Aniridia is a congenital disorder that causes severe eye problems, and also affects metabolism sometimes resulting in severe obesity. It is associated with mutation of a major developmental gene, called PAX6. People born with aniridia have no irises in their eyes, often are legally blind, and whatever eyesight they have continually worsens with age. The disease is uncommon, but disorders associated with genetic mutations can involve common eye problems, including cataracts and glaucoma.

To better understand and treat aniridia and other disorders involving the PAX6 gene, researchers and clinicians at the University of Virginia are combining clinical research, patient treatment and powerful basic science investigations.

They have organized for this weekend a major symposium focused on congenital eye disorders and the PAX6 gene, bringing together top researchers from the University and around the nation and Europe, along with patients living with aniridia and their families.

The organizers are Rob Grainger, W.L. Lyons Brown Professor of Biology, and Dr. Peter Netland, Vernah Scott Moyston Professor and Chair of Ophthalmology. Both are members of UVAs Brain Institute, and are research collaborators.

In his studies, Grainger uses frogs that are mutated to mimic aniridia and other eye disorders. Netland treats congenital eye disorders and conducts clinical research.

Here, the two colleagues explain for UVA Today readers their research and the goals of the 2019 John F. Anderson Symposium, Aniridia-PAX6 and Beyond

Q. Why did you organize this particular kind of symposium, connecting how eyes develop before birth and genetic diseases that can follow?

Grainger: Each of us works on different perspectives concerning eye formation. In my lab, we focus on how the eye is constructed during embryonic development; in Peter Netlands practice, on how to treat diseases that affect these processes.

These are complementary approaches two sides of the same coin. In one case we focus on the assembly of the eye, and in the other, what occurs when the eye is not constructed properly, leading to multiple serious consequences for the patient.

This interplay highlights the importance of looking at these two perspectives together, a collaboration in this case between the two of us (one in the School of Medicine and the other in the College of Arts & Sciences) each providing insights for the other.

Netland: The value of this kind of interaction has motivated us to bring together many of the worlds experts who pursue these two perspectives, including as well a third group: patients and their families who want to learn more about these diseases and treatments. There are few meetings held with this sort of three-way interaction in mind, and we anticipate that many fruitful insights and collaborations will emerge.

Q. Dr. Netland, why is aniridia an area of particular interest to you?

Netland: More than 20 years ago, I spent an extended period of time in the Middle East and India, where there are high rates of consanguinity and congenital eye disorders, which led to a book I produced about pediatric glaucomas, other scholarly contributions and development of my clinical skills. About 20 years ago, I cared for an infant with aniridia and the family of that patient. The potentially disabling issues for the patient, which involved all parts of the eye, and the compelling issues that the family were dealing with drew me toward this condition.

Another patient was very influential to me, because she was a patient advocate and mother of an affected child. I began to see increasingly larger numbers of patients with congenital eye disorders and aniridia, and I developed further clinical and academic interests in the topic.

Around 20 years ago, we started biannual meetings with the patient advocacy group Aniridia Foundation International, and developed connections with other patient support groups, which helped shape the direction of our efforts. With increasing contact with the patients and their families, I became deeply interested in trying to help these patients.

About 20 years ago, I cared for an infant with aniridia and the family of that patient. The potentially disabling issues for the patient, which involved all parts of the eye, and the compelling issues that the family were dealing with drew me toward this condition.

- Dr. Peter Netland

This is a disease that results from damage to the gene PAX6, already known to be perhaps the most fundamental gene involved in eye formation overall and consequently affecting the entire visual system. However, we knew much less about how to treat the many facets of this disorder; for example, cataract, glaucoma and corneal opacification (scarring), which are frequently acquired by patients. Some of these problems are common in the general population, and have broad significance. Many advances have been made in the past, but there is much more progress that is needed for the future.

Q. Why do you use frogs in your eye research, Professor Grainger?

Grainger: We have been examining eye development in frog embryos for over 20 years in my lab, initially because so much embryology, going back to the beginning of the 20th century, was done on these large, easy-to-obtain-and-raise embryos.

In the early days, we were learning how the different parts of the eye, notably the lens and retina, are formed by interactions between parts of the embryo to form a coordinated whole organ exactly the interactions that are disturbed when things go awry in aniridia patients.

Q. Six years ago the Grainger lab developed a gene-editing technique that allows you to mimic human lesions. How is this advancing eye research?

Grainger: While the utility of the frog system for understanding embryological processes is undisputed, during the decades that we have been doing research, the techniques allowing us to manipulate and understand gene function have blossomed, including genome projects and more recently gene editing the ability to inactivate genes of interest to learn how they function during normal development.

In 2013, we published our first paper using this new technology to inactivate genes critical for eye formation in frogs and to follow in precise detail how things go awry. This has allowed us to make important clarifications in how these genes contribute to development of the eye. Because the frog eye develops much as the human eye, these mutations help us look in detail in a way not feasible in human embryos; thereby allowing us to understand how these genetic errors lead to the problems that occur in human patients. Specifically, we have made mutations in frogs in the PAX6 gene that lead to frogs having aniridia, with features of the animals strikingly similar to those in human patients.

These are complementary approaches two sides of the same coin. In one case we focus on the assembly of the eye, and in the other, what occurs when the eye is not constructed properly, leading to multiple serious consequences for the patient.

- Robert Grainger

Q. What kind of clinical research and therapies are UVA conducting that connect with the basic research?

Netland: We have looked at many of the vision-threatening eye problems in our aniridia patients. We have also found that their mutation is linked with obesity, and have performed clinical trials to evaluate the causes of this. We have performed studies to better understand the mechanisms for some of their clinical problems, such as glaucoma.

We are excited about precision medicine trials identifying patients who can benefit from a specific gene-based therapy and we recently completed a two-year clinical trial evaluating targeted gene therapy. In parallel, similar problems are under study in the frog to complement and build on the work with human patients.

Q. What future do you see for patients with eye disease as this research moves forward?

Netland: We are working with patients with known mutations of a specific gene, so naturally we are excited about precision medicine approaches to these patients. We believe that genetic-based approaches will continue to increase understanding of these diseases and will provide the basis for rational therapy for affected patients, and more broadly for others in the general population who are suffering from the same clinical problems. We believe that new imaging techniques will produce new insights in this area.

Grainger: In the frog, our lab has developed a method for efficiently creating exact patient mutations, again amplifying the opportunities for an integrated approach to precision medicine. There are opportunities with in situ gene modification and other gene-based therapies for addressing problems and improving the quality of life of patients.

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Patients, Physicians and Researchers Gather to Probe Genetic Eye Disorders - University of Virginia

Recommendation and review posted by Bethany Smith

WASHINGTON, D.C., Nov. 05, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM) today released its most recent quarterly sector report, offering an in-depth look at cell therapy, gene therapy, tissue engineering, and broader global regenerative medicine sector trends and metrics in the third quarter of 2019.

By further curating information provided by ARMs data partner Informa, the quarterly sector report details industry-specific statistics compiled from 959 cell therapy, gene therapy, tissue engineering, and other regenerative medicine therapeutic developers worldwide, including total financings, partnerships and other deals, clinical trial information, key clinical data events, and ARMs current strategic priorities.

Amanda Micklus, a senior consultant for Pharma Intelligence at Informa, provided an overview of the commercial, clinical, and regulatory environment in the third quarter of 2019. The report also features commentary from founding members of ARM in honor of the organizations 10-year anniversary. Excerpts from panels at ARMs 2019 Meeting on the Mesa included in the report highlight the continued progress and innovation in the sector.

Highlighted findings from the Q3 2019 data report include:

ARM will continue to update this information through new reports to be released after the close of each quarter, tracking sector performance, key financial information, clinical trials by phase, and significant clinical data events.

The report isavailable online here, with interactive data and downloadable graphics from the reportavailable here. For more information, please visitwww.alliancerm.orgor contact Lyndsey Scull at lscull@alliancerm.org.

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

Lyndsey Scull202 213 7086lscull@alliancerm.org

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The Alliance for Regenerative Medicine Releases Q3 2019 Sector Report, Highlighting Industry Trends and Metrics - BioSpace

Recommendation and review posted by Bethany Smith

Former Thrive CEO and partner at Third Rock Ventures, Steve Kafka, has hopped aboard Section 23 as a managing partner. Within his new role, Kafka will focus on supporting and expanding the firms investment portfolio and presence in Boston. Currently, Kafka serves as the executive chairman at Thrive and at ArcherDX and he will continue to serve within these roles. Kafkas previous stints include roles as president and COO at Foundation Medicine and serving in positions at Aileron Therapeutics, Infinity Pharmaceuticals, Millennium Pharmaceuticals, Strategic Decisions Group and Forrester Research.

Takeda has grabbed $660 million to help pay down the big debt it took on to buy Shire. Germanys Stada is picking up the portfolio of pharmaceuticals including OTC products in the deal, which marks Takedas 4th auction since closing the Shire buyout. Takeda has garnered $6.5 billion of the $10 billion its set its sights on for debt reduction.

High-flying Chinese biotech BeiGene $BGNE has licensed Asian rights to a preclinical cancer drug out of Seattle Genetics. There are no specifics on what this drug is, but Seattle Genetics stands to earn up to $160 million from BeiGene if it pans out. They didnt break out the upfront. Seattle Genetics $SGEN is hanging on to US rights for this therapy.

Engitix, Inchas been awarded with a golden ticket providing one year of lab bench space and shared laboratory and office space to LabCentral by Takeda Pharmaceutical. The company says that the golden ticket will help further advance its human extracellular matrix (ECM) research in fibrosis and solid tumors disease progression.

CODA Biotherapeutics developing a chemogenetic gene therapy platform to treat intractable neurological diseases has closed its Series A financing round, bagging a total raised amount of $34 million. The companys existing investor, Versant Ventures, led the round and was joined by existing investors MPM Capital and Astellas Venture Management.

Allogene and immune cell therapy company, Notch Therapeutics, have entered into an exclusive worldwide collaboration and license agreement to research and develop induced pluripotent stem cell (iPSC) AlloCAR therapy products for initial application in non-Hodgkin lymphoma, leukemia and multiple myeloma. Under the partnership, the companies will create allogeneic cell therapy candidates from T cells or natural killer (NK) cells using Notchs Engineered Thymic Niche (ETN) platform. The collaboration comes two months after Allogene teamed up with researchers from Stanford University to investigate a nucleic acid delivery system that more effectively, safely and flexibly delivers intracellular RNA or DNA into lymphocytes, including T cells.

Black Belt TX and Praxis Biotech are teaming up in a strategic partnership to discover and develop new small molecule therapeutics to undisclosed targets which are aimed atmodulating key control mechanisms in the stress response pathways in cancer.

As reported by Business Quarter (BQ), Oxford Drug Design has bagged 2.2 million in a financing round. The round was led by new and existing investors the Angel CoFund and o2h Ventures along with grant funding from the UK Department of Health and Social Cares UK-China research competition. This brings the companys total 2019 funding to over 9m.

