Sunday, March 24, 2019

California Legislation Targets Unregulated "Stem Cell" Clinics but Not Until Next Year

Legislation that may lead to regulation of dubious stem cell clinics is now before California lawmakers, but it is not likely to result in action until sometime next year at the earliest. 

Assemblyman Kevin Mullin, D-San Mateo, announced the measure (AB617) last week. He said in a news release
Kevin Mullin, LA Times photo
“It is clear that more must be done to ensure the proper regulation of for-profit stem cell clinics."
More than 100 unregulated "stem cell" clinics exist in California and hundreds more throughout the nation. They use what they describe as stem cells in untested treatments that cost thousands of dollars. A number of injuries, including blindness, have been reported. (See here, here, here and here.)

Jonathan Thomas, chairman of the $3 billion California stem cell agency, told its governing board last week that the clinics were peddling "snake oil."

Mullin's bill would create an advisory board within the state Department of Public Health to study the problems involving unregulated stem cell clinics and make recommendations to the legislature. 

The panel would also have the power to enact emergency regulations. If passed by the legislature this year, it would not take effect until next January. In addition to requiring hearings, the board could not implement emergency regulations without at least a 90-day waiting period.

Art Torres

Asked for comment on the legislation, Art Torres, vice chairman of the state stem cell agency and a former state lawmaker, told the California Stem Cell Report that he would withhold remarks on the bill until  "we work out final language/amendments before its first hearing in April."

Generally, proposed laws, including Mullin's measure, do not take effect until January of the following year after they are approved by both houses of the legislature and signed by the governor. 

Legislation that has "urgency" status can go into effect immediately, but such measures require a two-thirds vote of both houses instead of a simple majority. Securing a two-thirds vote can sometimes be difficult. 

The problems surrounding unregulated clinics have existed for years, but received little attention at the state or federal level until UC Davis researcher Paul Knoepfler and Leigh Turner of the University of Minnesota documented the scope of the business in 2016.

California's state medical board, which regulates physicians, is also looking into the unregulated clinics. Sphere: Related Content

Thursday, March 21, 2019

Down to its Last $67 Million: California Stem Cell Agency Awards $4 Million to Improve Bone Healing


A look at the the spinal affliction targeted by CIRM-backed research. Video by NuVasiveInc

OAKLAND, Ca. -- California's state stem cell agency is down to its last $67.3 million following a decision today to back research to enhance bone healing in elderly patients who undergo spinal surgery.

The $4 million award went to Ankasa Regenerative  Therapeutics following little discussion among members of the governing board of the $3 billion California Institute for Regenerative Medicine or CIRM, as the stem cell agency is known. 

Ankasa, which has locations in La Jolla and South San Francisco, is supporting the award with a matching amount of $1 million. Sandy Madigan, CEO of Ankasa, told the California Stem Cell Report that his firm has raised $19 million in venture capital. Its only current potential product, he said, is tied to the CIRM-backed research, which the agency has previously funded with $8.6 million. 

(See here and here for more on the Ankasa work.)

CIRM said at today's meeting that the award leaves the agency with $67.3 million for research for the remainder of the year. It has budgeted a total of $93 million for clinical stage awards this year.

The stem cell agency was created in 2004 with $3 billion in funding. It expects to run out of cash for new awards later this year. CIRM is seeking to raise $220 million privately to tide it over until November 2020, when it hopes voters will approve another $5 billion in funding through the use of state bonds. 

CIRM board Chairman Jonathan Thomas did not report at the meeting today on the status of the private fundraising effort. 

The only significant source of cash for the agency is the $3 billion in bonds approved by voters in 2004 via the ballot initiative process. The 2020 effort would also involve a ballot initiative. 

The deadline for filing such a measure is Aug. 19. That would set the stage for actually gathering the 585,407 valid  signatures needed to qualify for placement on the ballot. 

Today's award was approved Feb. 28 behind closed doors by the agency's out-of-state reviewers, who sent it to the CIRM board for routine ratification. 

The six-page, CIRM summary of the review said the proposal (CLIN1-11256) addresses "the need for a spinal fusion material that can increase the chance for a solid bony union in the lumbar spine." The review said, "There are other products on the market that fulfill this need but have safety concerns that have been raised in the past."

Jill Helms, chief scientific officer of Ankasa and a professor at Stanford University, led the research backed by CIRM. 

The review summary said the proposed treatment could have application in other areas as well. It is likely to be some years before the procedure would be widely available. Today's award supports efforts by Ankasa to gain federal approval to begin clinical trials.

See here for the CIRM press release on the award.  Sphere: Related Content

Thursday, March 14, 2019

Making 'Stem Cell Lemonade' in California

Trump visiting lab in China in 2017, whose research output is
surpassing the U.S.
 Photo: Andy Wong/AFP/Getty Images
California's $3 billion stem cell agency has what some might call an "unconscious" ally in its search for more billions to fuel its drive to create stem cell therapies and cures.

