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Child dies in US during gene therapy trial – The News International

Posted: October 16, 2020 at 8:49 pm

PARIS, France: A five-year-old girl with a rare neurodegenerative disease died in the US while taking part in a gene therapy trial run by French biotechnology company Lysogene, the firm said on Thursday.

The little girl was suffering from Sanfilippo syndrome or mucopolysaccharidosis type III, a rare genetic disease that alters brain development after birth and leads to premature death. In a statement Lysogene, a company developing gene therapy for central nervous system diseases in children, said "the immediate cause of death is currently unknown" and that there was as yet "no evidence that the event is linked to the study drug administration".

It said it was "profoundly saddened by the passing of this child" and was collecting "additional information" about the circumstances. The share price of the company dropped 19 percent to 2.05 euros in morning trading in Paris.

The girl was one of 19 people being treated in the trial conducted at eight hospitals in Europe and the United States. She died at home several months after receiving the therapy, consisting of a single injection, at one of four treatment sites in the US, Lysogene told AFP. In its statement the company said it was following the remaining 18 patients closely and remained "committed to the LYS-SAF302 development program".

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Foundation to Fight H-ABC, University of Massachusetts Medical School and Yale University Initiate Gene Therapy Study Targeting Cure for Rare Disease…

Posted: October 16, 2020 at 8:49 pm

ROCKVILLE, Md., Oct. 13, 2020 /PRNewswire/ --Foundation to Fight H-ABC, a non-profit organization dedicated to increasing awareness and driving development of a cure for the degenerative children's disease, H-ABC, today announced a sponsored research agreement with the University of Massachusetts Medical School and Yale University to advance a targeted gene therapy for H-ABC.

"We have high hopes to quickly prove efficacy with this approach to move research forward and find a permanent cure for this devastating disease," said Michele Sloan, Co-Founder, Foundation to Fight H-ABC.

H-ABC (hypomyelination with atrophy of the basal ganglia and cerebellum) belongs to a group of conditions called leukodystrophies, diseases that affect the white matter of the brain. These diseases disrupt the growth or maintenance of the myelin sheath, a protective layer that insulates nerve cells and allows for the transmission of messages between cells.

Caused by a mutation in the TUBB4A gene, H-ABC is a rare genetic disorder that affects certain parts of the brainspecifically the basal ganglia and the cerebellum, which control movement. H-ABC targets these important structures, reducing both their size and function. As a result, children who suffer from H-ABC often experience motor problems, cannot walk, talk, or sit on their own. Currently, there is no known cure for this disabling and life-threatening condition.

The teams of Dr. Guangping Gao (University of Massachusetts Medical School) and Dr. Karel Liem (Yale School of Medicine) will combine extensive expertise in the fields of Adeno-associated virus (AAV), a platform for gene delivery for the treatment of a variety of human diseases and H-ABC disease models, to develop AAV vectors to silence or outcompete the mutated TUBB4A gene.

"To date, AAV-based gene delivery system is the vector of choice for in vivo gene therapy of many currently untreatable rare diseases including H-ABC," said Guangping Gao, Ph.D. "We are very excited for starting close collaborations with Dr. Liem's team at Yale and the Foundation to Fight H-ABC to develop potential gene therapeutics for this devastating disease."

"With the support from the Foundation to Fight H-ABC, we are excited to build upon our mechanistic studies of the disease and to collaborate with Dr. Gao of the University of Massachusetts to develop and test AAV approaches to H-ABC," saidKarel F Liem Jr., M.D., Ph.D.

For more information, please visit https://www.h-abc.org/donate.

CONTACT: Sawyer Lipari, [emailprotected]

SOURCE Foundation to Fight H-ABC

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Stevenage named ‘high potential opportunity’ zone for cell and gene therapy – In Your Area

Posted: October 16, 2020 at 8:49 pm

InYourArea Community

We are delighted that Stevenage is being recognised for its significant contribution to the cell and gene therapy field."

Stevenage Bioscience Catalyst campus

Submitted by Charlotte Connors

The Department for International Trade (DIT) has classified Stevenage, Hertfordshire, as a 'High Potential Opportunity' (HPO) zone in recognition of its thriving cell and gene therapy cluster of national and international significance.

The successful HPO nomination was made by Hertfordshire Local Enterprise Partnership (LEP) and is expected to help attract further overseas investment into the county.