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Steve Kafka joins Section 23 as managing partner; Takeda auctions off $660M drug portfolio in bid to reduce post-Shire M&A debt load - Endpoints News

Recommendation and review posted by Bethany Smith

HAIFA, Israel, Nov. 04, 2019 (GLOBE NEWSWIRE) -- Pluristem Therapeutics Inc. (PSTI) (PSTI), a leading regenerative medicine company developing novel placenta-based cell therapy products, today announced that the RESTORE Consortium is hosting its 1st Advanced Therapies Science Meeting (ATSM), which is being held November 25-26, 2019 in Berlin. As a leading member of the large-scale research initiative, Pluristem, along with additional respected members, is committed to accelerating the availability of advanced therapies to all those in need, a main motivation standing behind RESTORE.

Led by Charit-Universittsmedizin Berlin, and coordinated by Professor Hans-Dieter Volk from the BIH-Center for Regenerative Therapies in Berlin, RESTORE aims to promote groundbreaking research, drive Europe to the forefront in advanced therapies and deliver a pipeline of potentially transformative cures to patients in need. Advanced Therapies are a potential game changer in health care, aiming to shift our focus from chronic treatment of disease to regeneration of health, said Prof. Volk. We are determined to translate promising research findings into safe therapies, and we are working across disciplines and national borders in order to achieve this goal. The 1st Advanced Therapies Science Meeting provides the opportunity to discuss the still numerous obstacles in the way of implementing these promising therapies in routine clinical care.

This initiative may hold the key for changing the approach towards medicine in Europe, and advancing solutions for patients in need, said Zami Aberman, Executive Chairman of Pluristem. The European Commission is poised to make a significant investment of up to 1 billion in a consortium of companies that can drive forward the development of novel regenerative therapies, and we are pleased to be a leading part in this effort. Given our proprietary cell manufacturing technology and broad, late-stage pipeline, we believe we can play a key role toward making the transforming promise of advanced therapies into a reality.

The 1st ATSM will bring together experts from industry, patient organizations and academia to discuss the challenges within the field of advanced therapies, which include gene and cell therapies and tissue-engineering approaches. The ATSM is focused on trying to drive forward a concerted interdisciplinary effort, making use of science, infrastructure and funding within Europe to make regenerative therapies available to the broadest possible patient population.

The two-day program will include talks from Nobel Prize winner Ada Yonath (Director of Weizmann Institute of Science, Israel), Michele De Luca (University of Modena, Italy), Timothy OBrien (National University of Ireland, Galway, Ireland), Maksim Mamonkin (Baylor College of Medicine, USA), Manuela Gomes (University of Minho, Portugal) and others.

RESTORE partners include the Charit Universittsmedizin Berlin and Berlin Institute of Health (Germany), the University of Zurich (Switzerland), Cell and Gene Therapy Catapult (United Kingdom), TissUse GmbH (Germany), Pluristem (Israel), Miltenyi Biotec GmbH (Germany), INSERM Institut National de la Sant et de la Recherche (France), Innovation Acta S.r.l. (Italy), Fondazione Telethon Milan (Italy), and the University of Minho (Portugal).

About Pluristem TherapeuticsPluristem Therapeutics Inc. is a leading regenerative medicine company developing novel placenta-based cell therapy product candidates. The Company has reported robust clinical trial data in multiple indications for its patented PLX cell product candidates and is currently conducting late stage clinical trials in several indications. PLX cell product candidates are believed to release a range of therapeutic proteins in response to inflammation, ischemia, muscle trauma, hematological disorders and radiation damage. The cells are grown using the Company's proprietary three-dimensional expansion technology and can be administered to patients off-the-shelf, without tissue matching. Pluristem has a strong intellectual property position; a Company-owned and operated GMP-certified manufacturing and research facility; strategic relationships with major research institutions; and a seasoned management team.

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Safe Harbor StatementThis press release contains express or implied forward-looking statements within the Private Securities Litigation Reform Act of 1995 and other U.S. Federal securities laws. For example, Pluristem is using forward-looking statements when it discusses the potential for the RESTORE Consortium to receive up to a 1 billion award by the European Commission and the timing of the potential award, that RESTOREs aim is to promote groundbreaking research, drive Europe to the forefront in advanced therapies and deliver a pipeline of potentially transformative cures to patients in need, that RESTORE and the 1st ATSM may hold the key for changing the approach towards medicine in Europe, and advancing solutions for patients in need, and its belief that given its proprietary cell manufacturing technology and broad, late-stage pipeline, it believes it can play a key role toward making the transforming promise of advanced therapies into a reality. These forward-looking statements and their implications are based on the current expectations of the management of Pluristem only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; Pluristem may encounter delays or obstacles in launching and/or successfully completing its clinical trials; Pluristems products may not be approved by regulatory agencies, Pluristems technology may not be validated as it progresses further and its methods may not be accepted by the scientific community; Pluristem may be unable to retain or attract key employees whose knowledge is essential to the development of its products; unforeseen scientific difficulties may develop with Pluristems process; Pluristems products may wind up being more expensive than it anticipates; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; Pluristems patents may not be sufficient; Pluristems products may harm recipients; changes in legislation may adversely impact Pluristem; inability to timely develop and introduce new technologies, products and applications; loss of market share and pressure on pricing resulting from competition, which could cause the actual results or performance of Pluristem to differ materially from those contemplated in such forward-looking statements. Except as otherwise required by law, Pluristem undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. For a more detailed description of the risks and uncertainties affecting Pluristem, reference is made to Pluristem's reports filed from time to time with the Securities and Exchange Commission.

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RESTORE Consortium to Host the 1st Advanced Therapies Science Meeting, Aiming to Translate Promising Research into a Game Changer in Healthcare -...

Recommendation and review posted by Bethany Smith

Alignment With FDA on GMP Commercial Manufacturing Process for KB103 (Bercolagene telserpavec, B-VEC)KB105 Granted Fast Track Designation by the FDAPlatform Patent for Delivering any Effector to the Skin Granted by USPTO

PITTSBURGH, Nov. 04, 2019 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (Krystal) (KRYS), a gene therapy company developing medicines to treat dermatological diseases, announced financial results for third quarter 2019, recent corporate highlights and upcoming milestones.

"Following our CMC meeting, we believe we have a scalable GMP commercial process in place to fulfill future patient demand and anticipate a modest impact to our previously disclosed B-VEC timeline, said Krish S. Krishnan, chairman and chief executive officer of Krystal Biotech. We plan on announcing our agreement with the FDA on trial design and endpoints prior to initiating the B-VEC pivotal trial.

Corporate Highlights

CMC alignment and End of Phase 2 meeting

Positive results from Phase 1/2 trial of B-VEC

KB105

Patents

Pipeline

Financial results for the quarter endedSeptember 30, 2019

For additional information on the Companys financial results for the quarter ended September 30, 2018, refer to form 10-Q filed as with the SEC.

About KB103KB103 is Krystals lead product candidate, currently in clinical development, seeks to use gene therapy to treat dystrophic epidermolysis bullosa, or DEB, an incurable skin blistering condition caused by a lack of collagen in the skin.KB103 is a replication-defective, non-integrating viral vector that has been engineered using the HSV-1 virus employing Krystals STAR-D platform to deliver functional human COL7A1 genes directly to the patients dividing and non-dividing skin cells.Krystals vector can penetrate skin cells more efficiently than other viral vectors.Its high payload capacity allows it to accommodate large or multiple genes and its low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.

About Dystrophic Epidermolysis Bullosa, or DEBDystrophic epidermolysis bullosa, or DEB, is an incurable, often fatal skin blistering condition caused by a lack of collagen protein in the skin. It is caused by mutations in the gene coding for type VII collagen, or COL7, a major component of the anchoring fibrils, which anchor the epidermis to the underlying dermis, and provide structural adhesion in a normal individual. The lack of COL7 in DEB patients causes blisters to occur in the dermis as a result of separation from the epidermis.This makes the skin incredibly fragile, leading to blistering or skin loss at the slightest friction or knock. It is progressive and incredibly painful.

The most severe form of DEB is recessive DEB, or RDEB, which is caused by null mutations in the COL7A1 gene.DEB also occurs in the form of dominant DEB, or DDEB, which is considered to be a milder form of DEB.There are no known treatments which affect the outcome of either form of the disease, and the current standard of care for DEB patients is limited to palliative treatments.Krystalis developing KB-103 for the treatment of the broad DEB population, including both recessive and dominant forms of the disease.

About KB105KB105 is Krystals second product candidate, currently in clinical development, seeks to use gene therapy to treat patients with TGM-1 deficient ARCI. KB105 is a replication-defective, non-integrating viral vector that has been engineered employing Krystals STAR-D platform to deliver functional human TGM-1 gene directly to the patients dividing and non-dividing skin cells.

About Autosomal Recessive Congenital IchthyosisTransglutaminase 1 (TGM-1) is an essential epidermal enzyme that facilitates the formation of the epidermal barrier, which prevents dehydration, and protects the skin from unwanted toxins and surface microorganisms. The loss of TGM-1-activity results in the severe genetic skin disease autosomal recessive congenital ichthyosis (ARCI). Most patients with a TGM-1-deficiency exhibit life-long pronounced scaling with increased trans epidermal water loss (TEWL). The scales are plate-like, often of a dark color, and cover the whole-body surface area. Erythroderma is either absent or minimal. Such patients usually have ectropion and, at times, eclabium, hypoplasia of joint and nasal cartilage, scarring alopecia, especially at the edge of the scalp, and palmoplantar keratoderma. Additional complications include episodes of sepsis, fluid and electrolyte imbalances due to impaired skin barrier function, and failure to thrive, especially during neonatal period and infancy. Severe heat intolerance, and nail dystrophy are also frequently observed. TGM-1-deficient ARCI is associated with increased mortality in the neonatal period and has a dramatic impact on quality of life. No efficient treatment is available; current therapy only relieves some symptoms. There are approximately 23,000 cases of TGM1 deficient ARCI worldwide and about 400 new cases per year globally.

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About the STAR-D Gene Therapy PlatformKrystalhas developed a proprietary gene therapy platform, the Skin TARgeted Delivery platform, or STAR-D platform, that consists of an engineered HSV-1 viral vector and skin-optimized gene transfer technology, to develop off-the-shelf treatments for dermatological diseases. We believe that the STAR-D platform provides an optimal approach for treating dermatological conditions due to the nature of the vector. Certain inherent features of the HSV-1 virus, combined with the ability to strategically modify the virus in the form employed as a gene delivery backbone, provide the STAR-D platform with several advantages over other viral vector platforms for use in dermatological applications.

AboutKrystal BiotechKrystal Biotech, Inc.(KRYS) is a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases. For more information, please visithttp://www.krystalbio.com.

Forward-Looking StatementsThis press release includes certain disclosures that contain forward-looking statements, including, without limitation, statements regarding development timelines for KB103, the ability of KB103 to be a transformative treatment option for DEB patients and the ability of our Ancoris manufacturing facility to supply KB103 for the forthcoming clinical trial . You can identify forward-looking statements because they contain words such as anticipate, believes and expects. Forward-looking statements are based on Krystals current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees nor assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Krystals filings with theSecurities and Exchange Commission, including its registration statement on Form S-3, and in its Forms 10-K and 10-Q, as modified or supplemented from time to time, under the caption Risk Factors.

