed bone marrow cells from Grays body, edited a gene inside them with CRISPR and infused the modified cells back into her system this summer. And it appears the cells are doing what scientists hoped producing a protein that could alleviate the worst complications of sickle cell.

We are very, very excited, says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating Gray.

Frangoul and others stress that its far too soon to reach any definitive conclusions. Gray and many other patients will have to be treated and followed for much longer to know whether the gene-edited cells are helping.

We have to be cautious. Its too early to celebrate, Frangoul says. But we are very encouraged so far.

Collins agrees.

That first person is an absolute groundbreaker. Shes out on the frontier, Collins says. Victoria deserves a lot of credit for her courage in being that person. All of us are watching with great anticipation.

This is the story of Grays journey through the landmark attempt to use the most sophisticated genetic technology in what could be the dawn of a new era in medicine.

The study took place at HCA Healthcares Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

The study took place at HCA Healthcares Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Meredith Rizzo/NPR

Life filled with pain

When I first meet her, Gray is in a bed at the TriStar Centennial Medical Center in Nashville wearing a hospital gown, big gold hoop hearings and her signature glittery eye shadow.

Its July 22, 2019, and Gray has been in the hospital for almost two months. She is still recovering from the procedure, parts of which were grueling.

Nevertheless, Gray sits up as visitors enter her room.

Nice to meet yall, she says.

Gray is just days away from her birthday, which shell be celebrating far from her husband, her four children and the rest of her family. Only her father is with her in Nashville.

Its the right time to get healed, says Gray.

Gray describes what life has been like with sickle cell, which afflicts millions of people around the world, including about 100,000 in the United States. Many are African American.

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

Meredith Rizzo/NPR

Its horrible, Gray says. When you cant walk or, you know, lift up a spoon to feed yourself, it gets real hard.

The disease is caused by a genetic defect that turns healthy, plump and pliable red blood cells into deformed, sickle-shaped cells. The defective cells dont carry oxygen well, are hard and sticky and tend to clog up the bloodstream. The blockages and lack of oxygen wreak havoc in the body, damaging vital organs and other parts of the body.

Growing up, Victoria never got to play like other kids. Her sickle cells made her weak and prone to infections. She spent a lot of time in the hospital, recovering, getting blood transfusions all the while trying to keep up with school.

I didnt feel normal. I couldnt do the regular things that every other kid could do. So I had to be labeled as the sick one.

Gray made it to college. But she eventually had to drop out and give up her dream of becoming a nurse. She got a job selling makeup instead but had to quit that too.

The sickle-shaped cells eventually damaged Grays heart and other parts of her body. Gray knows that many patients with sickle cell dont live beyond middle age.

Its horrible knowing that I could have a stroke or a heart attack at any time because I have these cells in me that are misshapen, she says. Who wouldnt worry?

Gray says she understands the risks involved in the treatment. This gives me hope if it gives me nothing else, she says.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

Gray says she understands the risks involved in the treatment. This gives me hope if it gives me nothing else, she says.

Meredith Rizzo/NPR

Gray married and had children. But she hasnt been able to do a lot of things most parents can, like jump on a trampoline or take her kids to sporting events. She has often had to leave them in the middle of the night to rush to the hospital for help.

Its scary. And it affected my oldest son, you know, because hes older. So he understands. He started acting out in school. And his teacher told me, I believe Jemarius is acting out because he really believes youre going to die, Gray says, choking back tears.

Some patients can get help from drugs, and some undergo bone marrow transplants. But that procedure is risky; theres no cure for most patients.

It was just my religion that kind of kept me going, Gray says.

An eager volunteer

Gray had been exploring the possibility of getting a bone marrow transplant when Frangoul told her about a plan to study gene editing with CRISPR to try to treat sickle cell for the first time. She jumped at the chance to volunteer.

I was excited, Gray says.

CRISPR enables scientists to edit genes much more easily than ever before. Doctors hope it will give them a powerful new way to fight cancer, AIDS, heart disease and a long list of genetic afflictions.

CRISPR technology has a lot of potential use in the future, Frangoul says.

To try to treat Grays sickle cell, doctors started by removing bone marrow cells from her blood last spring.

Next, scientists used CRISPR to edit a gene in the cells to turn on the production of fetal hemoglobin. Its a protein that fetuses make in the womb to get oxygen from their mothers blood.

Once a baby is born, a switch will flip on. Its a gene that tells the bone marrow cells that produce red cells to stop making fetal hemoglobin, says Frangoul, medical director of pediatric hematology/oncology at HCA Healthcares TriStar Centennial Medical Center.

The hope is that restoring production of fetal hemoglobin will compensate for the defective adult-hemoglobin sickle cells that patients produce.

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells dont carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

Ed Reschke/Getty Images

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Ed Reschke/Getty Images

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells dont carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

Ed Reschke/Getty Images

We are trying to introduce enough fetal hemoglobin into the red blood cell to make the red blood cell go back to being happy and squishy and not sticky and hard, so it can go deliver oxygen where its supposed to, Frangoul says.

Then on July 2, after extracting Grays cells and sending them to a lab to get edited, Frangoul infused more than 2 billion of the edited cells into her body.

They had the cells in a big syringe. And when it went in, my heart rate shot up real high. And it kind of made it hard to breath, Gray says. So that was a little scary, tough moment for me.

After that moment passed, Gray says, she cried. But her tears were happy tears, she adds.

It was amazing and just kind of overwhelming, she says, after all that I had went through, to finally get what I came for.

The cells wont cure sickle cell. But the hope is that the fetal hemoglobin will prevent many of the diseases complications.

This opens the door for many patients to potentially be treated and to have their disease modified to become mild, Frangoul says.

The procedure was not easy. It involved going through many of the same steps as a standard bone marrow transplant, including getting chemotherapy to make room in the bone marrow for the gene-edited cells. The chemotherapy left Gray weak and struggling with complications, including painful mouth sores that made it difficult to eat and drink.

But Gray says the ordeal will have been worth it if the treatment works.

She calls her new gene-edited cells her supercells.

They gotta be super to do great things in my body and to help me be better and help me have more time with my kids and my family, she says.

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

Meredith Rizzo/NPR

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Meredith Rizzo/NPR

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

Meredith Rizzo/NPR

Concerns about risk

Other doctors and scientists are excited about the research. But theyre cautious too.

This is an exciting moment in medicine, says Laurie Zoloth, a bioethicist at the University of Chicago. Everyone agrees with that. CRISPR promises the capacity to alter the human genome and to begin to directly address genetic diseases.

Still, Zoloth worries that the latest wave of genetic studies, including the CRISPR sickle cell study, may not have gotten enough scrutiny by objective experts.

Read more:
Sickle Cell Therapy With CRISPR Gene Editing Shows Promise : Shots - Invest Records

Recommendation and review posted by Bethany Smith

Protein Associated with Leukemia May Lead to Targeted Therapy for Currently Incurable Acute Lymphoblastic Leukemia

ALL is a form of blood cancer that primarily affects children and young people and causes large quantities of malignant progenitor cells to build in a patients blood instead of healthy white blood cells. This is often caused by 2 chromosomes fusing together to create new abnormal genes that disrupt the system controlling normal blood development. Because of this process, certain types of leukemia are extremely resistant and unable to be cured with intensive chemotherapy or stem cell transplantation.