Stevenage is at the centre of the largest cluster of cell and gene therapy companies outside of the US. The growing cell and gene therapy campus in Stevenage is delivering over 1,000 jobs and is home to over 45 companies, 13 of which are in the cell and gene therapy space.

The campus was awarded Life Science Opportunity Zone status for advanced therapies in 2019 by the Office for Life Sciences.

It is jointly anchored by Stevenage Bioscience Catalyst, whose occupiers have raised over 1.6bn of investment to date, of which over 65 per cent has been invested into cell and gene therapy companies; and the pioneering Cell and Gene Therapy Catapult, which has built its Manufacturing Centre in Stevenage, and which has been instrumental to the growth of the UKs cell and gene therapy industry.

DITs High Potential Opportunities programme was launched in 2018 to encourage and accelerate foreign direct investment into the UKs key growth sectors, in line with Governments Industrial Strategy.

There are currently 17 HPOs throughout the UK, and an additional 19 HPOs will be developed over the course of Round 2.

Sally Ann Forsyth, chief executive officer at Stevenage Bioscience Catalyst said:

Shine a spotlight on your neighbourhood by becoming an Area Ambassador.

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Tag: Gene Therapy – The Think Curiouser

Posted: October 16, 2020 at 8:49 pm

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

To order this 550+ page report, which features 190+ figures and 355+ tables, please visit this link

Key Market Insights

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Amicus Therapeutics Announces Additional Positive Interim Clinical Data for CLN6 Batten Disease Gene Therapy at 49th Annual Meeting of the Child…

Posted: October 16, 2020 at 8:49 pm

CRANBURY, N.J., Oct. 12, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced additional positive interim results from its CLN6 Batten disease gene therapy program, AT-GTX-501. The results are featured in a virtual poster presentation at the Joint 16th International Child Neurology Congress and 49th Annual Child Neurology Society Meeting being held October 12-23, 2020. The presentation is also available in the Events and Presentations section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/events-and-presentations.

The Abigail Wexner Research Institute (AWRI) at Nationwide Childrens Hospital is conducting the ongoing Phase 1/2 clinical study of a single one-time intrathecal administration of AT-GTX-501 gene therapy for variant late-infantile neuronal ceroid lipofuscinosis 6 (vLINCL6) disease, also known as CLN6 Batten disease. With no approved treatments, CLN6 Batten disease is a fatal neurologic disease that rapidly robs children of their ability to walk, speak, think, and see.

Clinical Data Highlights:Interim safety data are available for 13 children with CLN6 Batten disease. Interim efficacy data are available for the first 12 children reaching the 12-month timepoint and for eight children up to 24 months, post-administration of the AAV-CLN6 gene therapy.

Jeff Castelli, Ph.D., Chief Development Officer ofAmicus Therapeutics, stated, We are pleased to share these positive interim clinical data for our intrathecal AAV gene therapy with the CLN6 community. The data continues to suggest that our gene therapy has the potential to be a treatment option for children living with CLN6 Batten disease, an ultra-rare, debilitating condition that leads to progressive declines in cognitive and motor function, and often results in death early in life.

Emily de los Reyes, M.D., Ph.D., Principal Investigator of the CLN6 clinical trial at AWRI at Nationwide Childrens andProfessor of Clinical Pediatrics and Neurology at The Ohio State University College of Medicine, stated, I remain pleased with the progress of this trial as well as our collection of natural history data to further inform the results for the AAV-CLN6 gene therapy. The interim results show that this investigational gene therapy has the potential to slow the neurological disease progression in children with CLN6 Batten disease.

Regulatory interactions for AT-GTX-501 are ongoing and the Company expects to provide feedback on the path forward in 2021.

Amicus has exclusive rights to the CLN6 gene therapy program developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About AT-GTX-501AT-GTX-501 is a novel gene therapy in Phase 1/2 development for CLN6 Batten disease, a rare, fatal, inherited lysosomal disorder with no approved treatment that primarily affects the nervous system. AT-GTX-501 is dosed in a one-time intrathecal infusion to deliver a functional copy of the CLN6 gene to cells of the central nervous system. The therapy is designed to address the underlying enzyme deficiency that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., AT-GTX-501 was granted Rare Pediatric Disease and Orphan Drug designations by the United States Food and Drug Administration. In the EU, the Company holds PRIME and orphan medicinal product designations.