CONTACTS:

Investors:Ashley R. RobinsonLifeSci Advisorsarr@lifesciadvisors.com

Media:Darren Opland, PhD LifeSci Public Relationsdarren@lifescipublicrelations.com

Source: Krystal Biotech, Inc.

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Krystal Biotech Reports Third Quarter 2019 Financial and Operating Results - Yahoo Finance

Recommendation and review posted by Bethany Smith

Worldwide Market Report:

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CNS Gene Therapy Market: Potential and Niche Segments, Geographical regions and Trends 2018 2028 - Health News Office

Recommendation and review posted by Bethany Smith

Many for-profit stem cell clinics advertise therapies that are not backed by science and may actually cause harm.

For-profit stem cell clinics have popped up around California in recent years, advertising that they can treat everything from arthritis to Alzheimers, without FDA approval.

They claim that injections of stem cells (naturally occurring blank slate cells that can grow into any type of cell) can help alleviate pain or illness by replacing or regenerating diseased tissue claims that are not supported by existing research. The procedures can cost thousands of dollars out-of-pocket, and regulators have warned that patients have developed tumors, suffered infections and even lost eyesight after unapproved procedures.

No one knows how many clinics there are, but California reportedly has more than any other state. We asked Arnold Kriegstein, MD, PhD, director of the UC San Francisco Developmental & Stem Cell Biology Program, about whats real and whats not in stem cell medicine.

How do these clinics operate?

There has been an explosion of so-called clinics offering stem cell treatments for a wide range of ailments, none of which have been shown to be effective. They are largely unregulated. Many clinics claim that they can treat untreatable illnesses like Alzheimer's disease, autism, muscular dystrophy, or stroke. The list is quite extensive.

The majority are using fat tissue for their stem cells, obtained through liposuction. These are usually autologous cells, which means that they are taking the patient's own tissue and extracting cells to re-administer to the same patient, usually through an intravenous route. In addition to fat cells, some clinics administer bone marrow stem cells or umbilical cord or placental stem cells, which come from unrelated donors.

The clinics often advertise through testimonials from patients who've received their therapies. Many of the conditions that the testimonials address are the kinds that normally improve or fluctuate over time, such as joint pain, low back pain, arthritis, or multiple sclerosis.

The problem is that patients will receive a treatment, and then, within a month or two, they'll notice that the aches and pains in the joints are improving, and they will attribute the improvement to the stem cell therapy, when in fact it would've happened regardless.

What is the risk of trying an unproven stem cell treatment?

Reports of physical harm have included infections and the development of tumors. When using cells that are not the patients own, umbilical cord cells for example, immune responses can occur often triggering inflammatory conditions.

In cases where stem cells have been delivered into the eye, blindness has been reported, and when they have been delivered to the central nervous system through lumbar puncture (spinal tap), adverse outcomes including serious infections of the central nervous system and tumors have occurred.

Then there's the emotional cost associated with raising false hope, and the financial loss that comes from exorbitant fees charged for ineffective, potentially harmful therapies.

Why arent there more legitimate stem cell therapies available?

Stem cells have been in the news so much over the last decade or so that I think it has created the impression that therapies are already on the market. The reality is that it is very early days for the science. The most interesting, most promising animal studies are only now beginning to be translated into clinical trials, and the process for approval of therapies takes many years and very few are likely to succeed.

Unfortunately, the public needs to be patient, but the good news is that potential treatments are progressing along the pipeline.

What are some examples of proven stem cell therapies?

For the last 50 years or so, there have been countless patients successfully treated with hematopoietic stem cells, commonly known as bone marrow transplants. This remains the prototype for how a stem cell therapy can work. Other successful examples include corneal stem cell grafts for certain eye conditions, and skin grafts for burn victims.

There are efforts to see if stem cells could successfully treat diseases like Parkinson's and diabetes, particularly type 1 diabetes. There are clinical trials testing whether stem cell therapy might work against macular degeneration, a blinding disease that is very common as people age. There are also early stage clinical trials for nervous system disorders including stroke, spinal cord injury, and ALS (Lou Gehrigs disease).

All of these examples are still at a very early stage, where the primary goal is to make sure that the approaches are safe. To determine if they are effective will require large, well-controlled, relatively long-term clinical trials.

What will it take to advance stem cell therapy into more real treatments?

This is where basic research comes in. The field is evolving quickly, there's much to be done, and there's still a huge amount of promise in stem cell therapies down the road. But it's going to take a lot of very careful and very laborious research before we get there.

Original post:
Stem Cell Therapy: What's Real and What's Not at California's For-Profit Clinics - UCSF News Services

Recommendation and review posted by Bethany Smith

Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.

The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.

Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.

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Three UCLA scientists receive grants totaling more than $18 million - UCLA Newsroom

Recommendation and review posted by Bethany Smith

Saving the life of a fellow Canadian can be as easy as checking a box online or saying yes to being an organ donor when you renew your drivers license. But, thats just the beginning for those wanting to make a difference.

Deceased donations

In Alberta, individuals over the age of 18 can register their intent to become an organ or tissue donor when they die by using the Alberta Organ and Tissue Donation Registry. (Go to myhealth.alberta.ca online and search organ donation registry.) As well, agents and provincial registries are required to ask the donor question when clients are renewing a drivers licence or identification card.

For those who have Alberta Health Cards issued prior to 2018, the back of the card can be signed (with a witness) to declare their intention to donate.

The Alberta registry has been integrated into the provinces health care system through the use of donor co-ordinators. If a person has declared his or her intent to donate and is in a position to be considered for organ or tissue donation, a co-ordinator will discuss it with family members, who ultimately make the final decision.

Each deceased donor can provide up to eight organs (both lungs, both kidneys, liver, heart, pancreas, intestines), while donated tissues can benefit up to 75 individuals.

Living donations

The vast majority of living organ donors spares one of their two functioning kidneys to a person in need, though living liver donations also occur to a lesser extent.

In most cases, family members or acquaintances donate a living organ if theyre healthy enough to safely act as a donor. Once a viable donor is found, transplant programs in both Calgary and Edmonton perform the surgeries for kidneys, while live liver transplants are only performed in Edmonton.

Theres also been a rise in so-called altruistic donors, who are willing to share their organs with a stranger. Both the Kidney Foundation of Canada and Canadian Blood Services can advise prospective living donors on where to turn, while Alberta Health can connect donors to local living donor programs.

Canadian Blood Services also operates the Kidney Paired Donation Program, an inter-provincial initiative that maintains prospective donors in a registry if they arent a compatible match for their intended recipient. Since January 2009, some 500 living donors across Canada have entered the KPD program, including 90 anonymous donors who joined the program without a specific recipient in mind. Non-directed, anonymous donations are responsible for more than two-thirds of the transplants in the KPD program, and all patients with a match have received a transplant in less than a year.

The Living Donor Services Program Edmonton: Phone 780-407-8698; toll free 1-866-253-6833; email: livingdonors@ahs.ca.

Southern Alberta Transplant Program Calgary: Phone 403-944-4635.

More information on kidney health is available from the Kidney Foundation of Canada: http://www.kidney.ca; 780-451-6900 or 403-255-6108.

More information on liver health is available from the Canadian Liver Foundation: http://www.liver.ca; 403-276-3390 or 1-800-563-5483.

Details about Green Shirt Day and Logan Boulet are at greenshirtday.ca.

Stem cell donations

Stem cell transplants replace a patients unhealthy stem cells with a donors healthy ones, and can be used to treat cancers and other diseases. The three sources of stem cells are from bone marrow, peripheral (circulating) blood and umbilical cord blood.

Prior to any donation, the donor will undergo a comprehensive health assessment before undergoing the procedure. Peripheral blood stem cell donation only requires blood to be drawn from a needle in hospital following five days of under-the-skin injections to boost the number of blood cells in the bloodstream.

Bone marrow donations are performed under anesthesia, with hollow needles used to withdraw stem cells from bone marrow in the back of pelvic bones. The procedure lasts between 45 to 90 minutes and the marrow replenishes itself in four to six weeks.

Those who wish to become a stem cell donor can call Canadian Blood Services at 1-888-2-DONATE (1-888-236-6283) or by visiting the agencys website at blood.ca.

Read the original post:
Organ donations: What you can do to help save a life - Calgary Herald

Recommendation and review posted by Bethany Smith

The research provides in-depth study and analysis on Stem Cell Banking market. This report also gives complete overview of the global market, covering the different aspects such as product definitions along with leading market players. To get better perspectives of global market, relevant chart and graphs are included in the report.

Stem cell banking or preservation is a combined process of extraction, processing and storage of stem cells, so that they may be used for treatment of various medical conditions in the future, when required. Stem cells have the amazing power to get transformed into any tissue or organ in the body. In recent days, stem cells are used to treat variety of life-threatening diseases such as blood and bone marrow diseases, blood cancers, and immune disorders among others.

Stem Cell Banking market report gives the reasonable picture of the current industry situation which incorporates authentic and anticipated market estimate in terms of esteem and volume, technological advancement, macroeconomic and governing factors in the market. The report provides detailed statistics and strategies of the best key players in the industry. The report additionally gives a broad study of the distinctive market sections and areas.

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Some of the leading key players profiled in this study:Cordlife, ViaCord (A Subsidiary of PerkinElmer), Cryo-Save AG, StemCyte India Therapeutics Pvt. Ltd., Cryo-Cell International, Inc., SMART CELLS PLUS, Vita 34, LifeCell, Global Cord Blood Corporation, CBR Systems, Inc

What the report features:-

Global analysis of Stem Cell Banking market from 2017 2027 illustrating the progression of the market.

Forecast and analysis of Stem Cell Bankingmarket by Dosage, Route of Administration and Application from 2017 2027

Forecast and analysis of Stem Cell Banking market in five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America

The market of stem cell banking is anticipated to grow with a significant rate in the coming years, owing to factors such as, development of novel technologies for stem cell preservation and processing, and storage; growing awareness on the potential of stem cells for various therapeutic conditions. Moreover, increasing investments in stem cell research is also expected to propel the growth of the stem cell banking market across the globe. On other hand rising burden of major diseases and emerging economies are expected to offer significant growth opportunities for the players operating in stem cell banking market.

The Global Stem Cell Banking Market is characterized by the presence of a large number of global, regional, and local players and is highly-competitive. These international players are increasingly focusing on expanding their geographical presence and they have huge production facilities located across the world. Several vendors are increasingly competing against each other based on factors such as innovations, price, and quality of the product. Vendors with better financial and technological resources can withstand changes in different market conditions when compared to their competitors.

The various factors supporting the markets growth and those posing threat are studied in detail in this report. Additionally, the market study segments the Global Stem Cell Banking Market based on end-users, verticals, and size. In these sections, it shields various factors impelling the markets trajectory across the segments. Furthermore, it recognizes the most lucrative of them all to help investors take the well-informed decision.