Researchers analyzed a protein called TCF3-HLF, which is typically associated with this type of leukemia and does not occur naturally. It is produced through the fusion of 2 chromosomes and contains elements of transcription factors, which activate the transcription of certain genes.

The analysis revealed that TCF3-HLF activates a whole range of genes, but it does so in the wrong contextat the wrong point in the blood development process. The formation of malignant white blood cells is then triggered, causing leukemia.

The study authors also discovered that the abnormal protein does not act alone, but instead gathers more than 100 other proteins around it, which helps to activate the genes. The researchers investigated the function of the individual proteins in the genetic machinery and used it to identify key elements that could be targeted through therapy.

Using the CRISPR/Cas9 method, researchers detached the specific parts they had identified from the machinery and found 11 critical factors that are crucial to the build-up of malignant abnormal blood cells in leukemia.

One of the essential components now identified is the protein EP300, a cofactor that boosts gene activation. The researchers used a new kind of substance called A-485, known to bind to EP300 and inhibit its activity. When A-485 was administered to human leukemia cells, the malignant cells died off.

The study authors noted that it is possible to stop the fundamental driving force behind the leukemia directly and thus develop a targeted type of therapy. Given that other forms of leukemia are caused by similar mechanisms, it may also be possible to identify a common denominator for developing new drugs to combat cancer.

REFERENCE

New approach to treating incurable leukemia in children discovered [press release]. University of Zurich. BioPortfolio website. Published November 24, 2019. https://www.bioportfolio.com/news/article/4148041/New-approach-to-treating-incurable-leukemia-in-children-discovered.html. Accessed December 4, 2019.

More:
Protein Associated with Leukemia May Lead to Targeted Therapy for Currently Incurable Acute Lymphoblastic Leukemia - Pharmacy Times

Recommendation and review posted by Bethany Smith

Throughout the year we have published the views of company executives, government regulators, industry analysts and scientists on a variety of topics and, in our popular annual feature, we include a selection of these that paints a picture of the significant events that shaped 2019. The major talking point was on the capital markets front where investors turned their backs on the biopharmaceutical sector for most of the year returned big time in the final quarter. M&As helped sparked life, with blockbuster deals taking place throughout the year, particularly in the red hot cell and gene therapy space, with an exclamation point added this week as Roche Holding AG followed up the completion of its $4.8 billion acquisition of Spark Therapeutics Inc. with the announcement of a blockbuster deal with Sarepta Therapeutics Inc., underscoring big pharmas appetite for gene therapy technologies.

Our favorite quote from the year

The Wright brothers showed that you could fly a plane, but it wasnt very far, and it wasnt very safe. Thats where cell therapy is now.At a scientific session at the American Society of Hematology meeting on gene editing, Wendell Lim, professor and chair of cellular and molecular pharmacology at the University of California, San Francisco, and the co-founder of Cell Design Labs, discussed his laboratorys work that uses gene editing to improve cell therapies, specifically CAR T cells

Capital markets

The biopharmaceutical sector headed into 2019 on a negative note, with most of the damage inflicted in December 2018 a month that will go down as one of the worst for equities, with the Dow Jones Industrial Average stumbling by almost 9% and the Nasdaq Composite performing just as poorly, dropping 9.5%.

Summing up the situation, the Cowen and Co. biotechnology analyst team wrote in its monthly biotech thermometer report to clients: "Investors are shell-shocked by the depreciation that was so quick, so dramatic, and so close to the end of the year. Sentiment is worse than we can remember over the last five years, and in fact many investors have developed a bear market mindset."

Fast forward 12 months and the curtain is now closing on a year where, for many months, the biopharmaceutical industry languished and company executives undoubtedly were wondering, during that difficult period, whether there would be a reversal of fortunes.

The answer came in the fourth quarter. To the surprise of industry pundits, public biopharmaceutical companies turned the corner and are on a major upswing, with the sector outperforming the general market in final frame with a 23% upswing year-to-date.

Reflecting on the change, Cowen and Co. analysts, writing in their monthly Biotech Thermometer noted, "Those who stuck with the sector through the famine of Q3 have feasted on a cornucopia of returns during the first two months of Q4."

Bear to bull

Most analysts speculated at the beginning of the year that the recipe to create an upswing in the sectors fortunes would be tied to an increase in M&A activity. SVB Leerink analyst Joseph Schwartz was certainly prescient in a note to investors earlier this year pointing to gene therapy companies as being prime targets. "With increasing pricing pressure and the emphasis on 'value' from payers, we believe curative gene therapy programs represent ideal acquisition targets for large pharma or biotech companies looking to diversify their product portfolios and reduce their reliance on price hikes."

RBC Capital Markets analyst Brian Abrahams, writing in the 2019 RBC Biotech Outlook Report, struck a similar note: "For 2019, we are more neutral vs. 2018, as growth and competitive headwinds, alongside more tangible manifestations of drug cost containment, potentially mitigate increasingly attractive valuations among larger-caps and what we expect will be increasing M&A activity and enthusiasm backstopping the smids."

Industry reels from FDA commissioner resignation

The announcement from Scott Gottlieb that he was resigning as FDA commissioner to return to the American Enterprise Institute in April took the industry by surprise, and the news weighed heavily on biopharmaceutical equities, with the BioWorld Biopharmaceutical index falling almost 4% on the news.

"Scott has helped us to lower drug prices, get a record number of generic drugs approved and onto the market, and so many other things. He and his talents will be greatly missed."President Donald Trump's tweet in response to Scott Gottlieb's announcement that he was resigning as FDA commissioner

The industry had to wait until December for a replacement when the U.S. Senate voted 72-18 to confirm Stephen Hahn as the commissioner of the FDA, providing the agency with another commissioner with a deep background in oncology. Secretary of Health and Human Services Alex Azar thanked the Senate for prioritizing the candidates nomination, adding that the development will be a major boost to the already rapid pace of the Presidents aggressive public health agenda.

Setting the agenda

Looking at the BioWorld Biopharmaceutical index it reveals that it did attain an almost 12% increase in value by February. Catalyzing the jump was the positive vibe that emanated from the January J.P. Morgan 37th annual health care conference, which every year sets the industrys agenda. From the market reaction to the upcoming plans laid bare by presentations from many of the leading biotechnology and big pharma companies in attendance, it appeared that the sector came out ahead.

Business development

"Once again we're seeing a lot of billion-dollar deals and they tend to be preclinical-stage deals that have multiple assets. It's a trend that started in 2016."David Thomas, vice president of industry research and analysis for the Biotechnology Innovation Organization, on its 2019 Emerging Therapeutic Company Trend Report, which showed that the number of global R&D-stage licensing deals surged by 107% in 2018 over the prior year

"The shift away from larger market drugs to more niche indications reflects the change in R&D focus of biopharma companies."Roger Humphrey, executive managing director and leader of JLL's Life Sciences group

Despite a decade of effort to streamline discovery and development and increase productivity, the projected return on investment in R&D at the worlds leading pharmaceutical companies has hit an all-time low, according to the 10th annual analysis by management consultancy Deloitte.

