Wednesday, June 12, 2019

California's Slow Pace on Stem Cell Snake Oil: State Medical Regulators May Take First Stab This Summer

California regulation of an industry that sells dubious and risky "stem cell" treatments now appears to be solely in the hands of the state Medical Board, which has been mulling the matter for at least 11 months.

Both nationally and in California, the number of unregulated clinics has risen sharply with estimates of more than 1,000 nationwide, with the largest number in California. 

Jonathan Thomas, chairman of the $3 billion state stem cell agency, has described the fast-growing clinics as snake-oil enterprises.

The clinics peddle what they call stem cell treatments to desperate patients. The treatments cost thousands of dollars. Some patients have reported losing their vision as a result. Last December, the federal government reported that 12 persons were hospitalized because of infections from treatment by a California clinic. Some of the material involved was contaminated with fecal bacteria.

The New York Times reported this week that across the nation the clinics have "attracted huge numbers of patients, who pay thousands of dollars for unproven, risky procedures." The Times piece echoed a bleak national picture that was painted earlier by the Washington Post. 

A spokesman for the California Medical Board, Carlos Villatoro, said this week that a task force of the board is expected to hold its first meeting this summer to deal with stem cell clinics. "The task force should be meeting soon and potentially holding an interested parties meeting, hopefully before the next board meeting," he said in an email to the California Stem Cell Report.

The next board meeting comes in August. The task force consists of two members of the state board:
Randy Hawkins, LA Sentinel photo
Hawkins of Los Angeles, who is clinical assistant professor of medicine at the Charles Drew University of Medicine and Science in Los Angeles, and Howard Krauss of Santa Monica, Ca., who is a clinical professor of ophthalmology and
Howard Krauss, PNI photo
neurosurgery at
 the John Wayne Cancer Institute at Providence Saint John’s Health Center in Santa Monica. 

State legislation to deal with the burgeoning problem is moribund and may or may not be resurrected.

Art Torres, vice chairman of the board of the California stem cell agency, has been working on the measure (AB617). He served for 22 years in the state legislature and is a former head of the California Democratic Party. 

Last month, Torres described the stem cell clinic measure as "dead." He said the reasons for its demise are "unclear," along with the failure of another bill dealing with sickle cell disease. Torres told the board, 

"They haven't told us why they opposed it, and even the author of the bills can't figure out why their bills remained in what's called the suspense file of the Assembly Appropriations Committee."

Torres later told the California Stem Cell Report that it was possible that the content of the bill could be amended into another measure that is farther along in the legislative process. But he said that no clear pathway to final passage was evident. 

In New York, the state attorney general has charged one stem cell clinic with fraud. In California, Attorney General Xavier Becerra has remained mum on the subject. 
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Sunday, June 09, 2019

Captain Kirk, Snake Oil and Stem Cell Treatments

It's time for stem cell whack-a-mole.

That's the game where regulators -- coming in late -- try to prevent snake-oil clinics from harming desperate patients with purported stem cell treatments and ripping off them for thousands of dollars.  

More than 1,000 dubious clinics are estimated to exist across the country with the largest number in California, which is lagging in its regulatory efforts.  More spring up every day with even Captain Kirk of Star Trek weighing in on the alleged benefits of the unregulated procedures.

William Wan of the Washington Post captured the national scene in an article late on Friday. He said don't get too excited about a favorable court decision involving one operation in Florida, which claims to turn body fat into beneficial stem cells.

Wan wrote,
"(T)he company said it would follow the federal judge’s ruling and stop selling the fat-based procedure. But it quickly followed up with a clarification: It would continue offering stem cell treatments, but instead of fat, rely on patients’ bone marrow and other tissues to harvest the cells it claims can cure conditions as varied as spinal cord injuries and erectile dysfunction.
"'It’s a bit like playing whack-a-mole,' said Peter Marks, FDA’s top official for biologic products."
The situation has been building for long time. Wan wrote,
“After years of largely ignoring the issue, the FDA is finally making more of an effort, but it’s not as if this marketplace is melting away,” said Leigh Turner, a bioethicist at the University of Minnesota. 
"Because no one had documented exactly how many stem cell clinics existed, Turner — working with collaborators (UC Davis researcher Paul Knoepfler) — began tracking them in a database several years ago. In 2009, there appeared to be only two in the United States; by 2017, there were at least 700. Turner believes there are currently more than 1,000."
"Turner compared the for-profit stem cell industry to a balloon. As the FDA and consumer advocates squeeze one end, other parts of the enterprise expand. 'For every one that’s disappeared, we’re finding seven more that are popping up.'"
Wan noted another effort in California that could affect a number of clinics. 
"Meanwhile, the government is pushing forward in a second federal court lawsuit against another stem cell business in California. That lawsuit has the potential to halt the fat-based stem cell treatment in multiple clinics because the government filed it against a company called Cell Surgical Network, which serves as an umbrella for dozens of affiliated clinics around the country."
An attempt to step up state regulation in California, however, has ground to a halt. Legislation (AB617) by Assemblyman Kevin Mullin, D-San Mateo, is now not even on life support. It is buried in the Assembly Appropriations Committee. Its content could surface in another measure that still has some technical life in the Capitol, but the outlook is more than dim. 