About Batten DiseaseBatten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. In these disorders, a defect in a specific gene triggers a cascade of problems that interferes with a cells ability to recycle certain molecules. Each gene is called CLN (ceroid lipofuscinosis, neuronal) and given a different number designation as its subtype. There are 13 known forms of Batten disease often referred to as CLN1-8; 10-14. The various types of Batten disease have similar features and symptoms but vary in severity and age of onset.

Most forms of Batten disease/NCLs usually begin during childhood. The clinical course often involves progressive loss of independent adaptive skills such as mobility, feeding and communication. Patients may also experience vision loss, personality changes, behavioral problems, learning impairment and seizures. Patients typically experience progressive loss of motor function and eventually become wheelchair-bound, are then bedridden and die prematurely.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials and the prospects and timing of the potential regulatory approval of our product candidates. In particular, this press release relates to interim data from an ongoing Phase 1/2 study to investigate intrathecal administration of AAV-CLN6 gene therapy. The inclusion of forward-looking statements arising from this interim data, ongoing study and natural history preliminary data should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. The interim data and Phase 1/2 study discussed herein is inherently preliminary and early in the study, derived from a limited patient set, and later trial results with this patient set or others may not be consistent with these preliminary results. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2019 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2020. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.

CONTACTS:

Investors:Amicus TherapeuticsAndrew FaughnanDirector, Investor Relationsafaughnan@amicusrx.com(609) 662-3809

Media:Amicus TherapeuticsDiana MooreHead of Global Corporate Communicationsdmoore@amicusrx.com(609) 662-5079

FOLDG

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Amicus Therapeutics Announces Additional Positive Interim Clinical Data for CLN6 Batten Disease Gene Therapy at 49th Annual Meeting of the Child...

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2020 Nobel Prize in Chemistry Awarded for Genetic Scissors that could revolutionise epilepsy treatment – Epilepsy Research UK

Posted: October 16, 2020 at 8:49 pm

This years Nobel Prize for Chemistry has been awarded to Professor Emmanuelle Charpentier and Dr Jennifer Doudna for their discovery of natures sharpest genetic scissors CRISPR the tool that makes gene therapy possible. Gene therapy is an emerging key treatment for many conditions with an underlying genetic cause, including epilepsy.

The Nobel Prizes were first awarded in 1901 and are given annually for outstanding work in physics, chemistry, physiology or medicine, literature, economics, and the promotion of peace. The Chair of Nobel Committee for Chemistry, Professor Claes Gustafsson, said of this years award, There is enormous power in this genetic tool, which affects us all. This technology is a method that will lead to groundbreaking new medical treatments. This also marks the very first time in history that a Nobel Prize has been shared between two women.

The discovery of this gene editing tool known as CRISPR/Cas9 has revolutionised the life sciences and medicine. The tool is faster, cheaper and more accurate than previous techniques of editing DNA and has huge potential for a wide range of applications. Discovered in bacteria, CRISPR/Cas9 is a mechanism by which bacteria chop up the DNA of invading viruses hence the tool adopting the title of the sharpest genetic scissors. Bacteria then integrate these chopped up virus DNA into sections of their genome (the complete set of genes or genetic material present in a cell or organism). This process is known as CRISPR Clustered Regularly Interspaced Short Palindromic Repeats. These repeats are then used to detect and destroy similar viruses by Cas9 in the future.

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Blood donors needed to keep blood supply stocked for local patients – The B-Town (Burien) Blog – The B-Town Blog

Posted: October 16, 2020 at 8:48 pm

Bloodworks Northwest officials report that blood supplies are not keeping pace with the needs of local hospitals, and theyre seeking Blood Donors.

Hospital requests are 120% of normal the last seven days, especially for type O blood, Bloodworks said. This increased need for lifesaving blood has placed pressure on the supply making the need for more donors urgent in October for Pacific Northwest hospitals that count on Bloodworks for even common procedures like surgeries.

Hospitals are seeing an increase in traumas, transplants, and emergency situations requiring blood, said Bloodworks President and CEO Curt Bailey. Overall blood usage is up 20% which translates to an additional 600 units of blood needed each week. This is unsustainable unless more community members step up to fill these growing needs of our hospitals and those lives depending on them.