No other industry would operate on such low R&D returns.Substantive change is needed to shorten R&D cycle times, according to Karen Taylor, director of Deloittes health practice

Cash flows

Despite a rough ride on the capital markets for much of the year, particularly in the second and third quarters, the biopharmaceutical sector had no difficulty in attracting capital.

[This is] "the most exciting time to be investing in life sciences in a generation."Otello Stampacchia, managing director, Omega Funds, which raised $438 million to deploy into new and existing life sciences companies

"Capital is so available, it strikes me insane that almost half the industry needs to raise money this year."Dennis Purcell, founder and senior advisor at Aisling Capital LLC, during a session at the BIO CEO & Investor Conference

"It's very much a chess game in the current market; you have to think multiple steps out."Nol Brown, managing director, health care investment banking, at Cantor Fitzgerald, on planning whether to IPO during a session at the BIO CEO & Investor Conference

"It's a great time to be an entrepreneur. There are so many sources of capital. And there's a lot of it."Chau Quang Khuong, private equity partner at Orbimed Advisors, speaking at the Biocom Global Life Science Partnering Conference on the advantages of starting a company now

M&As

Bristol-Myers Squibb Co.'s announced $74 billion bid for Celgene Corp., the largest M&A in biopharma history, made for what J.P. Morgan analyst Cory Kasimov called "one of the most significant pre-conference announcements ever."

Although the deal did not spark a wave of M&A activity at the time, transactions remained sporadic for the rest of the year. According to the 2020 RBC Biotech Outlook report, Despite the unevenness throughout the course of 2019, the forces we believed would stimulate a meaningful pickup in acquisitions coalesced to generate new highs in biopharma acquisitions, with a record number of announced $1B+ public company deals.

"Two turkeys don't make an eagle. Both businesses have seen their share of challenges, with ABBV's Humira seeing competitive pressures both in the U.S. and EU and Allergan's rather well-documented business challenges. However, financial potential does have our attention, with the deal being immediately accretive to earnings."Christopher J. Raymond, Piper Jaffray analyst, in a research note on the first reaction to the announcement that Abbvie Inc. was proposing to acquire Allergan plc in a cash and stock deal worth about $63 billion

Gene therapy is hot

Barely a day went by this year without news relating to cell and gene therapy companies being announced. Given the ongoing developments, it was not surprising that the sector continued to attract financing and business development, with biopharma companies ensuring that they secured a position in the space for themselves through partnering and acquisitions.

"Gene therapy gets around many of the drug delivery challenges that have plagued other treatment modalities like small molecules. In part, that is because it's a one-time approach and also it really enables you to target the tissue bed of interest, in some cases direct to the CNS."Shankar Ramaswamy, chief business officer, Axovant Gene Therapies Ltd.

"Gene therapies are among the most exciting medical advancements of our time. However, they also pose a great technical challenge, since the AAV particles designed to deliver genes to tissues are subject to immediate and overwhelming immune attack, specifically by complement C3, which may result in significant safety and efficacy constraints. We believe that the targeted control of C3 may prevent the C3-mediated attack on AAV particles."Cedric Francois, CEO and co-founder of Apellis Pharmaceuticals Inc.

"[Many gene therapies] are introduced with much higher levels of uncertainty about their long-term safety and effectiveness than standard treatments, and patients and insurers are being asked to pay extremely high prices up front for the promise of long-term benefit."Steven Pearson, president, The Institute for Clinical and Economic Review, which is proposing adaptations to the framework it uses in assessing the value of potential single or short-term transformative therapies (SSTs). The proposals are intended to help decision-makers conduct a more reliable and transparent evaluation of the uncertainty, value and value-based pricing of SSTs

Some of the gene therapeutic products and candidates have truncated the size of the genes and have done some pretty clever maneuvers to try and make the gene inserts smaller in order to be incorporated by AAV.Daniel Dornbusch, head of business development at Dnarx Inc., explained, while noting that the full length might be better and there are many genes that cant be truncated

"Gene editing is a rapidly developing technology that represents one of the most exciting developments in medicine. These techniques will be integral to the next generation of advanced therapeutics and we welcome their potential to provide important, and potentially life-saving, treatments for patients. As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles."Janet Lambert, CEO, The Alliance for Regenerative Medicine commenting on the release of the Alliance's Therapeutic Developers' Statement of Principles, setting forth a bioethical framework for the use of gene editing in therapeutic applications

Artificial intelligence and machine learning

There is no doubt that the next wave of drug discovery will be enabled by powerful supercomputers dining on complex algorithms to uncover potential new scientific approaches for the development of innovative therapeutics, and industry executives certainly provided BioWorld with a wide range of opinions on its value.

"The idea behind machine learning is that it's a method that should generalize and be able to make decisions or make estimates or classifications based on data it hasn't seen before."John Irvine, chief scientist for data analytics at Draper Laboratories, Siemens Healthineers. The firm is working with Brisbane's Translational Research Institute and using artificial intelligence to diagnose medical conditions ranging from breast cancer to post-traumatic stress disorder

"I think the drive in this field is that we saw the success of Google and Facebook to basically take what seemed like white noise out in the ether and squeeze out this gold; it's like the new gold rush. And in Alzheimer's we've been desperate for success, and it seems like we have this messy, noisy data it almost feels like static sometimes and we want a machine learning algorithm to come in and wrangle that up and squeeze gold out of what looks like noise. Unfortunately, I don't think it's going to be that easy."Newman Knowlton, a statistician at Millcreek, Utah-based statistical consulting firm Pentara Corp.

"When I have casual conversation about this, I think of the first cell phone and what iPhone is like today. At first, we wanted only a few functions, but we eventually realized we needed more functionality to benefit from the technology. It's the same in biomedicine."Ken Drazan, CEO, Arsenal Biosciences Inc.

"Data-driven medical innovation is growing exponentially, and our partnership with Google will help us lead the digital transformation in health care. It will empower us to solve some of the most complex medical problems; better anticipate the needs of people we serve; and meet them when, where and how they need us. We will share our knowledge and expertise globally while caring for people locally and always do it with a human touch."Gianrico Farrugia, president and CEO of Mayo Clinic

"Across the globe, clinicians spend more time doing data entry in patients' records than they do treating patients, and I think AI has a real opportunity to change that."Chris Nave, Managing Director, Brandon Capital

Editors note: See Fridays issue for Part 2.

More:
Perspectives on the year: The industry has its say on the markets, M&A and gene therapy - BioWorld Online

Recommendation and review posted by Bethany Smith

Abstract / Synopsis:

RGX-314 gene therapy, a treatment for wet age-related macular degeneration delivered subretinally, showed dose-dependent sustained/improved vision and a good safety profile.

This article was reviewed by Jeffrey S. Heier, MD

RGX-314 (REGENXBIO) gene therapy, delivered subretinally to patients with neovascular age-related macular degeneration (AMD), who required a large number of prior injections of anti-vascular endothelial growth factor (VEGF) annually, provided a marked decrease in the number of injections needed and retained or improved the vision and anatomy in this challenging patient population.