The scene is not much brighter with California's medical board. Last year said it would look into the dubious clinics. In April, it said hearings might begin no later than early this month. Nonetheless, a check of the agency's web site this weekend did not turn up any notice of such a meeting.
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Thursday, June 06, 2019

Trump Research Restrictions, the California Stem Cell Agency and Moral Obligations

The man expected to lead the drive for $5.5 billion more for California's stem cell agency today said the Trump restrictions on fetal tissue research represent a dangerous precedent that threatens the health of all Americans. 

Robert Klein, who was the first chairman of the state stem cell agency, said that "California has unique opportunity and obligation to maintain the scientific and medical options" that have led to development of the polio vaccine along with many others.

During an interview with the California Stem Cell ReportKlein said the people of California have a "moral" obligation to add more billions to the work of the 14-year-old, $3 billion stem cell agency.

Klein led the 2004 ballot initiative campaign that created the agency, formally known as the California Institute for Regenerative Medicine (CIRM).  The agency expects to run out of cash for new awards by the end of this year. It is staking its existence on a proposed ballot initiative that Klein would carry forward.

Klein's comments came as more reaction surfaced to the Trump action. San Francisco HIV advocate Jeff Sheehy, responding to a question, said in an email,
"Fetal tissue is used to make mice with human immune systems.  Testing new drugs for HIV is just one use--this animal model is used in research across a wide range of diseases to develop and test therapies, including vaccines for infectious diseases.  Stopping this research--which has been taking place for decades--is foolish, anti-science, and a threat to the health and safety of all Americans."
Kaiser Health News reported,
"The Trump administration’s announcement Wednesday about federal cutbacks in fetal tissue research is short of a total ban, but scientists in the field say it is concerning because it could affect work on treatments or preventions for key diseases, such as HIV and Parkinson’s."
Sara Reardon, reporting online for Nature, wrote,

"'It’s a decision that’s going to set back research,' says Andrew McMahon, a stem cell biologist at the University of Southern California in Los Angeles. 

"McMahon is studying ways to grow kidneys from human stem cells. He says that the only way to determine whether he and his colleagues have successfully mimicked natural development is to compare their proto-organs to kidneys in fetal tissue. Although biomedical research is often done using mice as proxies for people, mouse kidneys are too different from human kidneys to use in McMahon’s work."

McMahon was the recipient of a $5.7 million CIRM award dealing with kidney problems. A CIRM document filed in connection with his now concluded research said,

"Our analysis of the developing human kidney has provided the first comprehensive insight into developmental processes highlighting molecular and cellular events shared with the well-studied mouse model, but unique human features."
McMahon was recruited from Harvard to USC with the help of the CIRM grant. In response to an email query, he said that it was unclear whether his CIRM research would have become ineligible for federal support, given new Trump review processes. 
Bradley Fikes and Gary Robbins of the San Diego Union-Tribune wrote
"The sensitivity of the (fetal tissue research) matter surfaced recently when UCSD drew unwanted attention after one of its employees mistakenly solicited fetal pancreas samples from the Center for Medical Progress (CMP), an anti-abortion group whose surreptitious videos in 2015 galvanized efforts to end federal funding of Planned Parenthood."
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Wednesday, June 05, 2019

Trump Action on Fetal Tissue Research: Likely Impact on $5 Billion More for California Stem Cell Agency

President Trump today sharply cut back on federal funding for fetal tissue research in a move denounced as both politically motivated and destructive of the hopes of millions of Americans suffering from life-threatening diseases. 

The action has long been sought by anti-abortion activists who say tax dollars should not go to create a "marketplace for aborted baby parts."