It is no small matter. The agency expects to run out of cash for new awards by the end of this year. It is hoping that voters will approve, in November of 2020, another $5 billion to carry on with its 14-year-old program, which is a pretty big ask.

Now comes President Trump with his latest proposed budget, which whacks away at scientific research. He is seeking to slash as much as $6 billion from the National Institutes of Health, the chief source of research funding in the country.

The American Association of Immunologists said this week that Trump's cuts “would devastate important research intended to prevent, treat, and cure innumerable diseases."


Trump's cuts play into a narrative that worked successfully in 2004 when California voters created the stem cell agency with 59 percent of them voting for Proposition 71. The campaign pushed the ballot initiative with the argument that then President Bush was crippling stem cell research and thus preventing development of new, nearly miraculous therapies.

Like Bush, Trump is something of a scientific villain, so to speak, one that can be used as a foil to convince the people of California to provide more money for stem cell research. Never let good villain go to waste might be the marching orders for the 2020 ballot campaign.

If not for California and its stem cell agency, voters would be told, children would have died (see here and here) and more than 50 clinical trials for stem cell treatments would have not existed.

It is no matter that Congress may not go along with Trump's reductions. The threat, which is likely to continue as long as Trump is president, is sufficient to fuel a ballot campaign.

Obviously, cuts in federal research funding are not something the scientific and biotech community applauds. Nonetheless, they could be picked apart to find morsels to feed a ballot campaign. The agency's backers might even say, "When the president gives you stem cell lemons, make stem cell lemonade."
Sphere: Related Content

Tuesday, March 12, 2019

Cash and the California Stem Cell Agency: A Critique Notes August Deadline for New Funding Initiative

California's $3 billion stem cell program is facing a cash crunch this year, and the latest commentary on its financial fate raises a host of questions. 

The critique comes from the Center for Genetics and Society, a Berkeley group that has long been critical of the California Institute for Regenerative Medicine (CIRM), as the state stem cell agency is known. 

Writing on the center's blog, Pete Shanks briefly recapped the history of CIRM and its current financial situation. The agency expects to run out of funds for new awards this year and is  trying to raise privately more than $200 million. The cash is intended to bridge the gap between now and presumed voter approval of an additional $5 billion in November 2020. 

Quoting CIRM board transcripts, Shanks wrote: 
"They are still looking for an 'anchor investor,' who might encourage others (as an anchor store draws people to a shopping mall). Which makes CIRM board member Jeff Sheehy (long-term AIDS activist and former San Francisco Supervisor) sound prescient, since in September 2017 he had suggested that savvy voters might say (pp. 78–9 ): 
'So you went to fund-raise. You didn’t get enough to keep you going, so you’re coming back to me with your hand out. So why didn’t you get enough? Why did the people who you’ve been asking for money not think you were a good investment? Why should I?' 
"Good point. Indeed, some of the board members were over-optimistic in that 2017 assessment."
Shanks also raised other questions about the nature of CIRM's private fund raising effort and looming deadlines for qualifying a bond measure, along with a ballot initiative that will likely retool the existing law that created the agency in 2004. 

Shanks concluded,
"Propositions take considerable time to be approved. The deadline for submitting a proposed measure to the attorney general that’s intended for the November 2020 election is August 20, 2019 . In practice, that means that the proposition is probably being written now, or will be completed very soon, and preliminary backers have likely been identified and contacted already. Unless, of course, such funders cannot be found. 
"Will CIRM’s problems be solved? We’ll soon know."

Sphere: Related Content

Stem Cell Hype: The Latest Warning from Researchers

The Scientist journal last week carried a cautionary note concerning iPS cell therapies, particularly a recent, "exciting" announcement concerning their use for treatment of spinal paralysis.

"Ethical Challenges in Using iPS Cells to Treat Paralysis," was the headline on the opinion piece. It carried a subheading that said,
"Uncertainties about the cells’ risk profiles and the potential for hyping unproven therapies mean scientists and the media must tread carefully."
The article dealt primarily with what it termed "exciting" news about an experiment in Japan. But the article has broader implications for the entire field including in California, where the state stem cell agency is currently helping to fund 46 research projects involving iPS, induced pluripotent stem cells. 

The piece was written by John D. Loike, a professor of biology at Touro College and University Systems in Brooklyn and who has a regular column on bioethics in The Scientist, and Martin Grumet, a professor of cell biology and neuroscience at Rutgers.

Writing about the Japanese research, they said, 
"Caution is warranted here, for at least three reasons: the uncertainty of the stem cell type to be used in their clinical trial, the safety of transplanting stem cells into humans, and the responsibility of scientists and the press to communicate clearly the benefits and risks of the stem cell treatments, especially to desperate patients who would seek such unproven treatments."
They continued.
"The excitement of the press release may be misinterpreted by patients, who may think that now is the time to treat human spinal cord injury with stem cell transplants. With difficulties getting admitted to legitimate sub-clinical trials, could this hype for hope lead poorly informed patients to seek out other “stem cells” to treat their spinal cord injuries or other conditions? We must avoid anything that may promote “medical tourism” to unapproved interventions.