RGX-314 uses a proprietary gene delivery platform that is hypothesized to deliver longer and higher protein expression with a lower immune response that earlier generation adeno associated viruses (AAVs) used for gene therapy.

The AAV8 vector is encoded to deliver a gene that leads to anti-VEGF antibody fragment protein production within the retina, said Jeffrey S. Heier, MD, co-president and director of Retinal Research, Ophthalmic Consultants of Boston.

Related: Anti-VEGF targeted as potential nAMD treatment

All 42 patients included in this study had undergone previous treatment for neovascular AMD and had a high need for anti-VEGF therapy, i.e., patients received more than 30 injections on average prior to coming in and an average of 9.6 annualized injections in the year prior to RGX-314.

In addition, the included patients had to have shown an anatomic response to anti-VEGF therapy during screening. After RGX-314 was delivered to the subretinal space, the patients were evaluated monthly to assess safety and the need for additional anti-VEGF therapy, Dr. Heier noted.

Re-treatment with an anti-VEGF drug could be provided beginning at four weeks after RGX-314 was delivered and then as needed every four weeks thereafter based on physician discretion, Dr. Heier explained. The criteria for retreatment was per investigator discretion which included choroidal neovascularization-related increased, new, or persistent fluid; vision loss of five or more letters as a result of fluid, or a new ocular hemorrhage.

Related: The ABCs of VEGF treatment for diabetic macular edema

The resultsFollowing delivery of RGX-314, Dr. Heier reported that the drug was well tolerated. No serious adverse events were reported; most adverse events were classified as mild, i.e., grade 1 in 79% of cases. In addition, no clinically determined immune responses occurred.

Two deaths that occurred were not related to RGX-314. Two serious adverse effects that were related to the delivery procedure were reported, specifically, a peripheral retinal detachment that was repaired successfully and a case of endophthalmitis that occurred after collection of an aqueous sample.

We observed a dose-dependent increase in RGX-314 protein across the five dose cohorts, he reported. The protein levels were measured in aqueous samples collected one month after delivery of RGX-314.

Cohort 3, which included six eyes treated with a dose of 6 x 1010 genome copies (GC)/eye, did well.

Cohort 3 had a gain in vision of +9 letters and stable anatomy of -40 m over 18 months despite relatively few injections, Dr. Heier reported.

Three of the six patients in this cohort were injection-free at 18 months. They had a mean increase of +11 letters of vision at 18 months and a concomitant mean decrease in the central retinal thickness of -21 m. The RGX-314 protein levels were sustained over one year.

Related: Decreasing burden of nAMD therapy

Originally posted here:
Study targets subretinal option for AMD treatment - Ophthalmology Times

Recommendation and review posted by Bethany Smith

Beyond the bright colors and hallucinogenic imagery of psychedelic artthe visuals of Ram Dass's 1971 book Be Here Now has never left public consciousness there has long been a crusade to clinically research substances such as LSD, psilocybin, MDMA, DMT, and ibogaine. We've been informed, again and again and again, about the various ways that current pharmaceutical treatments in our for-profit mental health system is not only not working, but doing more damage than healing. Discussion over health care inevitably defaults to mechanisms for paying for a broken model, rarely touching upon the root causes of why so many people are depressed, sick, anxious, and suicidal in the first place.

We. Need. Better. Solutions.

In regard to psychedelics, an entire herd of elephants remain locked in a room. Thanks to the questionable (and admittedly racist) wars launched by the Nixon and Reagan administrations (first dreamed up during the Anslinger crusades), we've been denied access to these potentially therapeutic substances. Fortunately, a renaissance is occurring in psychedelics research, with ketamine being the first to be legally prescribed psychedelic for treatment-resistant depression and both psilocybin and MDMA being fast-tracked by the FDA after being labelled breakthrough therapies.

One challenge psychedelics advocates will have to face is how these drugs are treated moving through the current medical model. Regardless of personal feelings on the subject, these substances have to contend with a system that requires expensive clinical trials and will be sold in a capitalist marketplace. There will inevitably be patent issues and territorial fights. Unlike cannabis, which is a relatively mild substance with few documented consequences, psychedelics need to be rigorously evaluated and tested. While some label everyone working in medicine as minions of Big Pharma, we need to separate researchers and scientists from the shady dealings of shareholders and profiteers.

Michael Ehlers is an industry figure that has long taken an interest in psychedelics, predominantly from an outsider perspective. Now the former executive vice president for research and development at Biogen is accepting an advisory role with Field Trip Health, the psychedelics-focused organization that recently opened the world's first psilocybin research center. (You can listen to my talk with Field Trip co-founder, Ronan Levy, here.)

I chatted with Ehlers, he is also the former chief scientific officer for neuroscience at Pfizer, about his interest in psychedelics, their potential efficacy, their historical usage in ritual, and how the current model will deal with their vetting and potential applications. With every question, he was informed and honest, offering what he knows and being truthful about what he does not. There is a lot of work ahead in pharmaceuticals, yet it is undeniable the mental health industry needs a reboot, in the same way psychedelics are said to reboot the neural circuitry of the brain, making this class of substances an ideal medicine for study.

Part of my conversation with Ehlers is below; you can read the full transcript here.

Photo courtesy of Michael Ehlers

Derek: You have an accomplished career in the pharmaceutical industry. Now you've taken on an advisory role with a company specializing in psychedelics. I would love to know when you first became interested in psychedelics as a potential therapeutic tool.

Michael: I've followed this area for quite some time. I've been intensely involved in different aspects of drug discovery and development, particularly, although not exclusively, within CNS or neuroscience drug discovery, including neuropsychiatric disease. I've followed more peripherally some of the efforts both in standard pharmacology and then some of the emerging work, whether it was more acute, high-dose psychedelics or microdosing psychedelics in neuropsychiatric disease.

At the same time, I was following a lot of the work on some of the core receptor biology and neurobiology, which was really advancing in systems neuroscience. Following this field and some of the early indications of potential clinical efficacy were some of the things that really got me quite excited. I was particularly close with aspects of what's been done over the past 10 years with ketamine, which is a very different agent but also in the class, initially leading from small trials on ketamine for acute, anti-depressive actions, now to Janssen and J&J using a variation of this, esketamine, to get full-on FDA approval for the first new mechanism in depression in 20 years. The combination of these things indicated to me that there could be a new paradigm change or highly-active psychopharmacology to potentially treat some of these otherwise fairly intractable types of neuropsychiatric disorders.

There are some other things that were also on the horizon. The history of CNS drug development, particularly in neuropsychiatric disease, has been one where the empirical observations in human patients have really guided efficacious therapeutics by and large. Even though I know we like to talk a lot about rational drug discovery and development, at least in the field of neuropsychiatry, because there's still so much that is not known that we've had to rely a lot more on empirical observations in humans.

There's probably no more profound CNS pharmacology out there than that with psychedelics like psilocybin or LSD or ketamine. I've actually long thought it was just a matter of figuring out what a treatment paradigm could look likehow maybe when you dose it could you alter aspects of its dose exposure and distribution and then in what exact disease or syndrome.