Trump's moves immediately cost UC San Francisco a $2 million grant aimed at new therapies for HIV. It also drew reaction from California's $3 billion stem cell agency. 

Asked for a comment, Kevin McCormack, senior director of communications, said the Trump action will not have any impact on the agency. He said in an email, 
"Because our money comes from California this does not affect any project we fund or our ability to fund any projects."
Trump's action echoes a situation that played a major role in the ballot initiative campaign of 2004 that created the stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM). 

In one of their key arguments, backers of the stem cell measure said it was needed because of then President Bush's restrictions on federal funding for human embryonic stem cell research.

Today's action by Trump is virtually certain to be cited as justification for an additional $5 billion for the agency, which will run out of money for new awards around the end of this year. CIRM supporters expect to mount another ballot initiative in November 2020.

The scope of Trump's move was described by the New York Times, which wrote, 
"As of last year, the N.I.H. spent about $100 million of its $37 billion annual budget on research projects involving fetal tissue. The tissue is used to test drugs, develop vaccines and study cancer, AIDS, Parkinson’s disease, birth defects, blindness and other disorders. For much of that work, scientists say there is no substitute for fetal tissue."
Sam Hawgood, chancellor of UC San Francisco and a former member of CIRM's governing board, said in a statement that the decision was "politically motivated, shortsighted and not based on sound science.”

Lawrence O. Gostin, a professor specializing in public health law at Georgetown University, told the New York Times that the federal action "is akin to a ban on hope for millions of Americans suffering from life-threatening and debilitating diseases." 

Scientist Jeanne Loring, who is with Scripps Research and Aspen Neuroscience in the San Diego area, said in response to a query, 
"Fetal brain tissue transplants containing immature dopamine neurons laid the groundwork for the Parkinson’s disease neuron replacement therapy we are developing now  The outcomes were inconsistent, but some patients recovered from the disease.  Without that pioneering work in the 1990s, I wouldn’t be so confident about the potential of our planned therapy using dopamine neurons derived from Parkinson’s patients’ own induced pluripotent stem cells.
"This is one specific instance of how fetal tissue profoundly changed our view of degenerative disease.  I think that going forward, most of the regenerative therapies will be based on pluripotent stem cells, which weren’t available 30 years ago. But I don’t like to rule out the possibility that there is still pioneering work like this to be done, and so I hope that some researchers will not lose access to fetal tissue for groundbreaking medical research."
(Editor's note: Look for additional news tomorrow on the impact of the Trump decision in California and elsewhere on the California Stem Cell Report.)
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Sunday, June 02, 2019

Proven Value -- California's Stem Cell Agency Garners Praise in State's Largest Newspaper

California's $3 billion stem cell agency scored a significant media win this morning with an endorsement from one of its harshest critics, a Pulitzer Prize-winning columnist in the state's largest circulation newspaper, the Los Angeles Times.

Michael Hiltzik, who has written two books on massive public projects, wrote in the Times,

"California’s stem cell program has proved its value to the state and to science. It has earned the opportunity to show what it can achieve with an additional 10 years of life and billions more."
Hiltzik's piece appeared this morning in the print version of the newspaper, which says it has a readership of 2.1 million in the print version alone on Sundays. Hundreds of thousands more would be added online, where the column surfaced on Friday. 

Hiltzik began his column with some very bright news for the agency, known formally as the California Institute for Regenerative Medicine(CIRM). He said that Jonathan Thomas, chairman of the agency, told him that a CIRM-funded cure is headed for the marketplace. Hiltzik described it as "a major achievement."

He wrote,

"A stem cell-based cure developed by a team at UCLA for a rare disease — a 'bubble baby' disease that deprives children of a functioning immune system — is on a path toward expected approval by the Food and Drug Administration as early as next year. It would be the first treatment funded by the program to reach the market."
The achievement would come as the agency faces what Hiltzik called an "existential inflection point." CIRM expects to run out of cash for new awards later this year. The agency was created by voters in 2004 with $3 billion that the state borrowed.

The agency's existence depends on whether voters approve an additional $5 billion in additional bonds that would come from a yet-to-be-written ballot measure on the November 2020 ballot. Voters will need to be convinced that the agency has not frittered away its original $3 billion.

Hiltzik noted the agency's "ups and downs," including hype, conflicts of interest concerns and the affordability of stem cell therapies. He said a new ballot initiative offers an opportunity to make improvements in CIRM's operations.