"The fact that there are more than 700 'stem cell clinics' advertised in the United States alone highlights the desperation of so many patients with terminal illnesses seeking unapproved or unproven therapies. There are no studies documenting the therapeutic successes of these clinics and some of their patients have developed serious side effects. Scientists and the press must ensure an ethical and realistic presentation and communication of new and potentially exciting discoveries and caution readers about the realities of initial clinical trials.
Sphere: Related Content

Wednesday, March 06, 2019

The FDA and the Loss of Gottlieb: What Does it Mean for Stem Cell Research?

Scott Gottlieb at his earlier Senate confirmation hearing

"Panic Attack" --That's what the headline this morning said about the resignation of Scott Gottlieb as head of the Food and Drug Administration(FDA). 

The STAT article addressed the general biotech field and its worries. Gottlieb's departure, however, also creates some uncertainty involving California's $3 billion stem cell agency. Known as the California Institute for Regenerative Medicince (CIRM), the agency is backing 51 clinical trials, all of which are regulated by the FDA.

STAT is one of the more authoritative outlets dealing with medical research news. Its Gottlieb piece reviewed his impact on stem cell and regenerative medicine and what his loss might mean. The full headline said, 
"Scott Gottlieb's Sudden Resignation Will Give Biotech a Panic Attack"
The article by Matthew Herper and Adam Feuerstein said,
"Without Gottlieb at the helm, there is uncertainty. And there is nothing that scares investors and biopharma more."
Most of the news coverage of Gottlieb's surprise resignation focused on his efforts regarding vaping and opiods.  But STAT was not alone on discussing the stem cell/regenerative medicine issues. A piece on Politico said, 
"Gottlieb worked to advance cell and gene therapies, a promising new area of medicine. Under his leadership, the FDA put out a framework for regenerative medicines, designed to accelerate the approval of the most promising gene therapies. At the same time, Gottlieb balanced promoting legitimate new treatments with crackdowns on bad actors who were taking advantage of patients by marketing unapproved, unproven and potentially dangerous treatments."
In California, the state has invested roughly $2.5 billion so far in stem cell research with goal of bringing products to market. That is a process that heavily involves the FDA.

Indeed, a top official of the FDA will be speaking next month at a San Francisco conference sponsored by the agency and UC San Francisco. The topic? "Regulation and the Office of Cell Therapy and Gene Therapies Expedited Reviews"

It is far from clear what will emerge at the FDA concerning regenerative medicine or whether Gottlieb's approach will continue.  President Trump may leave the post vacant for some time. He will be heavily lobbied by interests that have opposed Gottlieb's more visible efforts concerning vaping and opiods. 

Trump also will feel pressure from the regenerative medicine industry. But given the multiplicity of issues facing the president, the FDA is not likely to be high on Trump's priority list. And then there are Senate confirmation hearings on any appointment.

Meanwhile we can expect to see more analysis in the coming weeks of what it all means for stem cells and regenerative medicine. 
Sphere: Related Content

Monday, March 04, 2019

A Chance to Peek Inside a $50 Million California Stem Cell Program


Video from 2017 Alpha Clinic symposium

One of the better stem cell, show-and-tell conferences is coming up this spring in San Francisco, plus it is free and open to the public.

The day-long session will provide a peek inside one of the signature efforts of California's 14-year-old, $3 billion stem cell research program, which is edging closer to ever more critical financial times. 


The meeting April 18 is the fourth annual Alpha Clinic symposium. The Alpha Clinic Network was created in 2015 by the California Institute for Regenerative Medicine(CIRM), as the stem cell agency is formally known. 

CIRM has pumped $50 million into the network, which is aimed at delivering clinical trials to patients, creating strategic relationships between science and industry and leveraging statewide resources. Sites for the network are located at the City of Hope and at UC campuses in San Francisco, Davis/Sacramento, San Diego, UCLA and UC Irvine.


For those interested in California public policy issues dealing with health, medical mattters and research, the symposium will offer an up-close opportunity to check out the impact of the state stem cell agency. 

The session promises updates on stem cell research and clinical trials on afflictions ranging from sickle cell disease to cancer along with a look at gene editing. Scientists and patient advocates will be telling their stories and detailing the research.

CIRM is scheduled to run out of cash for new awards this year. It is attempting to raise privately $220 million to bridge a financial gap before hoped-for voter approval of $5 billion more in funding in November 2020.

Geoff Lomax, CIRM's senior officer for strategic infrastructure, told the California Stem Cell Report via email last week:

"It’s an exciting time in medicine as we have the tools to literally mend cells at the DNA level. The annual symposium is an opportunity to hear from the pioneers who developed these tool, the Alpha Clinic doctors and nurses that use them and the patients that have benefited from them."
Here are links to more information about the Alpha Clinic Network: Agenda for the symposium. CIRM's Alpha Clinic pagea CIRM blog item on the April meeting. 

The session this spring is financed with $60,000 from the stem cell agency.
Sphere: Related Content