Derek: You have a history of working with rare diseases. Field Trip is going to tackle a wide range of studies, but the ones that are really on everyone's mind (in terms of what psychedelics could potentially help) ranges from PTSD to treatment-resistant depression and anxiety. These are much more common diseases. Do you have any background in those diseases and, in the advisory role, what will you be doing for them?

Michael: I've got a lot of background in that. I worked for nine years in large biopharma, six years at Pfizer. I started in neuroscience and pain, but ultimately ran several divisions of Pfizer R & D, that did include rare disease, but included a bunch of other things. Then I ran R & D advising for three-and-a-half years. I've done clinical trials in depression, schizophrenia, PTSD, generalized anxiety disorder, Alzheimer's disease, and Parkinson's disease. I've done both rare diseases and a lot of common disorders: hemophilia, genetic disease, and some of the rare diseases as well. I've done stroke trials. I've had experience across a range.

One thing I like is about what Field Trip is doing and the prospect of these diseases is that they're incredibly common. Roughly 25 percent of people will have some experience with major depression in their lives. One percent of the world has schizophrenia. These are serious and significant disorders. I really love the fact that this fieldand Field Trip is really part of that in a leadership roleis looking to take some of these on.

Although the lore has been that there hasn't been that much innovation, I actually think that's not true. I think we're just at the beginning of a whole new era of advances in neuropsychiatric disease. I can point to several things that indicate that. I have a feeling that if we really understand that the best way to dose and conduct trials with psychedelics like psilocybin and be able to segment patients who are the most likely to benefit, this can become quite important.

Derek: You mentioned that pharma companies stepping away from neuropsychiatric disease. There is obviously a problem with SSRIs over the long-term. Efficacy rates tend to be high in the short-term, but over the long-term prove problematic. When you're stepping into substances that potentially could help treatment-resistant mental health diseases in one dose (or just a couple of doses), how do you think that companies are going to be able to monetize this, especially given the incredible amounts of money that have to go into R & D and clinical trials?

Michael: It's a very good question. I think we haven't solved that problem yet. There are a lot of open questions. Will some of these therapies really be single dose or short regiments and you're done? Will it have to be that there's some degree of maintenance where there's some regularity in the need for therapy? Will it really be like antibiotics or gene therapy? We don't know.

A lot of these neuropsychiatric diseases, although they're complex, have genetic features that are polygenetic but they're related. Whether you're talking about, schizophrenia, autism, bipolar disorder, ADHD, there's a complex genetic architecture that has shared features across all of those. The risk of relapse and occurrences will be there in a given population. I tend to think the likelihood of things like ketamine or psychedelic treatments for depression will be one of periodic needs.

The question you raised is an excellent one, which is what ultimately is the commercial model for that? Certainly, the hope is that it doesn't go down the road of antibiotics for which the commercial incentivization for real R & D and drug development has been catastrophic. I don't see that in this space. I just don't think it's going to be quite as simple as "one and done." The prevalence alone will be a strong incentive for investment when there's real efficacy potential.

Derek: Please correct me if I'm wrong; I'm fascinated by neuroscience, but not having an academic background my knowledge is limited. That's why I love talking to people about this. From my understanding, SSRIs work in a much different manner in terms of the serotonin release then psychedelics. Do you see any potential benefits or dangers in the ways that psychedelics deal with the serotonergic system?

Michael: It is quite different. From a simple pharmacology point of view, SSRIs are, as their name indicates, selective serotonin reuptake inhibitors: they block serotonin transporters that would normally release serotonin back up into nerve cells so that it increases serotonergic tone. Once released, it stays released in the extracellular space for longer, acting on all the different receptors in the places that it does.

The psychedelics typically act directly on serotonin receptors within serotonin transporters, but their action at different receptors has different potency. It's not a clean pharmacology. People will talk about 5-HT-2A receptors and they're clearly important, and there's been a lot of study on that, but we also know that if you just give a pure 5-HT-2A receptor an agonist you do not reproduce the effects of psilocybin or LSD.

The pharmacology is complex; it's clearly different than SSRIs. Obviously, the behavioral and therapeutic groups are very different. It just highlights that we really need to understand it better. It's going to reveal I think very important things about psychiatric disease and fundamental neuroscience.

A shaman gathers the raw materials to make ayahuasca in the jungle outside of Iquitos.

Photo by Andrew Lichtenstein/Corbis via Getty Images

Derek: One of the criticisms of the way that the industry is right now is that, why would a doctor spend an hour talking to a patient when you can see six patients in an hour and write a script? Efficacy rates are different for different people, dealing with the microbiome, for example, and the way that their gut processes drugs. It's a very complex issue. One thing I believe is going to be important is that psychotherapy is going to be tethered with psychedelics, especially if people have never done them before. Will that coupling provide a sustainable model?

Michael: Here's an aspect of what's important to understand: the field has understandably taken a cautious approach, which I think is warranted in this whole guided therapy concept and that will probably be required for certain dosing regimens. I would personally like to see this converted into what is a very standard thing in a lot of drug administration in practice or trials, which is more about medical monitoring. Change it from the notion of it's guided therapy to monitoring like you would for a lot of things. People go to IV infusion centers to get their IV drug. It's different, but there's nothing that unusual about the notion of having a monitored pharmaceutical or pharmacological drug intervention even in standard practice. This will likely be part of that.

If you're a neurologist treating MS and you've got MS patients on Alemtuzumab or Natalizumab as your IV drugs. They come in, you've got your IV clinic. They come in regularly, every month or every quarter depending on the drug, and they get their IV infusion. They get monitored while it happens because they can have an immune response. I see a future for some of these psychoactive therapeutics where you have something similar.

Now the question will be to what extent does the guided as opposed to monitoring aspect of that influence the degree of efficacy? That's something which really would need to be studied. To the extent it really requires some special type of guided activity that will be a little bit more of a limitation. To the extent that it can be ultimately the design in a more monitoring approach with education, the more widespread this can become.

Does that analogy make sense to you? There's a lot of precedence for this in other areas. The way this has gotten utilized now is still a remnant of causing people to have profound hallucinations and behavioral stuff and paranoia. Some people get afraid of that, so we need to have some monitoring.

We need to understand doses. We need to know the extent to which those experiences are part and parcel to a therapeutic response or not associated with a therapeutic response.

Derek: How much do you think anecdote is going to matter? One main issue I have with the whole cannabis legalization process is the extraction of CBD being sold for every possible ailment out there when the actual evidence is almost nothing at this point, besides epilepsy. At the same time, dealing with mental health disorders, how much are we going to rely on anecdote? If people think they're getting better, there's placebo, and it actually helps them get better.

Michael: I hope we moved beyond anecdotes, and I think that you're right about CBD, but it's interesting the way you put that because of the fact that rigorous trials have been done in rare epilepsies, like Dravet and Lennox-Gastaut syndrome, nobody disputes that. Patients in need can get insurance companies or health systems in other countries to reimburse for that. That's what I mean by saying real location impact is going to require that component of it too. You'd like to be able to generate the evidence because nothing comes without safety concerns. The nice thing about putting this all through the lens of drug discovery and development is that it allows the communityand here I mean the medical community, policymakers, others to have a much clearer view of the benefit-risk, and where the benefit-risk is positive, in which case that's usually a required element for real access for patients.