The Global Stem Cell Banking Market Analysis to 2025 is a specialized and in-depth study of the biotechnology industry with a focus on the global market trend. The stem cell banking market report aims to provide an overview of global stem cell banking market with detailed market segmentation by source, service type, application, and geography. The global stem cell banking market is expected to witness high growth during the forecast period. The stem cell banking market report provides key statistics on the market status of the leading market players and offers key trends and opportunities in the market.

Relating to the latest hierarchy in the global Stem Cell Banking market, the report summaries some of the crucial players operative in the market. Discriminating information about the significant players including their revenue, business segmentation, product portfolio, and financial overview has been integrated in the report. Latest improvements in the industry have been taken into concern while anticipating the future perspective of the market. The report also exemplifies the various marketing channels prevailing in the global market and conveys information about few of the critical distributors functioning in the market. The report assists as a helpful guide for the new as well as prevailing players in the market.

Market Segmentation:

The global stem cell banking market is segmented on the basis of source, service type, and application. The source segment includes, placental stem cells (PSCS), dental pulp-derived stem cells (DPSCS), bone marrow-derived stem cells (BMSCS), adipose tissue-derived stem cells (ADSCS), human embryo-derived stem cells (HESCS), and other stem cell sources. Based on service type the market is segmented into, sample processing, sample analysis, sample preservation and storage, sample collection and transportation. Based on application, the market is segmented as, clinical applications, research applications, and personalized banking applications.

This report includes several arrangements, definitions, the chain assembly of the industry in one piece, and the various uses for the global market. This section also integrates an all-inclusive analysis of the different enlargement plans and government strategies that influence the market, its cost assemblies and industrialized processes. The second subdivision of the report includes analytics on the Global Stem Cell Banking Market based on its size in terms of value and volume.

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The most crucial key factors in the businesses have been elaborated to get ample and accurate data of market dynamics. Rising needs and popularity of Stem Cell Banking Market sector is driving the flow of the market towards progress. In addition to this, it lists the factors which are restraining the growth of the market.

Reason to Buy

Save and reduce time carrying out entry-level research by identifying the growth, size, leading players and segments in the global Stem Cell Banking Market

Highlights key business priorities in order to assist companies to realign their business strategies.

The key findings and recommendations highlight crucial progressive industry trends in the Stem Cell Banking Market, thereby allowing players to develop effective long term strategies.

Develop/modify business expansion plans by using substantial growth offering developed and emerging markets.

Scrutinize in-depth global market trends and outlook coupled with the factors driving the market, as well as those hindering it.

Enhance the decision-making process by understanding the strategies that underpin commercial interest with respect to products, segmentation and industry verticals.

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The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We are a specialist in Technology, Healthcare, Manufacturing, Automotive and Defense.

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Stem Cell Banking Market Anticipated to Grow at a Significant Pace by 2028 - Weekly Spy

Recommendation and review posted by Bethany Smith

How isleukaemia treated?

The treatment of leukaemia varies depending on the patient and type of leukaemia they have.

Acute leukaemia (fast developing) is usually curable with standard treatments, such as chemotherapy.

Chronic leukaemia (slow developing), is often incurablebut treatable. For CLL (a form of chronic leukaemia) some patients are not given treatmentstraight away;however if they do require treatment it will often involve chemotherapy.

The main treatments for leukaemia are:

Chemotherapy: This treatment involves theuse ofdrugs.Chemotherapy drugs either kill cancerous cells or stop them from dividing; they can also kill normal blood cells as a side effect.The type of leukaemia you have will depend on the amount and strength of chemotherapy you are offered, along with other factors such as your age and fitness.

Radiation therapy:Similar to chemotherapy, radiation therapy can be used to destroy the cancerous cells but using radiation waves rather than drugs.Again, the type of leukaemia you have will determine what treatment you're offered. External beam radiation therapy (EBRT) is often used for CLL.It is a fast, painless procedure which usually lasts just a few minutes.

Targeted therapy:Drugs are used to block the growth of cancer cells by disturbing specific molecules in the cells. Targeted therapy can also kill cancer cells by stimulating the patient's immune system to recognise the cells as a threat and consequently kill them.

Biological therapy:This treatment does not target the cancer cells directly, but instead helps to stimulate the body's immune system to act against the cancer. It is also often referred to as "immunotherapy". It is often usedfor patients with CML.

Stem cell or bone marrow transplant: Transplants for stem cells or bone marrow are commonly carried out for patients withacute leukaemia,if chemotherapy does not prove effective.By undergoing a stem cell or bone marrow transplant it can help replenish the healthy bone marrow in patients, and stimulate new growth that restores the immune system. It is usually given to younger, or more healthy patients.

Leukaemia Care, which provides support to individuals and families affected by blood cancer, is one ofthree charities supported by this years Telegraph Christmas Charity Appeal. Our two other charities are Wooden Spoon, which works with Britains rugby community to raise money for sick, disabled and disadvantaged children; and The Silver Line, a 24-hour helpline and support service for lonely elderly people. To make a donation, visit telegraph.co.uk/charity or call 0151 284 1927

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Leukaemia: what is it, how to spot the warning signs and who is at risk? - The Telegraph

Recommendation and review posted by Bethany Smith

Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways

Stem Cells In The Body

All humans are born and develop from a small tiny structure called an egg. The cells in the egg have a tremendous potential to develop, multiply and form different cells that are functional in the body. These cells are called mother cells or in scientific terms, they are called stem cells. And all human beings have these stem cells preserved in the body. It is these cells that help us in every day wear and tear and also for tissue repair.

The Body Can Heal Itself

Most of the cells in our body have a definite lifespan that need to be replaced by new cells. The stem cell reserves in the body make up for this and it is done without our knowledge! In fact, any cut or injury, external or internal is healed by the bodys innate mechanism. Our intelligent body recognises the signal of injury and recruits the required stem cells. These stem cells transform themselves into the cells that are required for the repair of the injury and it is always many types of cells in various permutations and combinations.

Where Stem Cells Reside

Bone marrow can be considered as the manufacturing unit of stem cells as it is continuously making blood cells and keeps our circulatory system working perfect all the time. Circulating blood is another source of stem cells, because it works as a courier, carrying cells and other essential enzymes, hormones from one organ to the other in the body. The body converts all the extra material into fat which gets accumulated around the belly. This fatty tissue works like a fixed deposit of stem cells.

Stem cells either from the donor (allogenic) or from the patient (autologous) are being used for more than 50 years and especially for treatment. Blood cancers and other blood-related diseases can be cured using a perfect matched donor stem cells obtained from bone marrow. Patients suffering from organ cancers like breast cancer etc. are given autologous stem cells as a supportive treatment along with chemotherapy and/or radiation.

Protocols for these treatments are standardised globally and considered as standard-of-care. In recent years, umbilical cord blood derived stem cells are being used as an alternative to bone marrow, especially in the paediatric age group. People fall victim to numerous degenerative diseases which occur, as the repairing stem cell system from the body fails slowly with age. Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways. It may also replace, rejuvenate or restore the damaged tissues.

Our body carnes its own repairing kit in the form of stem cells and the body tries its level best to make use of these stem cells to ward off diseases. However, it is possible that with age, the bodys power to recruit and make use of the stem cells diminishes slowly. This is when dreadful degenerative diseases like diabetes, arthritis, Parkinsons disease and heart problems, set in. Heres what the clinical applications of regenerative medicine have found novel mechanisms of:

It is increasingly observed that this kind of autologous therapy takes care of the root cause of disease and offers benefits to patients to whom there is no further solution in other modalities of treatment.

Since each tissue and organ of our body is made up of cells that are derived from the egg cell, any disease which is due to derangement or degeneration of cells can be cured using autologous cellular therapy. And though the list can be endless, here are some examples where there have been very promising results:

Read the original post:
How Stem Cells Can Heal The Body - Version Weekly

Recommendation and review posted by Bethany Smith

This level of disease stabilization has not been observed to this date in approved or investigational ALS therapies.

- Mr. Chaim Lebovits, CEO, Brainstorm Cell Therapeutics

In May of this year, I published an article on Brainstorm Cell Therapeutics (BCLI). This small company is developing a mesenchymal stem cell product called NurOwn, which is in late phase 3 trials targeting amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease. My article was bearish, deploring not only the company's cash position but also phase 2 trial data. The article can be read here.

That article received a lot of critical comments from the ALS community. That made me realize that a fair overview of the issues could be best addressed by going through the comments, as well as my own coverage, and by asking BCLI management, specifically its CEO, Chaim Lebovits, to clarify some of these issues. So, that's what I did. I emailed a set of 11 questions to Mr. Lebovits, and he was kind enough to respond to them in great detail. The entire interview, sans any edits, is available to Total Pharma Tracker members.

Mr. Lebovits has been with BCLI for well over 12 years, joining in 2007 as president and also becoming the CEO in 2015. He has helped develop NurOwn through its preclinical stage to its current stage and is, therefore, just the right person to talk to if we want to understand NurOwn and BCLI.

I began by asking him to locate NurOwn in the ALS therapy space and where it stands with respect to competitors. What's its mechanism of action, and how does that MOA distinguish it from the competition?

Mr. Lebovits said that there are "currently 4 products active in phase 3 ALS clinical trials (Brainstorm (NurOwn, autologous MSC-NTF cells secreting neurotrophic factors), Orion (levosimendan, muscle troponin calcium sensitizer), Orphazyme (arimochlomol, heat shock protein enhancer), and Biogen (SOD1, antisense oligonucleotide)." Top-line data from these ALS phase 3 trials is expected in 2020 (Q4 2020 for Brainstorm) and Orion, 2021 (Orphazyme), and 2022 (Biogen). He discussed a number of earlier-stage compounds as well as various stem cell therapies. He said that what distinguishes NurOwn among ALS therapies is that it "confers both neuroprotection and immunomodulation by delivering neuronal survival factors and immune regulatory molecules, including microRNA directly to the CNS compartment at or near the site of disease, and therefore directly addresses two important ALS disease mechanisms."

Among stem cell therapies, Mr. Lebovits said that NurOwn distinguishes itself by being autologous and because it can produce high levels of neurotrophic factors. Moreover, unlike most stem cell competitors, it's delivered directly into the spinal fluid through bimonthly lumbar punctures, unlike others that need an invasive surgical procedure "that carries considerable morbidity."

This feature it shares with a competing product from Corestem. However, it's differentiated from Corestem because "NurOwn is more convenient than the Corestem product as a single bone marrow cell harvest due to validated cryopreservation, whereas the Corestem product requires repeat bone-marrow aspiration for each treatment."

My next question was a technical question about pharmacoresistance. I wanted to know how NurOwn is managing to cross the blood-spinal cord barrier despite the strong pharmacoresistance (body's resistance to drugs) seen in ALS, specifically for disease-modifying neurotrophic factors. What was it about NurOwn's delivery mechanism that the company thinks is overcoming this natural resistance. So I asked: "Talking about MOA, pharmacoresistance is a disease driving mechanism in ALS. Can you discuss NurOwn's delivery mechanism vis-a-vis the inability of neurotrophic factors to effectively cross the blood-brain barrier, or, specifically, the blood-spinal cord barrier (BSCB)? Please correlate that discussion regarding the observed increase in CSF NTFs post-treatment as seen in the phase 2 trial."