It typically takes 1,000 people each day to make appointments to give blood at Bloodworks donor centers and unique pop-up blood drives happening throughout Western Washington and Oregon. This month, blood donors will learn if they have COVID-19 antibodies that may help patients currently fighting coronavirus. Thats because Bloodworks is testing all whole blood donations for COVID-19 antibodies through October 31 in conjunction with pandemic response efforts. A positive test result indicates if the donors immune system has produced antibodies to SARS-CoV2 (COVID-19) regardless of whether the person ever showed symptoms.

As this high usage trend continues, our deficit increases with our most-needed Type O blood types fast approaching critically low levels, said Vicki Finson, Executive Vice President of Blood Services. Local hospitals are counting on all of us to meet their commitment to provide the best patient care possible. Whether youre a first-time donor or longtime donor, please make an appointment now to keep our shelves stocked for patients.

Donations with Bloodworks provide 95% of the lifesaving blood supply to Pacific Northwest hospitals. It takes about an hour to give blood from check-in to post-donation cookie. Appointments and masks required.In accordance with current social distancing guidelines, no walk-ins, guests, or people under age 16 are permitted onsite. Bloodworks has posted information addressing questions and concerns for blood donors at bloodworksnw.org/coronavirus. Information about who can donate and where, is available at http://www.bloodworksnw.org.

Additional Resources:For the latest information on COVID-19 please visit theCDC website, Washington State Department of Health COVID-19 main page, and Oregon Health Authority.

Bloodworks Donor Centers and Pop-Up Centers:Appointments and information at BloodworksNW.org or 800-398-7888. See our list of Donor Centers and Pop-Up locations.

About Bloodworks NorthwestBloodworks Northwest is backed by 75 years of Northwest history and 250,000 donors. It is local, nonprofit, independent, volunteer-supported and community-based. A recognized leader in transfusion medicine, Bloodworks serves patients at hospitals in Washington, Oregon and Alaska partnering closely with local hospitals to deliver the highest level of patient care. Comprehensive services include blood components, complex cross-matching, specialized lab services for organ transplants, care for patients with blood disorders, and collection of cord blood stem cells for cancer treatment. Bloodworks Research Institute performs leading-edge research in blood biology, transfusion medicine, blood storage and treatment of blood disorders. Patients with traumatic injuries, undergoing surgeries or organ transplantation, or receiving treatment for cancer and blood disorders all depend on our services, expertise, laboratories and research. Blood donation appointments can be scheduled at bloodworksnw.org.

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COVID-19 and the trials of treatment | News, Sports, Jobs – Escanaba Daily Press

Posted: October 16, 2020 at 8:47 pm

Karen Wils photoShown at right is Karen Wils, the Chemo Beagler or the COVID Beagler.

ESCANABA The year 2020 has put me and most everybody else in a very unusual position.

We all feel a little trapped and uneasy thrown into a situation we have never faced before.

I always kind of thought I would need to have a stem cell transplant someday after I was diagnosed with multiple myeloma in 2008.

I often referred to this blood cancer as my pesky woodtick. Fortunately, I have been blessed with good control of the disease until this year.

Leaving home, going to the Mayo Clinic and going through transplant is enough excitement for one person, but to be doing that while COVID-19 plagues our country is almost too much.

To say the last few months have been an interesting journey would be putting it mildly.

As the summer drew to a close, many changes were closing in on me, too. My son went off to a new job, and my daughter left for college. The very next week, my husband and I left for Minnesota for an unknown amount of weeks for my stem cell transplant.

For three-and-a-half weeks we were at Mayo. My husband was my excellent care giver through the strong chemo, and the transplant of my own stem cells and the long road to recovery.

Doing all of this during a pandemic is really interesting. The Mayo Clinic takes COVID-19 very seriously. So with much testing, temperature taking, sanitizing and constant mask wearing, we maneuvered through the treatment.

Social distancing when you are staying at the Gift of Life Transplant House and so far from home is very difficult. Phone calls, Zoom and Facebook became my life link to the kids, family and friends.

After going through some pretty weak days, finally my new immune system was strong enough for me to go home.

Oh, the great joy of heading down those last miles from Menominee to Escanaba back into the land of trees and water!

Sleeping in my own bed, seeing my pets, and being able to stand in my own garden felt so wonderful, but the house was so quiet without the kids.

The COVID-19 cautions had to continue, too.