Of course, you could argue and say, "well, if it's just out there, people can try it, we'll see and that's fine," but this doesn't allow us from a clinical scientific vantage point to really know when and where we are going to provide benefits. That's what we really need to work toward. There's enough anecdotal evidence out there to justify rigorous evaluation.

--

Stay in touch with Derek on Twitter and Facebook. His next book is Hero's Dose: The Case For Psychedelics in Ritual and Therapy.

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Cell and Gene Therapy Market Size Will Escalate Rapidly in the Near Future - Market Research Sheets

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This report highlights market dynamics involving factors driving the Personalized Gene Therapy Treatment industry scenario, as well as market growth opportunities in the coming years. Market segmentation analysis was performed through qualitative and quantitative research, demonstrating the impact of economic and non-economic aspects.

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Personalized Gene Therapy Treatment Market to Exhibit Impressive Growth by 2030 Bulletin Line - Bulletin Line

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Sickle cell disease is a genetic condition in which red blood cells, which should be circular, adopt a crescent shape and are sticky and rigidALAMY

The first patients to receive gene-editing treatments for inherited blood diseases will enter the new year free of agonising symptoms.

The experiments suggest that altering DNA could treat sickle cell disease (SCD) and beta thalassemia, conditions both caused by faulty genes that hamper the bloods ability to carry oxygen.

The companies behind the trials said that a patient in the US with SCD had been well since July. A thalassemia patient in Germany had been free of symptoms for nine months. Previously she had 16 blood transfusions a year.

British patients could be offered similar experimental therapies next year. The treatment for both conditions involved a high-precision gene-editing tool called Crispr-Cas9. It was used to alter the DNA of some of the cells of Victoria

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Of all blood cancers, leukaemia and lymphoma are among the most curable.

However, many people, including doctors, still believe the disease leads to immediate death.

This is no longer true today as they are not fatal.

With optimal treatment, the majority of patients go into remission and are considered cured.

These two cancers have been more extensively studied than other forms of cancer, due to the ease in obtaining samples from blood, bone marrow or lymph nodes, spurring the advent of novel targeted therapies for a cure, says consultant haematologist Dr Ng Soo Chin.

Most blood cancers start in the bone marrow, where blood is produced.

Bone marrow contains stem cells, which mature and develop into red blood cells, white blood cells or platelets.

In most blood cancers, normal cell development is interrupted by the uncontrolled growth of an abnormal type of a particular blood cell.

These abnormal blood cells, which are cancerous, prevent your blood from performing many of its functions, like fighting off infections or preventing serious bleeding.

Leukaemia or white blood is classified into acute and chronic disease, which is then divided further into subtypes: acute lymphocytic leukaemia, acute myeloid leukaemia, chronic lymphocytic leukaemia (CLL) and chronic myeloid leukaemia (CML).

The presentation between acute and chronic leukaemia differs.

The acute person will tell you he was well a week ago and is now down with symptoms such as lethargy, anaemia and recurrent infection.

Suddenly, he may look pale, so we check his blood count for any abnormalities. A bone marrow exam will further confirm whether it is acute.

With chronic leukaemia, the patient can be unwell for a couple of months.

We are increasingly picking up cases early because of blood test availability.

The survival rate has improved tremendously for acute leukaemia, with more than 50% fully cured because bone marrow transplants are easily available in the country.

For CLL and CML, 95% of patients are alive at the 10-year mark, says Dr Ng.

Generally, chronic leukaemia patients belong to the older age group (50 years and above), but acute leukaemia can occur in all ages.

Leukaemia symptoms are often vague and not specific, so its easy to overlook them as they may resemble symptoms of the flu and other common illnesses.

In fact, chronic leukaemia may initially produce no symptoms and can go unnoticed or undiagnosed for years.

Lymphomas, a type of blood cancer that begins in a subset of white blood cells called lymphocytes, can be classified into Hodgkins and non-Hodgkins.

The main difference between Hodgkins and non-Hodgkins lymphoma is the specific lymphocyte each involves.

Lymphocytes are an integral part of your immune system, which protects you from germs.

Five-year survival rates are high with Hodgkins lymphoma at 86% and non-Hodgkins lymphoma at 70%.

You can beat the disease even if it is detected at a late stage.

Multiple myeloma, which is the third kind of blood cancer, forms in a type of white blood cell called a plasma cell.

Patients often complain of bone pain, and unfortunately, this type of cancer has no cure.

Blood cancers typically involve abnormal white blood cells and can affect paople of all ages, depending on the type of cancer. 123rf.com

Fear of treatment

Chemotherapy is a much dreaded word among cancer patients.

But with advances in medicine, newer chemotherapy-free treatments are now available.

Dr Ng says, Traditionally, cancer is treated via surgery or radiation the layman says we fry and poison them, which is not far from the truth!

Radiation means burning the cancerous area, but a lot of times, the cancer can also be present elsewhere, so there is limitation to this treatment.

With chemotherapy, we use cytotoxic (cell-killing) drugs they go in and knock off both cancer and normal cells.

The short-term effects include vomiting, hair loss, appetite loss and weight loss.

But as doctors, we are looking at a different perspective. We are more worried about white cells dropping (neutropenia) because the patient can pick up an infection that can potentially kill him.

Neutropenia is a condition that results when the body does not have enough neutrophils, a type of white blood cell that is an essential first line of defence against infections.

Thats one risk of chemotherapy, although we can now improve neutropenia by giving a growth factor injection.

But for certain cancers, we need to step up the drugs.

He adds: We are scared of neutropenia, but patients are more concerned about bodily changes.

The older ones get upset over losing hair because they cannot take it when others ask them what has happened to their hair.

Young people are not as concerned with hair loss because it can be trendy.

We understand that chemotherapy is less than pleasant and strong doses can impair fertility in young patients, especially women.

Despite current technology, only one-third of patients are successful in freezing their eggs.

What he is concerned about is that chemotherapy can actually increase the patients risk of getting another cancer, especially blood cancer.

It can happen the day after! says Dr Ng.

Most experts believe chemotherapy damages stem cells, so if youre unlucky, you might get acute myeloid leukaemia after undergoing chemotherapy for breast cancer.

Its just like crossing the road there is always a risk of being knocked down.

All our cells have a biological clock and there is an orderly exchange of old and new cells.

But with blood cancers such as leukaemia, there is a clone of abnormal cells.

Cancer cells have an advantage over normal cells because they can survive longer.

Chemotherapy is still needed to treat most acute blood cancers, although if the mutation is known, targeted therapies can be applied.

For chronic blood cancers, there is no need for chemotherapy. Oral drugs are enough to combat the disease.

Eventually, many patients are able to wean off the drugs.

As we may be aware, immunotherapy is the buzzword in cancer treatment today.

Also called biologic therapy, it is a type of cancer treatment that boosts the bodys natural defences to fight cancer.

It uses substances made by the body or in a laboratory to improve or restore immune system function.

One of the latest treatment modalities is the CAR T-cell therapy, a form of immunotherapy that uses specially altered T cells a part of the immune system to fight cancer.