Mr. Lebovits explained this with great clarity - for his entire response, take a look at the complete interview. Broadly, what he said was that NurOwn, being delivered through lumbar puncture directly into the spinal fluid, has an advantage. Moreover, the cells secrete neuronal survival factors as well as molecules that regulate the immune system, so that they are able to survive and overcome the pharmacoresistance. Systemically administered NTFs are unable to do that.

As he said, "In the phase 2 trial, CSF biomarkers obtained just prior to treatment and two weeks afterward demonstrated that MSC-NTF cell-secreted neurotrophic factors were significantly increased post-treatment and correlated with the reduction in inflammatory biomarkers, consistent with the proposed mechanism of action."

My third and fourth questions related to aspects of the phase 2 study. One, comparison of safety and efficacy data with competitors, and two, the relevance of the reported caspase-3 reduction of 60% in responders versus 30% in non-responders.

Mr. Lebovits said that although the phase 2 study was not powered for efficacy, it exhibited a "level of disease stabilization (that) has not been observed to this date in approved or investigational ALS therapies." About the ongoing phase 3 study, he said the following:

Those who read my original article will recall I was particularly puzzled by the increased occurrence of serious adverse events in active-treatment groups than in placebo groups. 8/36 or 22.2% patients in the treatment arm had an SAE compared to only one out of 12 placebo patients, or 8.3%. Most SAEs were related to the progression of the underlying ALS, most commonly dysphagia. No SAEs were related to study treatment. So I asked Mr. Lebovits how this data could be interpreted in the most positive way.

According to him, this decline was not an effect of treatment itself and simply indicated the need for repeat dosing in this patient group. His exact response was as follows:

The MSC-NTF treated group had a slightly more rapid rate of decline compared to the placebo group in the three-month run-in period and most ALS disease progression in the treated group was seen toward the end of the clinical trial, long after a single transplantation. In fact, the bulbar subscale, that includes assessment of swallowing, was the subscale most improved after MSC-NTF treatment in rapid progressors, suggesting that the late decline in motor function was not an adverse effect of treatment per se. Hence the need for repeated dosing.

Last week, the DSMB recommended continuation of the phase 3 trial without any modification. This was major good news, so we asked him about this. Mr. Lebovits said that this was a second interim safety review, and there were no significant safety concerns. Therefore, the DSMB recommended no modification in protocol and no other interim analysis is planned. Phase 3 data will be available by mid-2020 according to this interviewer's reading of the press release.

Now we moved on to another critical aspect of our analysis - funds, or rather, the lack of it. Since this is an important issue, here's the exact exchange we had.

Dr. Ashok Dutta: How does the company plan to fund its operations through the next couple years until the lead development candidate is approved and commercialized? Given the weak financial position, does Brainstorm see the possibility for ATM operations, or thinks about selling rights in regions like China, Japan or Europe to increase the financial condition?

CEO Chaim Lebovits: As you are aware we do receive proceeds from the hospital exemption pathway and also receive grant funding from CIRM and IIA. These avenues have allowed to fund and continue with our trials over the years with non-dilutive financing. From a business standpoint as our ALS phase 3 trial is now fully enrolled, the management team continues to hold high level conversation with some of the leading global pharmaceutical and biotechnology companies. We are actively engaged in strategic partnering and collaboration discussions and although we cannot disclose the details of our conversations due to NDAs we signed with them... we are exploring several opportunities with key interested parties to advance the opportunities for NurOwn development and commercialization. As you have rightly pointed out, we have a $20mm ATM facility in place with Raymond James. We may activate the ATM as required and raise up to $20mm by selling our stock "at the market" only if the prices are attractive to us. So far as of end of Q3'19, we have not activated the ATM. If the need arises and the prices are attractive to us, we may employ this tool to raise capital.

This is reassuring that the company intends to focus on non-dilutive financing. The ATM facility, coupled with the grants, should ideally see them through the approval phase. We still wonder how they will manage marketing and sales. Perhaps those commercialization NDAs they have signed will help.

Next, we discussed market potential and a question about a recent patent grant. The CEO's detailed responses can be found in the complete interview material.

The strong involvement of the ALS community impressed us previously, so we now asked the CEO about the recent roundtable convention they had with ALS advocacy groups. Since this will be important for the ALS community as a whole, here's Mr. Lebovits' entire response on the question:

Finally, we asked him what we ask everyone: Give us three simple and straightforward reasons why investors would be interested. Here's what he said:

Thanks to the ALS community for inspiring us to conduct this interview, and to Mr. Chaim Lebovits, CEO of Brainstorm Cell Therapeutics, for answering our questions.

Thanks for reading. At the Total Pharma Tracker, we interview management of important small biotech doing disruptive work in healthcare. Our members are given exclusive access to these interviews, which helps them with additional primary resource in doing DD on their investments. Sometimes, extracts from these interviews may be published for everyone; but TPT members always get the exclusive view.

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

Additional disclosure: General Disclaimer - This is to confirm that Avisol Capital Partners has neither requested, nor been offered, any monetary compensation for conducting this interview, by any party other than Seeking Alpha.

Also to be noted, this was an emailed questionnaire, and certain editorial material is present in this version, which may or may not reflect BCLI or its CEO's position on the issues discussed.

View original post here:
Interview With Chaim Lebovits, CEO Of Brainstorm Cell Therapeutics - Seeking Alpha

Recommendation and review posted by Bethany Smith

MULTIPLE sclerosis campaigners have hailed a huge step forward for patients in Scotland after a stem cell therapy was recommended for use on the NHS for the first time.

Haematopoietic stem cell transplantation (HSCT) has been described as a game-changer for MS after an international clinical trial showed that it could reboot patients immune systems and halt the progress of the disease.

Some patients who had been in wheelchairs prior to treatment said their condition improved so dramatically it was like they had never been diagnosed with MS.

READ MORE: Scots MS patients 'missing out' on pioneering stem cell treatment available in England

The Scottish Health Technologies Group (SHTG) said there is now sufficient evidence for it to recommend making HSCT available on the NHS in Scotland to MS patients who have the relapsing-remitting form of the disease, and who were not responding to drug treatments.

Iain Robertson, chairman of the SHTG, said: Our committee members were able to advise that this treatment should be considered for those with this particular type of MS who have not responded to treatment with disease-modifying therapies.

We hope that our advice will be of use in helping decide the best course of treatment for these patients.

The SHTG also stressed that patients must be made aware of the demands, risks and uncertainties of the treatment, which uses chemotherapy to wipe out patients' 'faulty' immune systems before replenishing it with a transplant of stem cells harvested from their own bone marrow.

It puts patients at high risk from infections, which can be fatal, but the theory is that the treatment works by enabling patients to 'reset' their immune system to stop it attacking the central nervous system as is the case in MS.

READ MORE: Anger of Scots MS patients travelling abroad for stem cell therapy available to some on NHS England

HSCT is not considered an effective treatment for patients with the progressive form of MS, however, as stem cells cannot regrow nerves or repair damaged myelin - the protective sheath which coats nerves.

It will also be unavailable to patients with relapsing-remitting MS who no longer show signs of inflammation on an MRI brain scan.

Scotland has one of the highest rates of MS in the world, but until now Scottish patients seeking HSCT have had to travel overseas to Mexico, Russia and Israel and bankroll their own private treatment at a cost of around 40-60,000.

It has also been available privately in London since 2017, but with a 100,000 price tag.

A small number of MS patients in England have been able to access the treatment on the NHS, however, because there are clinical trials into HSCT taking place at NHS hospitals in Sheffield and London.

Morna Simpkins, director of MS Society Scotland, said: The decision from SHTG to approve HSCT for the treatment of MS is good news and could help in the development of a clear pathway, for people who could potentially benefit, to access it.

We will push to ensure that this decision leads to real change for people with MS by continuing to engage with other groups to offer the treatments, including HSCT, which are right for them.

READ MORE: Stem cells help mother with MS make 'remarkable' recovery

The SHTG said eligible patients must have equal access to the procedures regardless of where they live, but it is unlikely all health boards will be able to provide it.

The MS Society wants a centre, or centres, of excellence set up where patients from across Scotland can be referred.

Lucy Clarke from the Scottish HSCT Network said the recommendation was "a huge step forward" for people in Scotland living with MS.

Ms Clarke underwent HSCT in Russia and credits it with substantially reversing her disability.

She added: This important decision supports HSCT as a treatment option where other treatments have failed. We will continue to push so that this treatment is available to people in Scotland who need it.

A Scottish Government spokeswoman said: We are grateful to the Scottish Health Technologies Group for this important work.

"NHS Boards are expected to consider their advice on technologies in the planning and provision of its services and clinicians are expected to follow their professional judgement, working within the management structure of their Board.

We will work closely with MS Society Scotland, other third sector bodies and the clinical community to consider what the Technologies Groups findings means for provision in Scotland, including the information that needs to be available to people about eligibility and risks.

Here is the original post:
Stem cell therapy approved for MS patients in Scotland - HeraldScotland

Recommendation and review posted by Bethany Smith

In September end, a good news greeted the biomedical world when a team led by Takanori Takebe at Cincinnati Children's Hospital Medical Center succeeded at growing a connected set of three organs: the liver, pancreas and biliary ducts, in the lab, from human stem cells. The findings were published in journal Nature.

While human organoids already provide a sophisticated tool for research, the connected set of three organs, for the first time, allow scientists to study how human tissues work in concert. This was dubbed as a significant step forward.

In October, another news came. At the annual meeting of the Society for Neuroscience, researchers said that brain cell clusters prepared in the lab- a type of organoid- show abnormal behaviour as compared to the normal brain cells.

They said that the cells in these clumps had ambiguous identities and made more stress molecules than cells taken directly from human brains. However, these abnormalities were found to be alleviated a little bit when the implanted into a more hospitable environment - a mouses brain.

What are organoids?

With the available technology, scientists can grow a group of cells in laboratories into three-dimensional, miniature structures that mimic the cell arrangement of a fully-grown organ.

This is done using stem cells.

Stem cells are special human cells that have the ability to develop into many different cell types, from muscle cells to brain cells.

The embryonic stem cells that are derived from unused embryos (These are created from an in vitro fertilization procedure and used for scientific research) are pluripotent, meaning, they can turn into any type of cell.

On the other hand are adult stem cells. They are derived from fully developed tissues, like the brain, skin, and bone marrow. These cells often have capability of turning into only certain types of cells. For example, a stem cell derived from the liver will only generate more liver cells.

However, the adult stem cells can be manipualted in the laboratory to act like embryonic stem cells. These are called induced pluripotent stem cells. (The technique was developed in 2006). However, scientists are yet to find adult pluripotent stem cells that can develop every kind of cell and tissue.

When scientists create right environment in the laboratory for them, these stem cells follow their own genetic instructions to develop into tiny structures that resemble miniature organs composed of many cell types.

Using these, researchers have been able to produce organoids that resemble the brain, kidney, lung, intestine, stomach, and liver etc.