Hiltzik wrote, 
"Klein told David Jensen of the California Stem Cell Report that he is contemplating a provision in the next initiative for “funding the infrastructure to work on access, to work with insurance companies, to work with Covered California, to work with Medicare, to make sure that there’s access, to make certain that public hospitals and public clinics in California get discounted prices.” That would be worthwhile, if it can be done."
Like most state agencies, CIRM labors invisibly. Its story and value proposition receive little public attention despite its attempts at ground-breaking science. Reporters are rarely seen at its meetings. Few articles are written about its affairs. 

That will change somewhat come the 2020 election season. However, media coverage will be dominated by national politics since it will be a presidential election year.  Given the scarce resources of the media nowadays -- along with other, expected, pressing California matters in the 2020 election -- stem cell research is unlikely to be on any front page (print or electronic) except infrequently, at best. 

Nonetheless, Hiltzik's column will be one of the key pieces that other journalists will dredge up electronically next year as they research ballot initiatives. And how the Times and Hiltzik have framed CIRM's work will be an important element in how other news stories are shaped. 
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Thursday, May 30, 2019

Faustian Bargains and Biotech Millions: A Fresh California Yarn

It is a California tale: The search for millions in cash to research and develop cures for terrible afflictions that destroy people's lives. 

But it is not centered in an office building in downtown Oakland, with great views of San Francisco Bay and overlooking Lake Merritt. 

The official plot summary goes like this:
"Desperate to secure funding for her med tech startup, an idealistic scientist and her husband strike an outrageous deal with a mysterious investor."
"There’s a new Netflix series involving biotech, venture capital, and Faustian bargaining. It's called 'What/If' and it’s about the founder of Emigen Molecular Sequencing who, despite her scientific acumen and noble goal of saving cancer patients, cannot seem to convince Silicon Valley’s ocean of vest-clad VCs to invest in her genomics company. ('I’m afraid Big Pharma would drown us in litigation,' says one, inexplicably.)
"That’s when she meets the all-powerful investor Anne Montgomery, played by RenĂ©e Zellweger, who seems to be splitting the difference between 'Basic Instinct' and 'Devil’s Advocate.' Montgomery offers her the money she needs to keep Emigen afloat, but the deal has dark implications for her earnest and unfortunately sideburned husband, who is a pro baseball player-turned-EMT with a night job as a bartender.
"Anyway, by the end of the pilot, there’s an embattled startup, a fractured marriage, and an unsolved assault. Your correspondent (Damian Garde) did not make it to episode two."
As for that Oakland enterprise mentioned earlier, it is the $3 billion California stem cell agency, which is running out of cash and looking for $5 billion more.   Sphere: Related Content

Wednesday, May 29, 2019

Stem Cell 'Teething Problems' -- Pricing to Manufacturing, the European View

From Europe this morning comes a perspective on the "three obstacles" that are blocking cell therapy from the mainstream.

It is a subject, of course, that resonates in the United States and particularly in California, where the $3 billion state stem cell agency is running out of cash and needs a boost.

The article appeared on Labiotech, which bills itself as "leading digital media covering the European biotech industry." Written by Helen Albert, the piece actually lists more than a trio of "teething problems" for the stem cell industry. They are:

  • "A reliable, consistent and scalable manufacturing system" 
  • "Excessively high pricing"
  • Safety, regulation and a knowledgeable workforce
Albert interviewed a number of industry executives in Europe. She also mentioned Orchard Therapeutics, which has a tie to California.  It has received an $8.5 million award from the state stem cell agency, formally known as the California Institute for Regenerative Medicine.

The United States is ahead of the game on regulation, according to the folks in Europe. Albert wrote, 
"The US regulators are currently in the lead when it comes to keeping up with the research and being open to changing their processes, according to (Alan O’Connell, partner at Irish life sciences VC Seroba). Although he says Europe is not too far behind. 'Certainly, the EMA (European Medicines Agency) is responding, but from my perspective, the FDA is taking the lead, at least currently.'"
The Albert piece said, 
"With the first CAR T-cell therapies (technically a combination of gene and cell therapy) to treat blood cancers gaining US and European approval last year and global investment of over €6.7B in cell therapy in 2018, the question on everyone’s lips is ‘is cell therapy about to hit the big time?’"
Labiotech's answer? "2019 certainly appears to be the start, if not the end, of cell therapy’s move to the big time."

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