A sample of a patients T cells are collected from the blood, then modified to produce special structures called chimeric antigen receptors (CARs) on their surface.

When these CAR T-cells are reinfused into the patient, the new receptors enable them to latch onto a specific antigen on the patients tumour cells and kill the cells.

At the moment, this intravenous therapy is available in the United States and hasnt reached our shores yet. It has to be properly regulated first, says Dr Ng.

A volunteer is having his head shaved to donate hair to make wigs for cancer patients in this filepic. Hair loss is one of the side effects of chemotherapy that affect patients the most.

Following natural remedies

The consultant haematologist errs on the side of caution when patients ask about natural cancer remedies, or the dos and donts during treatment.

We always believe there should be a scientific approach to the problem.

If patients are doing okay while undergoing treatment and there is no weight loss, I tell them to go ahead and do what they always do.

However, just be particular about food hygiene, as there is a chance you may get food poisoning.

If youre undergoing chemotherapy, then youll land yourself in hospital, and if your luck is bad, you may even land up in the ICU (intensive care unit).

So make sure the food is cooked and not left overnight to reduce your chances of infection.

Eat a balanced diet, he advises.

When it comes to exercise, he says to work out within your limit.

Instead of pushing the body and running marathons or climbing mountains, go for walks.

Dr Ng says, Life should go on, but be sensible.

Dont go to crowded places because you may pick up an infection, but dont be withdrawn either. All humans need social interaction.

With the billion-dollar dietary supplements industry, companies are constantly trying to lure customers into buying their products.

A lot of supplements are just glorified vitamins in different packaging.

The more expensive they are, the more people will buy them, thinking they are good.

There are people with good intentions, but unfortunately, there are also a lot of scammers out there that is life.

For the amount you spend on supplements, why not keep the money aside and go for a trip once your treatment is over? he suggests.

Often, the late diagnosis is due to preference for alternative treatment.

These alternative treatments are like fashion shows, after some time, they go out of trend.

For me, youre wasting valuable time because cancer is not your friend.

Yes, chemotherapy is tough, but with the latest chemo-free regimen, patients are more willing to come forward.

The earlier it is treated, the higher your chances of recovering, he says.

To share his 30-odd years of knowledge and experience in the field, Dr Ng has written his third book titled Understanding Blood Disorders.

Intended for patients, caregivers and healthcare professionals, proceeds from the sales of the 270-page book will go to the newly set-up Faith Hope Love Hospice Care Malaysia in Petaling Jaya, Selangor.

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December 2017, and the Christmas party season is in full flow. Everywhere I look are scenes from some hilariously awful Dickens pastiche: revellers squeezed into warm pubs; joyful chatter spilling out on to the street; the sound of carols and the scent of mulled wine in the air. Its as if I am peering in at it all through frosted glass, wishing my own Christmas could be as carefree.

Instead, I have spent a morning turned on my side on a hospital bed while a nervous-looking young doctor works up a sweat attempting to force a long needle into my hipbone. He needs it to go deep enough that he can suck out some of the marrow inside, but my tough bones are making life difficult for him.

I dont feel so tough. My wife and I spend the next fortnight anxiously waiting for the results. Results that should confirm why my body is behaving in unexpected ways: the unusual infections; the crushing fatigue; the old jeans that suddenly slip off my waist.

Christmas is never a nice time to feel alone. Yet, despite the fact I am surrounded by loved ones, that is how I feel: terrifyingly alone. The emotions of the season get warped and amplified. I attempt to go to one party, see a friend who is going through her own hellish time, and we both sob on each others shoulders for five minutes straight. Everything feels raw and heavy. My little girl is not even 18 months old, and I love her more than anything but I find it hard to even be in the same room as her. Its all too much.

If Christmas has lost its religious meaning, then it hasnt for me. I try praying for the first time in about three decades: Er, yes, it has been a while sorry about that but could you just help me out with this one thing? I promise God and Santa Ill be all sorts of good if things turn out OK.

***

My results arrive on 22 December. There is a wait in a hospital corridor that is still too triggering to think about properly. And then a doctor calls me in, sits me down and tells me that I have a rare blood cancer called essential thrombocythemia, which sounds like some cult artist signed to Warp Records in the 90s (the doctor doesnt say that bit). There is no known cure. But dont worry, he says, its manageable. I just need to take some aspirin and keep an eye on it. You will lead a normal life, he says. My wife tells me my face instantly changed colour, the pallid grey lifting for the first time in weeks.

My little girl throws up all over the seat when we pull out of the drive, and it doesnt even feel slightly annoying

Its a strange gift, receiving blood cancer for Christmas. In some ways I preferred the Mr Frosty slushy-making kit I got when I was eight, and maybe even the Scalextric that never quite played out the way you hoped it would from the adverts. And yet what the doctor is telling me you will lead a normal life feels like the biggest and best present I have ever received. Queueing up to be discharged, I let wave after wave of euphoria run through me and think to myself: This has to be the weirdest cancer diagnosis ever.

A day later, we pack up the car and head off to my parents. My little girl throws up all over the back seat as soon as we pull out of the drive, and it doesnt even feel slightly annoying. We laugh. Life is good. That Christmas, for the first time since I can remember, I am truly happy; just living in the moment. The light seems brighter and more beautiful. I notice dew drops on plants and the smell of fresh air. I hug my wife and daughter even more tightly than usual.

***

All this relief is not to last long. In the first week of 2018, I attend a follow-up appointment and am told that, sorry, they hadnt seen all of the bone marrow samples before. My condition is, in fact, developing into a much more serious disease called myelofibrosis, which needs treatment.

A week on from that, I turn up at the hospital, steeled to start chemotherapy. But there is worse news: a team of specialists have discussed my case and they believe I am at high risk of developing acute myeloid leukaemia, a swift and deadly cancer. They recommend you have a stem cell transplant, says the doctor. I ask when. As soon as possible. If I can find a match on the stem cell donor register, then I will be dosed up with drugs so intense that my entire immune system will be wiped out; then a strangers cells will be fed into me and we will all cross our fingers and hope that my body doesnt reject them. The chance of survival and the disease not returning does not seem to me to be all that much better than 50/50. Even if it all succeeds, the recovery process will be long and gruelling.

I spend the next few weeks in a state of catatonic depression. Or do I? Because I am somehow getting things done: I organise a will, I arrange a sperm bank visit (the transplant, even if successful, will leave me infertile), I cry myself senseless writing a letter to my daughter in case the worst should happen. I also drink all the good bottles of wine I had been saving for special occasions. A bottle of Domaine Dujac Morey Saint-Denis 2012 on a Tuesday night with defrosted Quorn chilli not the pairing Id had in mind, but saving it for the future seems silly.

Through all the gloom I see something with startling clarity. I realise that what Im mourning is not so much my old life before all this started a life of pointless anxieties, petty rivalries and overthinking but rather the carefree, optimistic version of life I had briefly glimpsed over Christmas. And yet no sooner have I understood all this than the chance to enact it has been snatched away. I feel like an old professor who has finally unravelled the mysteries of the universe with his dying breath.