For example, in the three-organ research mentioned above, Dr Takebe started with stem cells from human skin cells and then guiding and prodding those stem cells to form two very early-stage "spheroids" of cells loosely termed the foregut and the midgut (In human embryos, these form late in the first month of gestation. Over time, they merge and morph into the organs that constitute the digestive tract).

The spheroids were first placed next to each other in a lab dish suspended in a gel used to support organoid growth, then placed on top of a thin membrane that covered a carefully mixed batch of growth medium.

From this point on, the cells knew what to do, and 70 days later, the mini organoids began processing bile acids as if they were digesting and filtering food.

Why are organoids important?

The technique to develop organoids was named by The Scientist as one of the biggest scientific advancements of 2013.

Organoids are an excellent tools to study biological processes like uptake of nutrients, drug transport, secretion of hormones and enzymes etc. This way, diseases related to malabsorption of nutrients, and metabolism-related diseases like obesity, diabetes, insulin resistance can be studied at the cellular-level.

Recently, scientists at the at Memorial Sloan Kettering created a tumor organoid to develop a more accurate rectal cancer model.

In the case of the human brain, organoids opens a window to understand some of the most complicated and hidden aspects of our own biology. They can be used to study neuropsychiatric or neurodevelopmental diseases like schizophrenia or autism spectrum disorder, which are uniquely human diseases that affect the whole human genome.

Organoids also provide a window into how cells interact with each other and their environment. They can be used to create cellular models of human disease, which can be studied in the laboratory to better understand the causes of disease and identify possible treatments. The effects of different drugs and be tested.

Scientists have even used gene editing techniques (CRISPR-Cas9) on the stem cells to to introduce targeted mutations in genes corresponding to two different kidney diseases. When these modified pluripotent cells grew into human kidney organoids, they exhibited the diseases.

Using such organoids relieves the scientific community from experimenting on human and animal subjects. Also, certain treatments that would be unethical to administer on the latter, can be tested on the organoids.

With organoids, researchers can produce a limitless supply of tissue from each patient. This will also be extremely useful for the study of rare diseases, where the number of patients on which to conduct research and test treatments is limited.

Organoids are also being used to develop personalised and precision medicine.

For example, it was found that repairing the CFTR protein could give relief to a patient suffering from non-cystic fibrosis, an inherited disease caused due to a gene mutation. Using the Intestinal organoids grown from a patients stem cells, the doctors could quantify the patients response to the CFTR modulating therapy.

Organoids can have significant therapeutic applications. For example, pluripotent stem cells derived from a diabetes patient could be transformed into insulin-producing beta-like cells.

Organoids also offer an incredible opportunity to study developmental biology. Using them, for example, we can learn more about how organs are formed in embryonic stages and associated disorders.

Visit link:
Growing Human Organs In A Lab: As Scientists Develop Pathbreaking Three-Organ System, Heres All You Need To Know - Swarajya

Recommendation and review posted by Bethany Smith

WHEN Laura Farmer-Maia's daughter suddenly became clingy and unhappy, she initially brushed it off as nothing but "a phase".

And given little Beatriz was just three-years-old, the last thing to cross her mind was cancer.

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Yet, months after doctors repeatedly dismissed the symptoms as clinginess, the diagnosis was confirmed - Beatriz had an aggressive childhood cancer known as a neuroblastoma.

The horrifying news came after Beatriz's father Tiago Maia refused to leave the hospital after discovering the potential diagnosis himself on Google.

Shocked, Laura, 39, and Tiago, 40, are now urging all parents to be vigilant and check their kids for signs of the disease.

The mum, who works in advertising, first suspected something was wrong last July, when Beatriz's behaviour dramatically changed.

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She said: "Before she was diagnosed, Beatriz was quite naughty but when she reached two, she suddenly became clingy and picky with her food, and had a fever all the time.

"We took her to the GP who believed it was a virus and after recurrent visits they gave her some antibiotics to cover for a potential bacterial cause, which didnt have any effect.

"Beatriz started to complain that her legs hurt so we took her to A&E, where they did some more tests and still said it might be a virus.

"Its hard to get a diagnosis right when a child is too young to explain how theyre feeling, but in the back of our minds we knew it was something bad.

"We want to spread awareness of the difficulty of diagnosing cancer in young children - if your child doesnt seem right, you should push for further tests."

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Tiago pushed the GP for more tests, and blood tests showed something was wrong, so Beatriz was sent urgently to hospital.

It was there medics finally discovered a lump above her kidney and diagnosed her with neuroblastoma in September last year.

The cancer is aggressive and has a 40 per cent chance of long-term survival.

Everything moved so quickly and we all felt frightened as they carried out the tests

Tiago, originally from Portugal, added: "At the hospital, they twice said it was likely to be a virus and I refused to leave until I saw a specialist.

"I waited for three hours until a more senior doctor was free, and then Beatriz was examined by different specialists who admitted her to do all kinds of tests and observations including X-rays and ultrasounds - it was the last one that confirmed there was a lump.

"When my fears from Google turned out to be true, it was very strange because even though my life had just flipped upside down, I was almost relieved to be right - it was weird and confusing."

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Beatriz was referred straight to Great Ormond Street Hospital in London, where they carried out further tests including scans, blood tests and biopsies.

She began chemotherapy just a week after being diagnosed and underwent eight gruelling rounds of chemo over the next 18 months.

Doctors then carried out a stem cell transplant to regenerate bone marrow destroyed by high dose chemo, which meant Beatriz couldnt leave the hospital for eight weeks.

Tiago, a design director, said: "I was quite scared when Beatriz was diagnosed because my mum and dad had only recently died from cancer I thought of the worst.

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"Everything moved so quickly and we all felt frightened as they carried out the tests.

"Doctors found that the cancer had spread across her body, so she began chemotherapy just weeks after being diagnosed.

"We were told the treatment would last 18 months which was a massive shock to us.

"A week after Beatriz started chemotherapy she massively improved, but it was tricky being in hospital at first.

"Now, she still has periods of discomfort but sometimes shes happy to be in hospital because she has toys and people to come and play with her."

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Laura added: "The stem cell transplant was a hard time for us all because we had to spend a lot of time apart from our other daughter, Clara, six.

"Beatriz was diagnosed in Claras first week of school and it was difficult because that was supposed to be an exciting time for her."

After more scans and hopes of an all-clear, doctors found more metastatic growths still remaining in Beatrizs head, which meant that the cancer hadnt fully cleared up and she had relapsed.

The brave youngster is now undergoing immunotherapy and is due to start a six month medical trial on the NHS at Great Ormond Street Hospital, called the Beacon Trial.

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What is neuroblastoma?

Neuroblastoma is a type of cancer that most commonly afflicts babies and young children.

The disease develops from special nerve cells, known as neuroblasts, which get left behind from the child's development in the womb.

It mostly begins in the sufferer's adrenal glands located above the kidneys but can occur in the nerve tissue that runs along the spinal cord in the neck, chest, abdomen or pelvis.

The vicious illness can then spread to other organs like the bone, bone marrow, lymph nodes and skin.

Neuroblastoma afflicts around 100 children a year in the UK but the cause of the disease is still not known.

Its symptoms can include:

It is uncertain whether the trial will work and, even if Beatriz goes into remission, relapse rates are high but her parents are determined to do everything they can to stop the cancer from returning.

Laura and Tiago are now trying to raise 200,000 to help get their daughter into remission or to keep the cancer away if her treatment goes well.

The money is hoped to go towards further treatment, or if Beatriz gets the all-clear, a special vaccine in New York which helps keep the disease away.

Laura said: "After the stem cell transplant, the end was almost in sight but then she relapsed.

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"Were afraid that the cancer will get worse and worse and want to raise money to help get her into remission the ideal outcome is that the trial works and clears the disease.

"Luckily, compared to other two-year-olds, Beatriz has suffered less side effects with treatment and despite losing her curly hair shes powering through."

You can donate on Beatriz's JustGiving page here.

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Here is the original post:
Docs said our toddler was just clingy but we learned the truth on Google it was cancer - The Sun

Recommendation and review posted by Bethany Smith

PEORIA, Ill. November is National Bone Marrow Awareness Month.

The Greater Peoria Family YMCA is hoping you take the time to see if youre a match and it comes with a local tie.

Marsha Krone has been a teacher in the Peoria area for over 30 years.

Despite being diagnosed with a rare bone marrow cancer in 2016, shes yet to find a match.

You never know when youre going to be the link to saving someones life and its very simple. You come out, fill out a little paperwork online. You swab the inside of your cheek. You put it back on the cart and its sent back.

You can join the donor registry for bone marrow & blood stem cells this Wednesday, Nov. 6 from 3 p.m. 7 p.m.

It would be awesome to find a match for me, but if I dont, there are other people that their lives can be saved through that, said Krone . Their joy would certainly not be pain for me. I would be happy for them to find their cure and what a gift to be able to give someone.

The YMCAs is located 7000 North Fleming Lane, Peoria, IL 61614. The registry will be held in the upstairs lounge.

See the original post:
You can be the match, help local people in need Wednesday - CIProud.com

Recommendation and review posted by Bethany Smith

The proposal was nothing fancy. Shawn Russell arrived at the pub, placed a pint on the table in front of Sarah Hodgetts and asked her to marry him.

It was out of the blue, with no ring, nothing. And I thought: Yeah, thats a really good idea, we should just get married, says Sarah. Id been in love with him for ages.

Their story began seven years earlier, in 2009, also in a pub in north London. He was sitting in his flat cap, good Yorkshireman that he was, and was reading the sports pages. I had just got off the Tube after work. From my perspective, it was love at first sight, says Sarah.

But dating wasnt easy. Shawn, a boarding school-educated boy from an Army family, who had lost his mother to leukaemia when he was two, worked as a picture editor at The Telegraph. Although Sarah was down the road in Westminster, where she worked as a civil servant (and still does), their hours were long and their schedules largely incompatible. They managed just six months initially.

It was a disaster trying to date, so we ended up with a firework display of an argument and decided there was no way we could, says Sarah over coffee near her office.

Their split didnt last, however; they had far too much in common. Besides his ridiculous sense of humour, Shawn was so into news and politics, and I worked in politics, so we couldnt not communicate. We realised we were incredibly good friends, says Sarah. Plus, she adds with a smile, he was a very attractive, 6ft 4in blue-eyed man who I was completely smitten with.

Over time, they got back together in a non-committal way, sharing weekends when their busy lives permitted. It was during this period, three-and-a-half years ago, that Shawn, then 44, proposed. The following week, he moved in with Sarah, then 41, and her son Eddy, 10, from a previous relationship, in Kings Cross, and, like all newly engaged couples, they started making plans for their future. A pair of recovering workaholics, they were going to transform their lives.

Originally posted here:
I lost my fianc to leukaemia, but in my dreams hes just working the night shift - Telegraph.co.uk

Recommendation and review posted by Bethany Smith

SAN FRANCISCO--(BUSINESS WIRE)--

Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for malignant and life-threatening diseases of the bone marrow, today announced the appointment of James D. Watson as Chief Business Officer.