I wear my mask and feel a little bit trapped, like everybody else these days. I pray that I am making all healthy decisions when I encourage my children to go to church or my husband to go to the grocery store.

The COVID-19 virus threatens big things and little things like family traditions that have to be rethought. With great sadness, I postponed until next year our annual camp gathering/harvest festival. So many folks have been through canceled or postponed parties, birthdays, anniversaries and funerals.

As hard and as disappointing as this is, the important things remain. The trees still are changing to awesome autumn colors. The harvest season is in full swing. The wild animals are still thriving outside our windows, and our families are together even if we cant do everything we want to do right now.

As I get stronger every day, I thank God. I pray for a cure for COVID-19. Yoopers are tough and our traditions will thrive long after this pandemic!

Karen (Rose) Wils is a lifelong north Escanaba resident. Her folksy columns appear weekly in Lifestyles.

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Prop. 14: In the COVID age, can California still afford its stem cell research program? – CALmatters

Posted: October 15, 2020 at 11:57 pm

In summary

Proposition 14 asks voters to spend nearly $8 billion to continue the stem cell research program at a time when the coronavirus pandemic has decimated the state budget.

For the second time in 16 years, California voters will decide the fate of the states multi-billion dollar stem cell research program that established the state as a worldwide leader.

How the times have changed.

In November, as the pandemic drags on, Proposition 14 asks voters to spend nearly $8 billion to continue the program during a period when the research environment has significantly evolved and coronavirus has battered the states budget.

The bond measure would approve $5.5 billion in bonds to keep the states stem cell research agency running and grants flowing to California universities and companies.

At least $1.5 billion would be earmarked for brain and central nervous system diseases like Alzheimers and Parkinsons. The overall cost of the bonds and their interest totals about $7.8 billion, according to the state legislative analyst. The state would pay about $260 million annually for 30 years, or about 1 percent of Californias annual budget.

Proposition 14 is essentially a repeat with a bigger price tag and a few tweaks of Proposition 71, which California voters approved in 2004 after then-President George W. Bush prohibited, on religious grounds, all federal funding of any stem cell research using human embryos.

The bond measure would approve $5.5 billion in bonds to keep the states stem cell research agency running and grants flowing to California universities and companies.

That groundbreaking measure authorized $3 billion in state bonds to create the states stem cell research agency, the California Institute for Regenerative Medicine, and fund grants for research into treatments for Alzheimers disease, cancer, spinal cord injuries and other diseases.

The institute has nearly used up its original funding, so Prop. 71s author, real estate investor and attorney Robert N. Klein II, led a new effort to get Prop. 14 on the November ballot.

This time, embryonic stem cell research is in a much different place, with federal funding no longer blocked and more funding from the biotech industry.

Voters will want to consider what Californias previous investment in stem cell research has accomplished. Its a nuanced track record.

While many scientific experts agree that Prop 71 was a bold social innovation that successfully bolstered emerging stem cell research, some critics argue that the institutes grantmaking was plagued by conflicts of interest and did not live up to the promises of miracle cures that Prop. 71s supporters made years ago. Although the agency is funded with state money, its overseen by its own board and not by the California governor or lawmakers.

The agency had done a very good job of setting priorities for stem cell research, including research using human embryos, and doling out $300 million annually to build up California as a regenerative medicine powerhouse, according to a 2013 evaluation by the National Academies of Science, Engineering and Medicine.

But the report also found that because the institutes board is made up of scientists from universities and biotech firms likely to apply for grants, board members had almost unavoidable conflicts of interest.

Because human stem cells can develop into many types of cells, including blood, brain, nerve and muscle cells, scientists have long looked to them for potential treatments for currently incurable diseases and injuries. Researchers use two types of stem cells: embryonic stem cells, derived from unused human embryos created through in vitro fertilization, and adult stem cells, which are harder to work with but in some cases can be coaxed in a lab into behaving more like embryonic stem cells.

From the start, stem cell research has been ethically charged and politically controversial because human embryos are destroyed in some types of studies. Federal restrictions on the research have waxed and waned, depending on which political party holds power. While former President Bush restricted federal money for embryonic stem cell research, former President Obama removed those restrictions.

The Trump administration has restricted government research involving fetal tissue but not embryonic stem cells. However, anti-abortion lawmakers have called on the President to once again end federal funding for embryonic stem cell research.