***

Over the next few months, something happens that I still find hard to believe. I am transferred to a new hospital with a more specialist team on the case. There are more blood tests and scans, and another long needle is forced into my hip. And then I get another gift, this one in time for Christmas 2018: my condition is not so serious as I was led to believe. It appears to be a peculiar version of a peculiar cancer caught somewhere between the relatively benign essential thrombocythemia and the more concerning myelofibrosis. But it is stable, at least for now, with no signs to suggest it will progress any time soon.

***

I like to think that this year I have made good on my promise to live like I did during the Christmas of 2017. My outlook has certainly changed. When people ask how, I always say the same thing: that its great to get older. The idea of panicking about a milestone such as my imminent 40th seems so ridiculous now. Instead, just think what a privilege it is to be able to get there.

I am more present for my family these days, and less consumed with things I cant control. I have returned to the volunteering role I thought I didnt have time for; I have got fit; I dont let work define my happiness; I am kinder to myself. I have bought lots more nice wine to replace the nice wine I drank with defrosted Quorn chilli.

Do I still get annoyed by delayed trains, lost keys or the fact my daughter is taking half an hour to put on a pink tutu, the only item of clothing in the house that shell wear? It would be a lie to say no. But the second I think: But youre not quite likely to die any more, the problem disappears. I am, undeniably, a happier person.

I still have a malfunction inside me and I still have to think about it every day. Its hard not to my spleen, inflated with excess blood cells, gently nudges against my ribs like an annoying acquaintance who would hate me to forget that all is not quite right. At some point in the future and not even the best doctors can predict exactly when the disease might whirr into life and start scarring my bone marrow, turning it into a barren wasteland that can no longer produce enough blood to keep me alive. Im hopeful that science will find a fix before that time comes. There are encouraging signs on the horizon. And if not? Well, these days I try not to dwell on the future. I am here, instead, for the present. I am alive. I am alive with the spirit of Christmas.

MPN Voice provides information and emotional support to people diagnosed with a myeloproliferative neoplasm

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Dec. 24 (UPI) Those with weak and brittle bones caused by a rare genetic disorder have reason for new hope with the discovery of a cell transplant treatment that may finally resolve the underlying cause of their disease.

In an article published Tuesday in the journal STEM CELLS, researchers show how they transplanted healthy donor bone marrow cells directly into the femur of mice with osteogensis imperfecta, or OI, a rare disease that affects bone strength.

One month following transplantation, the researchers found 18 percent of the surface that was injected with the donor cells expressed osteoblasts cells that help form new bone tissue an indication of engraftment, or growth.

In addition, long-term engraftment was then observed at three and six months post-transplantation.

This is a basic research study with potential for future translation into practice, study co-author Ivo Kalajzic, a researcher at the Center for Regenerative Medicine and Skeletal Development at the University of Connecticut, said in a statement.

Kalajzic and his colleagues also found that healthy donor cells replacing mutant collagen have the ability to improve bone strength and structure in transplant recipients.

OI are genetic disorders that mainly affect the bone. Patients with OI have bones that break easily, sometimes with no obvious cause.

In recent years, research has shown that the disorders are caused by genetic mutations associated with type 1 collagen molecules. These molecules help the body process collagen, and damage to them results in defective bone structure.

Currently, treatments for OI attempt to correct the defective bone structure, but dont focus on the underlying collagen defect.

The new findings demonstrate, however, that healthy donor stem cells that produce normal collagen in OI patients have the potential to increase bone mass and correct the collagen defect causing the condition.

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Stem cells effective against rare bone disorder in trial with mice - Breitbart

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The Galloping Ghosts are looking beyond the scoreboard to help classmate Juwan Adams battle pediatric cancer.

Khalis Whiting and her Abington girls basketball teammates have a different perspective this holiday season. A perspective that changed when through Kisses for Kyle they were put in touch with two area families who have a child battling pediatric cancer.

One George Hamlin is a student in Upper Dublin High School. The other, Juwan Adams, is a senior at Abington. Both families were on the receiving end of gifts and gift cards at the Abington-Upper Moreland game last week. It is the 10th annual giving back game since coach Dan Marsh initiated the project.

It was an amazing experience, said Whiting, the Ghosts sophomore point guard. I was not familiar with the families, but I have seen (Adams) a few times in school, but I never really knew the story until this came up and I researched him.

He has a powerful story, and hes such a strong young man to be going through this. Every time I see him in the hallways, hes always smiling and so positive. So you know give it to his family.

Adams, who has been battling Hodgkins Lymphoma since April of 2016, is in need of a match for an allogeneic stem cell transplant.

Right now, he is out of options, his mother, Andrea Adams, said. We know for sure that an allo stem cell transplant will cure him, but unfortunately, in order to do that, we need a donor.

We have tested everyone in our family, and no one is a full match, so were relying on the Be the Match registry to locate a donor. Sadly, for minorities theres a very slim chance to actually find a full 10-out-of-10 match because we dont have enough minorities on the registry.

Hes had almost every treatment available for Hodgkins, including an auto stem cell transplant, which is where he gave himself his own cells in February of 2017. He was in remission for about a year, and unfortunately, in May of this year, we found out the cancer is back, and its been spreading consistently since May.

Despite missing more than half of the last three-and-a-half years of school, Juwan maintains a 4.25 GPA and is a member of the National Honor Society.

Abington School District has been really great with accommodating him, giving him tutors, and whenever hes an in-patient, he does hospital school, Andrea said.

Juwan is the drum captain and lieutenant of Abingtons marching band, and since September is Pediatric Cancer Awareness month, for the past four years, the marching band has worn gold ribbons on their uniforms.

Since his diagnosis in eighth grade, Juwan has been active in pediatric cancer awareness events, and for the past four years, he has held toy and book drives that he presents to the children at CHOP on his birthday in July.

Last week, Juwan and his family were on the receiving end.

It was great because Juwan never wants gifts, Andrea said. Having a child with cancer, financially its tough, especially now that we have only have one person working fulltime, and that lifted a tremendous burden, and it was so unexpected.

To have him honored by his school and his peers, he was super excited about it, and he really appreciated it because a lot of kids (battling cancer) have to give up school, or their friends tend to abandon them.

I want to thank the team and the Abington students because it is very easy to turn your back on kids that are going through these things. These kids have really rallied around him. This is one of the reasons he fights so hard to be in school. He sometimes sneaks to school because he feels the love from his school and his peers. That was one thing he definitely wanted to do to finish his senior year with his class. They have really rallied around him, and this kind of thing gives him the extra push he needs to keep fighting.

Statistics say Juwan has a 23 percent chance of finding a perfect match, but the Abington senior has been proactive.

When they told him the odds of finding his match, he said he had to do something about it, Andrea said. He set a goal to register a thousand people on the national registry, half of those being minorities.

We have gone around holding bone marrow drives. Hes now an ambassador for Be the Match, and hes been an ambassador for CHOP for about two years.

Juwan is inviting everyone to join his fight.

"People are starting to wake up and realize kids with cancer more and more are dying each day, and if they have a chance to do something, they should take every step they can," Juwan said earlier this fall. It's been hard sometimes, but I have my friends and family to support me.

Read more:
Abington working toward big win - Sports - The Intelligencer

Recommendation and review posted by Bethany Smith