James has a long history of success in biotech financing, business development, and commercial planning. That experience will guide the strategic direction and growth of Imago, said Hugh Young Rienhoff, Jr., M.D., Chief Executive Officer of Imago BioSciences. His experience in corporate development and financings will help ensure the advancement of bomedemstat (IMG-7289) through clinical development in myelofibrosis and other indications.

Mr. Watson most recently served as Chief Business Officer at Sigilon Therapeutics where he closed a $473 million strategic partnership with Eli Lilly for a treatment for Type 1 diabetes. Prior to Sigilon, Mr. Watson was Chief Business Officer for Alvine Pharmaceuticals and led strategy, corporate development, new product planning, and finance during which he closed a transaction giving AbbVie the right to acquire Alvine for $345 million. Previously, Mr. Watson was CEO of a San Francisco-based, boutique investment bank focused on mergers, acquisitions, partnering, and raising capital for life science companies.

I am excited to join Imago BioSciences at this important stage of growth, helping the company realize its full potential. Bomedemstat has great promise for the day-to-day management of myelofibrosis and it offers the additional possibility of altering the course of this disease. Furthermore, its broader myeloproliferative neoplasm platform and expertise in life-threatening diseases of the bone marrow represent an opportunity to address areas of high unmet clinical need and to build a valuable company. said Mr. Watson.

About Bomedemstat (IMG-7289)

Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of growth factors and cytokines that drive myelofibrosis pathogenesis.

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat for the treatment of myelofibrosis which is currently being studied in an international Phase 2b study. Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, private therapeutics company focused on malignant and life-threatening diseases of the bone marrow. The initial clinical focus is on myeloproliferative neoplasm (MPN) disorders including myelofibrosis, essential thrombocythemia and polycythemia vera. Investors in Imago include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191104005265/en/

Link:
Imago BioSciences Further Expands Executive Team with Appointment of James D. Watson as Chief Business Officer - Yahoo Finance

Recommendation and review posted by Bethany Smith

CHICAGO In a cohort of adults who received a thyroid function test during hospitalization, most did not have a thyroid disease and those who did generally had mild conditions, according to findings presented at the annual meeting of the American Thyroid Association.

Ruaa Al-Ward

Thyroid function tests are very frequently ordered in the hospitalized patients, Ruaa Al-Ward, MD, a hospital medicine senior associate consultant at Mayo Clinic in Rochester, Minnesota, told Endocrine Today. When abnormal, they are likely related to established diagnosis of thyroid disorders. However, in patients with no prior history of thyroid disorder, thyroid abnormalities resolved in two-thirds of the cases upon follow-up, therefore observation should be considered for the majority of these patients.

Al-Ward and colleagues analyzed data from 116 adults (mean age, 63.8 years; 50% women) who received a thyroid function test between January 2018 and January 2019 at Mayo Clinic. An abnormal test was required for all participants as was a lack of thyroid disorder history, although the researchers noted that risk factors for such disorders were present in 32% of the cohort.

Researchers identified thyroid disease in 35 of patients, with 31 having hypothyroidism and 23 having minimally elevated thyroid-stimulating hormone. Among the total cohort, 82% had a TSH level between 4.3 mIU/L and 10 mIU/L, 9.4% had a TSH level of more than 10 mIU/L and 7.7% had a TSH level below 0.2 mIU/L. According to the researchers, an arrythmia accounted for 22% of the tests, whereas 39% of requests came from ED providers who the researchers labeled as the group that most commonly ordered tests.

Although it is widely known that acute illness can affect thyroid function test, TSH is still commonly ordered. Physicians should keep in mind that, most of the time, thyroid testing is normal and not the explanation for the patients problem, and when the abnormalities are present they tend to be mild and they resolve during follow-up without any specific thyroid intervention, Al-Ward said. Thyroid testing should only be pursued when there is a high likelihood of thyroid disease. by Phil Neuffer

Reference:

Al-Ward R, et al. Poster 340. Presented at: 89th Annual Meeting of the American Thyroid Association; Oct. 30-Nov. 3, 2019; Chicago.

Disclosure: Al-Ward reports no relevant financial disclosures.

Continue reading here:
Thyroid function tests frequently yield no evidence of thyroid disease - Healio

Recommendation and review posted by Bethany Smith

The Fulton County Health Department has scheduled the following health clinics and services.

CANTON The Fulton County Health Department has scheduled the following health clinics and services. Please call the number listed with each service for an appointment or more information.

Maternal child health: Health screenings, WIC nutrition education and supplemental food coupons for women, infants and children. To make an appointment or for more information call 647-1134 (ext. 254). For Astoria clinic appointments call 329-2922.

Canton - Clinic - Monday, Nov 4 - 8-4 - Appt needed

Canton - WIC Nutrition Education - Tuesday, Nov 5 - 8-4 - Appt needed

Canton - Clinic/Immunizations - Tuesday, Nov 5 - 4-7 - Appt needed

Canton - Clinic/Immunizations - Wednesday, Nov 6 - 8-4 - Appt needed

Astoria - Clinic, WIC Nutrition Educ. - Wednesday, Nov. 6 - 9-3 - Appt needed

Canton - Clinic - Thursday, Nov 7 - 8-4 - Appt needed

Adult Health Immunizations: Various vaccines are available. There is a fee for immunization administration. Medicaid cards are accepted. To make an appointment or for more information call 647-1134 (ext. 254).

Canton - Immunizations - Tuesday, Nov 5 - 4-7 - Appt needed

Canton - Immunizations - Wednesday, Nov 6 - 8-4 - Appt needed

Other times available by special arrangement at Canton, Cuba and Astoria.

Blood Lead Screening: Blood lead screenings are available for children ages one to six years. A fee is based on income. To make an appointment or for more information call 647-1134 (ext. 254). For Astoria appointments call 329-2922.

Canton - Clinic - Wednesday, Nov 6 - 8-4 - Appt needed

Family Planning: Confidential family planning services are available by appointment at the Canton office for families and males of child-bearing age. Services provided include physical exams, pap smears, sexually transmitted disease testing, contraceptive methods, pregnancy testing, education and counseling. Services are available to individuals of all income levels. Fees are based on a sliding fee scale with services provided at no charge to many clients. Medicaid and many insurances are accepted. After hours appointments are available. To make an appointment or for more information call the 647-1134 (ext. 244). *Program funding includes a grant from the US DHHS Title X.

Pregnancy testing: Confidential urine pregnancy testing is available at the Canton and Astoria offices. This service is available to females of all income levels. A nominal fee is charged. No appointment is needed. A first morning urine specimen should be collected for optimal testing and brought to the health department. Services are provided on a walk-in basis on the following days each week:

Canton: Every Wednesday & Thursday, 8-3:30 (for more information call 647-1134 ext. 244)

Astoria: Every Wednesday, 9-2:30 (for more information call 329-2922)

Womens Health: A womens clinic for pap tests, clinical breast examinations and vaginal examinations is available by appointment. There is a nominal fee for this service. Medicaid cards are accepted. Financial assistance is available for a mammogram. Cardiovascular screenings may be available to age and income eligible women. To make an appointment or for more information call 647-1134 (ext. 244).

Mammograms: Age and income eligible women may receive mammograms at no charge. Speakers are available to provide information to clubs and organizations. For more information or to apply for financial assistance, call 647-1134 (ext. 254).

Mens Health: Prostate specific antigen (PSA) blood tests are available for men for a fee. To make an appointment or for more information call 647-1134 (ext. 224).

Sexually Transmitted Disease (STD) Clinic: Confidential STD and HIV testing services are available by appointment to males and females at the Canton office. Services include physical exams to identify STDs, a variety of STD testing, HIV testing, education, counseling, medications and condoms. There is a nominal fee for services. Services are available to individuals of all income levels. Medicaid cards are accepted. To make an appointment or for more information call 746-1134 (ext. 224).

HIV Testing and Counseling: Confidential HIV testing and counseling services are available by appointment through the sexually transmitted disease (STD) clinic at the Canton office. To make an appointment or for more information call 647-1134 (ext. 224).

Tuberculosis (TB) Testing: TB skin tests are available at no charge by appointment. To make an appointment or for more information call 647-1134 (ext. 254).

Blood Pressure Screenings: The Fulton County Health Department provides blood pressure screenings at no charge on a walk-in basis during the following times:

Cuba - Screening - Monday, Nov 4 - 8-12 - Walk in

Astoria - Screening - Wednesday, Nov 6 - 9-12 - Walk in

Health Watch Wellness Program: The Health Watch Program provides low cost lab services. Through this program adults can obtain venous blood draws for a variety of blood tests. Blood tests offered without a doctors order Comprehensive Metabolic Panel (CMP), Complete Blood Count (CBC), Lipid Panel, Prostate Specific Antigen (PSA) test, Hepatitis C test, and Thyroid Stimulating Hormone (TSH). A wide variety of blood tests are also available with a doctors order. There is a charge at the time of service. To make an appointment or for more information call 647-1134 (ext. 254).

Canton - Clinic - Monday, Oct. 28 - 8-12 - Appt needed

Dental Services: The Dental Center offers a variety of basic dental services to children and adults. An appointment is needed. Medicaid and Kid Care cards are accepted. To make an appointment or for more information call 647-1134 (ext. 292).

See the original post:
Health Department announces services for the week of Nov 4 - Orion Gazette

Recommendation and review posted by Bethany Smith

What is diabetes?

Diabetes is a lifelong health condition in which the bodys levels of blood glucose and the hormone insulin are out of balance. Symptoms include increased thirst, increased frequency of passing urine, and fatigue. There are two main forms:

Type 1, in which the body doesnt produce enough insulin.

Type 2, where either the body doesnt produce enough insulin, or the bodys cell no longer react to the insulin produced.

An inability to produce insulin, or use it effectively, results in raised levels of blood sugar (hyperglycaemia). If present over a prolonged period, hyperglycaemia is associated with damage to organs and tissue within the body including the heart, blood vessels, nerves, kidney and eyes.

The risk factors for Type 1 diabetes are still being researched, but several have been identified for the commonest form, Type 2. These include a family history of the disease, being overweight, physical inactivity, and unhealthy eating.

What is a podiatrist?

A podiatrist is an important part of a team of health professionals who manage complications associated with diabetes. Diabetes can affect circulation, nerve sensation (feet go numb or tingly), skin health, healing of wounds, and fighting infections. If you have no other complications with your diabetes, an annual foot exam can help detect problems early. Pressure points that turn into calluses, can result in foot ulcerations and infections.

Ingrown or fungal toenails, can lead to infections as well. Some patients require foot care every 2-3 months, to avoid problems with their feet, and closely monitor for problems.

Podiatrists can assess shoes, and even prescribe shoes and orthoses for your feet. They will help prevent the complications, along with accommodating deformities like hammertoes and bunions. If you have had a wound of some kind on your foot, it can also be prevented from recurring.

How does diabetes link to podiatry?

Foot complications in diabetes are common, accounting for more hospital admissions than any other diabetic complication. Foot ulcers present as one of the most significant pathologies, and are associated with neuropathy (nerve damage), and/or peripheral arterial disease (poor circulation). These greatly increase the risk of amputation, with up to 80 per cent of amputations attributed to foot ulceration.