California-funded research has led to one stem cell treatment for a form of Severe Combined Immunodeficiency known as the bubble baby disease. Children with the rare disease dont make enough of a key enzyme needed for a normal immune system. Without treatment, they can die from the disease if not kept in a protective environment. The U.S. Food and Drug Administration is now reviewing the treatment but has not yet approved it for widespread use.

Although many of the agencys early grants were for basic science, the institute also has supported 64 clinical trials of treatments for many types of cancer, sickle cell disease, spinal cord injuries, diabetes, kidney disease and amyotrophic lateral sclerosis, commonlyknown as Lou Gehrigs disease.

A June 2020 analysis by University of Southern California health policy researchers estimated that taxpayers initial $3 billion investment in the research institute helped create more than 50,000 jobs and generated $10 billion for the states economy.

Gov. Gavin Newsom has endorsed Proposition 14, and other supporters include the Regents of the University of California, the California Democratic Party, the Juvenile Diabetes Research Foundation, patient advocacy groups like the March of Dimes, and some local politicians and chambers of commerce.

Supporters have raised more than $8.5 million, including about $2 million from billionaire Dagmar Dolby, to pass the measure, according to California Secretary of State campaign finance reports.

The passage of Proposition 71 helped save my life, Sandra Dillon, a blood cancer patient, wrote in a San Diego Union-Tribune commentary supporting Proposition 14. She wrote that she had benefited from a drug developed with Institute-funded research that has been designated by the FDA as a breakthrough therapy.

It is unimaginable to think that Californians would vote to discontinue this amazing effort I dont know where I would be or what condition I would be in if it wasnt for the investment Californians made nearly two decades ago.

I think the agencys done good work, but this was never planned to be funded forever with debt.

Lawrence Goldstein, a UC San Diego professor of cellular and molecular medicine and stem cell researcher, said the grants were instrumental in furthering his research on treatments for Alzheimers disease and that Prop. 14 will help create new jobs. The agency has funded a great deal of very important stem cell medical research thats already produced terrific results and has the prospect of saving many more lives in the decade to come, he said.

Opponents include one member of the institutes board and a nonprofit that advocates for privacy in genetic research. They contend that the proposition seeks too much money and does not sufficiently address the conflicts of interest that cropped up after Prop. 71 was passed. They also note that private funding, including venture capital, for stem cell research has grown in recent years. Opponents had raised only $250 by late September, from a single contribution by the California Pro Life Council.

The editorial boards of some of Californias biggest newspapers also have opposed the measure, including the Los Angeles Times, the Orange County Register, the San Francisco Chronicle and the San Jose Mercury News/East Bay Times. The Fresno Bee, Modesto Bee, and San Luis Obispo Tribune newspaper editorial boards support Prop 14.

Jeff Sheehy, the only institute board member not to support Proposition 14, told CalMatters that the research environment has changed since voters initially approved state funding for stem cell research in 2004 and that California should prioritize other needs like education, health care, and housing.

I think the agencys done good work, but this was never planned to be funded forever with debt, Sheehy said. At this point the state cant afford it; were looking at a huge deficit.

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Prop. 14: In the COVID age, can California still afford its stem cell research program? - CALmatters

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The Infodemic: Was Regeneron COVID-19 Treatment Developed Using Stem Cells and Fetal Tissue? – Voice of America

Posted: October 15, 2020 at 11:57 pm

Fake news about the coronavirus can do real harm. Polygraph.info is spotlighting fact-checks from other reliable sources here.

Claim:The development of REGN-COV2, the experimental anti-viral antibody cocktail given to U.S. President Donald Trump to treat COVID-19, involved the use of human fetal tissues and embryonic stem cells.

Verdict:Mixture

Read the full story at:Snopes

Circulating on social media:An article claiming to give ultimate proof that the novel coronavirus pandemic was planned to create a new world order.

Verdict:False

Read the full story at:Reuters

More Testing, Faster TestingMore types of tests for the coronavirus are becoming available, but how do we know which to use when?-- American Scientist, October 12

Watch: Understanding dexamethasone, the steroid used to treat Trumps Covid-19It is inexpensive and widely prescribed. But side effects of dexamethasone can be quite serious.-- Stat, October 12

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The Infodemic: Was Regeneron COVID-19 Treatment Developed Using Stem Cells and Fetal Tissue? - Voice of America

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