The prognosis for individuals with ulceration and amputation is poor, with a five year mortality rate of 43-55 per cent, and up to 74 per cent respectively. Podiatrists play a leading role in the management of ulceration.

They provide treatments, including wound debridement, dressing and pressure relief. It has been suggested that 80 per cent of amputations are potentially preventable, through the provision of well structured, quality care.

Why is

awareness important?

The consequences of diabetes manifest slowly over a period of time. As a result, by the time they occur, it is too late, and the focus of treatment is the prevention of ulceration, and the subsequent fallouts.

It is why patient education is so important from the outset, and should be an ongoing process. Optimising blood glucose control is key; by providing regular foot checks, it can be reinforced, serving as a warning to those who are developing the complications.

Every person with diabetes, should pay careful attention to the systems of the body that can be affected directly and adversely These are the cardiovascular system, the renal system (kidneys), the eyes, and feet. It is recommended that every patient with diabetes have at least an annual check on all of these systems.

As far as the feet are concerned, people with diabetes can have severe, even life-threatening foot problems. This can happen because of a snowball effect the disease can have on the circulatory and nervous systems of the body. Essentially, diabetes can cause:

decreased foot blood flow

decreased healing potential

decreased infection fighting capability

numbness of the feet

These problems can all come together in a diabetic foot, causing drastic results.

It is entirely possible, for example, that a diabetic could step on a nail, and being numb, not even be aware.

Then follows a severe infection (which the person may not be able to fight), decreased healing potential, decreased blood flow, and problems can spiral quickly.

Often this patient may not even realise he/she stepped on a nail for a few days; by then, a severe infection has set in, and the problem is serious.

Every diabetic should have a podiatrist, whom those with numbness in the feet, should see on a regular basis each year. These problems are preventable, and are not inevitable. Regular check-ups with your podiatrist can prevent problems; as well, nail and callus trimming can be done, if you cannot manage same at home.

November is World Diabetes Month, so stay tuned, and look out for varied topics.

Your feet mirror your general health . . . cherish them!

Leana Huntley is a UK trained Podiatrist attached to Almawi Limited The Holistic Clinic, and Clinical Director-Fit Feet, Special Olympics Trindad and Tobago. E-mail: info@almawiclinic.com or visit the website at http://www.almawiclinic.com.

Read the original:
Diabetes and podiatry...the connection | Features Local - Trinidad & Tobago Express Newspapers

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SEATTLE, Oct. 31, 2019 /PRNewswire/ RareCyte announces an addition to the RarePlex Staining Kit product line, enabling customers to evaluate HER2 and ER expression on circulating tumor cells (CTCs) in their own laboratory. HER2 and ER are biomarkers that direct treatment recommendations for invasive breast cancer, and liquid biopsy offers a blood-based method to evaluate biomarker expression for insight into receptor status, response to treatment, and potentially therapy selection in clinical research.The RarePlex HER2/ER CTC Panel Kit includes all the reagents necessary for immunofluorescent detection of CTCs along with HER2 and ER expression. When combined with the RareCyte platform for CTC analysis, the RarePlex HER2/ER CTC Panel Kit enables the first blood-to-result breast-specific CTC assay deployable in customer laboratories.The HER2/ER Panel Kit was validated based on rigorous requirements set to clinical standards with guidance from CLIA and industry experts. Tad George, PhD, Sr. VP of Biology R&D at RareCyte noted Our approach to CTC assay development and validation is centered on creating deployable products that combine the sensitivity, specificity, and precision required for multi-center trials, not only for CTC enumeration but also for classifying CTCs based on their biomarker phenotypes.Dr. Minetta Liu, MD, Professor and Research Chair in the Department of Oncology at Mayo Clinic recently presented her work with the HER2/ER CTC Panel Kit at the Advances in Circulating Tumor Cells Conference in Corfu, Greece. Our early work with this assay has revealed interesting patterns of HER2/ER expression that vary widely across patients. Efforts will now focus on defining thresholds for HER2 and ER positivity that are specific to CTCs. This platform will facilitate important investigations into the clinical significance of CTC heterogeneity relative to standard tissue-based definitions of HER2 positive and/or hormone receptor positive advanced breast cancer.The HER2/ER Panel Kit is available for purchase and more information on the HER2/ER Panel Kit and the RareCyte platform is available at rarecyte.com.RareCyte products are for research use only. Not for use in diagnostic procedures.About RareCyte, Inc.RareCyte offers next generation instruments, consumables and reagents for the analysis and capture of rare cells from blood and tissue, enabling its foundational liquid biopsy and tissue analysis platform. The company has deep experience in developing advanced precision life science systems used in cutting-edge labs worldwide. Our customers perform innovative research, bring new therapeutics to market, and perform a wide-range of single cell applications in oncology, prenatal testing, and infectious disease. For more information about RareCyte, visit rarecyte.com.

FOX28 Spokane

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RareCyte expands liquid biopsy offering with the release of a HER2/ER breast cancer CTC Panel Kit - FOX 28 Spokane

Recommendation and review posted by Bethany Smith

More than 20 million Americans have some form of thyroid disease, according to the American Thyroid Association, but up to 60 percent don't know it.

A simple blood test to check your thyroid's hormone levels is all that's needed to find out if you have this disease.

The thyroid is a butterfly-shaped gland in the neck that can have a dramatic impact on your body. It controls your metabolism, regulates your body temperature, keeps your heart pumping, and so much more.

So thyroid disease can occur in one of two ways: you can either have a high-functioning thyroid or hyperthyroidism, or you can have a slow-functioning thyroid or hypothyroidism, said Amistad Community Health Center internal medicine physician Dr. Jacqueline Phillips.

Dr. Phillips said more than 12 percent of Americans will develop an underactive thyroid or an overactive thyroid.

Symptoms you will have if you have hyperthyroid is a rapid weight loss; you will feel nervous, you will have palpitations, or you will feel your heart beating irregular," Dr. Phillips said. "You will also have some diarrhea, and be hot all the time.

"Hypothyroidism is when your thyroid is acting slow and will have opposite symptoms. So people with hypothyroidism will tend to gain weight, feel cold most of the time, you will have hair loss, dry skin, and you will just overall feel tired and fatigued."

People of all ages and races can get thyroid disease. However, women are five to eight times more likely than men to have thyroid problems. In fact, 1 in 8 women will develop thyroid problems during her lifetime.

Your doctor will check two labs, the first lab is TSH and the second lab is a free T4," Dr. Phillips said. "Based off your levels, you will either be diagnosed of having too much thyroid or too little thyroid. If you have too little thyroid (hypothyroidism), it is replaced with a small tablet that contains thyroid hormone. If you have too much thyroid (hyperthyroidism) then what you will need to do is take a medication that will help your thyroid stop making hormone, and then they will refer you to an endocrinologist for further treatment."

Thyroid disorders are fairly common in adults. Fortunately, nearly all thyroid problems can be managed successfully when identified early.

Prevention would be with regular screening, and especially if you are having any symptoms of either hyperthyroidism or hypothyroidism, that you talk to your doctor, and you get the appropriate blood work done, said Phillips.

Dr. Phillips says you should really see your doctor if you are having rapid weight gain or rapid weight loss.

Consider seeing your primary care provider if signs and symptoms of hypothyroidism or hyperthyroidism are present, or if a nodule is noted in the lower front sides of your neck. Your provider will perform a detailed medical history and physical exam.

Workup may include lab tests, X-rays and referral to an endocrinologist. If surgery is needed, you will be referred to an ear, nose and throat, or general, surgeon with expertise in performing thyroid procedures to evaluate and talk with you about your options.

The following are symptoms for hypothyroidism:

The following are symptoms for hyperthyroidism:

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Local News Blood tests are all that's needed to detect thyroid disease Roland Rodriguez 6:33 - KRIS Corpus Christi News

Recommendation and review posted by Bethany Smith

The population of women in Duluth over 45 is estimated by the U.S. Census Bureau at 28,000.

Women most affected by menopausal symptoms are between 45 and 64. My mother, aunts, and grandmother are all of this age and all have weathered their menopausal changes, some with the aid of prescription hormone replacement therapy.

A shared experience is of a healthy, shift-working woman, 45 years old, struggling with significant night sweats and insomnia for three months. She makes an appointment with her primary-care provider, who has been seeing her regularly for health care for the last few years, since after the birth of her daughter. She previously had regular care with her OB/GYN doctor but felt too old for her follow-up routine visits. So she transitioned to a local primary-care provider. At this visit, she hoped to receive some aid for her bothersome symptoms.

I dont have the luxury to be this tired all the time from not sleeping due to these horrible night sweats, she said to her doctor. I need to be efficient at work so I can provide for my family. Is there something I can take or do?

Her primary replied, I have to refer you to an OB/GYN, because I do not have the expertise to manage patients with hormone replacement therapy, which is a treatment that can help you.

She is frustrated because she took off work for this visit and now has to make another visit in the OB/GYN office in three weeks and has an additional drive of 35 minutes. But she complies.

At her next visit, she is struck with how old she feels, surrounded in the waiting room by young expecting mothers and their buzzing children. She meets with the OB/GYN provider, who spends a brief seven minutes with her, and she leaves feeling very comfortable with a new prescription of hormone replacement therapy.

The problem is this common treatment of hormone replacement therapy, or HRT, is not routinely instructed to primary-care providers in their mandatory continuing-education requirements, particularly in low-risk women. Primary-care providers are required to have continued education as part of their licensure to practice medicine.

These providers serving female patients include medical doctors, nurse practitioners, advanced-practice registered nurses, and physician assistants. This problem leads to increased costs and referrals for low-risk patients and a loss of revenue for providers who have the capacity to manage this highly effective and safe therapy for close to half of their patient population.

As a womens-health nurse practitioner, I have heard this common story from many female patients, and it is time for the health care system to listen. All primary-care providers need regular, mandatory continuing education on hormone replacement therapy treatments. This mandatory education should include the large body of evidence that supports the safe use of HRT in menopausal women and the important risk factors when HRT is not appropriate.

HRT can be counseled, prescribed, and managed in low-risk women by primary-care providers.

Regionally, the practice of managing HRT is diverse and is very typical practice in urban settings while much less common practice in rural areas where primary-care providers are pivotal.

My primary-care colleagues continue to ask how to treat female patients who have multiple high-risk factors and who shouldnt be on HRT. My rebuttal is that their keen knowledge is correct: HRT is likely not the treatment for these female patients. A referral to an expert in womens health is highly encouraged for high-risk women. The population of high-risk women is typically amongst younger ages or is found during pregnancy or through routine surveillance in all care settings.

With mandatory continuing education for primary-care providers in managing safe hormone replacement therapy for women, their confidence in the medications and the management of these low-risk women will increase, along with provider satisfaction from patients.

Kelsey Thompson of Duluth is a womens-health nurse practitioner in Minneapolis. She received a bachelors degree in nursing from the College of St. Scholastica. She earned a masters in womens health and a doctorate in nursing from Duke University in North Carolina.

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Nurse's View Column: Health care system failing the women in our families - Duluth News Tribune

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