California's 15-year Stem Cell Journey: Where to After 1,000 Research Awards, 56 Clinical Trials, $904 Million for Basic Research?

BURLINGAME, Ca. -- California's $3 billion stem cell research agency this year is nearing only its 15th birthday, but tomorrow it will wrestle with the question of whether there is life after 2020.

The agency, known formally as the California Institute for Regenerative Medicine (CIRM), is convening a passel of out-of-state scientists here to address the very real matter of what to do next year and thereafter. 

CIRM expects to run out of cash before the end of this year. It is hoping that California voters will approve a $5.5 billion bond measure in 2020 to continue its work. 

The scientists arriving for tomorrow's public meeting are non-California folks who have evaluated some of the more than 3,000 applications from California scientists for cash from CIRM.  The agency is barred by law from financing work outside the Golden State.

A presentation prepared by the agency for the session asks:

• "What should CIRM be thinking about now to prepare for a possible life beyond 2020?
• "How can CIRM deliver the greatest impact in the future?
• "What opportunities might CIRM seize to accelerate the field?• 
• "What challenges must be addressed?
• "What types of CIRM programs should be sustained or expanded?
• "What is missing, or needs more support?"

The day-long program also includes a 49-page, information package dealing with CIRM programs, ranging from basic research to clinical activities. It is the most comprehensive, recent, public look at CIRM's efforts that the agency has prepared.

Here are some of the highlights of the fresh data overview from CIRM: 

• 56 clinical trials
• More than 1,000 awards
• More than 1,200 patients in CIRM-initiated Alpha stem cell clinics
• $904 million for basic research
• Stimulation of $3.7 billion in outside investments
• 2,700 alumni from CIRM training programs 

The session begins at 8:30 a.m. PDT. It will be available also on online. Instructions for access can be found on the meeting agenda.

State Medical Regulators To Take Up Dubious Stem Cell Clinics in November: Adverse Events, More Education on Table

The next step in possible California state regulation of rogue stem cell clinics will come Nov. 7 at a meeting of the state's medical board. 

The news follows a meeting last week by a two-person task force of the medical board to deal with the growing use of unproven treatments. (See here and here.)

The California stem cell agency reported on its blog, in a piece by Yimy Villa, about possibilities the board is considering:

• "Guidance and education materials for medical practitioners
• "Sample informed consent documents designed to inform patients about the potential risks and benefits of stem cell treatments
• "Public education materials
• "An adverse event reporting system"

In response to a query by the California Stem Cell Report, Carlos Villatoro, a spokesman for the board, said it would hear an update at its meeting in November in San Diego.
He released a statement that said.

"The board’s Stem Cell and Regenerative Therapy Task Force will continue to work on this issue with the goal of providing recommendations on stem cell and regenerative therapies and developing some guidelines that California physicians and patients can follow. The task force would meet again to obtain input on any recommendations and/or guidelines."

The California stem cell agency has taken a "Three Rs" position on the rogue clinics: "Regulated, Reputable and Reliable."

No public meeting of the task force is scheduled at this time. Interested parties can send comments and suggestions to April.Alameda@mbc.ca.gov.

Peering into California's Stem Cell Future: Public Conclave Scheduled for Next Week

California's nearly 15-year-old stem cell research program, which expects to run out of cash this year, has called a meeting for next Thursday to explore its future if voters approve a proposed, $5.5 billion ballot measure next year. 

"The goal of the meeting is to gather feedback, suggestions and recommendations for how (the agency) might deliver the greatest impact in regenerative medicine should it have the opportunity to do so in the future," said Maria Bonneville, vice president of administration for the agency.

In response to a query yesterday, Bonneville said about 30 persons were expected to be involved in the session, including a number of the scientists who participate in the closed-door reviews of grant applications from other researchers. The grant reviewers are all from outside California and make the de facto decisions on awards.

The session is open to the public.   Bonneville said parts of the meeting will be audiocast on the Internet. She said the $3 billion agency expects to produce a report out of the meeting. 

Members of the public can submit written testimony to the agency in advance of the meeting and afterwards by emailing it to info@cirm.ca.gov.

Backers of the $5.5 billion initiative to be placed on the November 2020 ballot expect to formally file it with state election officials by the end of this month.

California Regulators and Dubious 'Stem Cell' Clinics: Possible New Rules Sometime Next Year

The California state Medical Board is expected to come up with new regulations to deal with rogue "stem cell" clinics sometime next year, the San Francisco Chronicle reported yesterday. 

The rules could require more patient education about the treatments and risks as well as providing more information to physicians about federally permissible treatments. The chairman of the state's $3 billion stem cell agency has described the unproven treatments as "snake oil."

The Chronicle story by Erin Allday emerged from a hearing yesterday by a small panel of the Medical Board. Any proposal for more regulation would have to go to the full board and run through the lengthy state administrative process before taking effect.

UC Davis stem cell scientist Paul Knoepfler, who also testified at the hearing, wrote about the session on his blog. He said that physician and patient education could be "helpful." He added,

"(B)ut I wonder whether the board will actually take more direct action on the few physicians who are arguably running the riskiest clinics here in our state. Such a step would do the most to rein in the problem."

Knoepfler, who has long studied the dubious clinics, described yesterday's hearing as a "positive development." He added, 

"I just don’t know how much concrete action will come out of it and when."

Chronicle reporter Allday wrote,

"The state has come under increasing pressure by stem cell experts to tamp down on the booming consumer industry in recent years, but almost no action has been taken. Wednesday’s meeting in Sacramento, attended by a few dozen scientists, patient advocates and stem cell providers, was largely informational, and medical board representatives repeatedly noted that they have limited authority over the industry.

"But they also acknowledged concerns that 'some providers are deceiving patients and placing them at risk,' said Dr. Randy Hawkins, co-chair of the stem cell task force organized by the California Medical Board."

Also testifying was the president of the state stem cell agency, Maria Millan. Here is a link to an item earlier this week on Millan's presentation.  

Here are links to a presentation by Mehrdad Abedi of UC Davis, a specialist in bone marrow transplantation,  on biologics regulation of stem cells and to a presentation by Charity Dean, assistant director of the State Department of Public Health.

Update on California's $72 Million Bet on a Diabetes Cure: Gene Editing Holds Promise

Viacyte video

A San Diego stem cell firm fueled by $72 million from the state of California this week announced an "important step" in its search for a diabetes cure in collaboration with a Massachusetts gene-editing enterprise.

The California business is ViaCyte, Inc., a privately held company that has received more funding from the California stem cell agency than any other business. 

It ranks 9th on the list of all recipients of cash from the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. The ranking places it ahead of such highly regarded research institutions as Salk in La Jolla and Gladstone in San Francisco.

The East Coast firm is CRISPR Therapeutics, AG, a publicly traded firm that aims at "developing transformative gene-based medicines for serious human diseases."

CRISPR and Viacyte announced on Tuesday that their research is now showing that ViaCyte's "CyT49 pluripotent stem cell line, which has been shown to be amenable to efficient scaling and differentiation, can be successfully edited with CRISPR. The CyT49 pluripotent stem cell line is currently being used to generate islet progenitors for clinical trials."

Paul Laikind, CEO of ViaCyte, said in a news release that the latest news brings the firms "potentially one step closer to a transformational therapy for patients with insulin-requiring diabetes through the development of an immune-evasive gene-edited version of our technology."

Laikind described the gene editing result as "an important step" in achieving "yet another first, the development of an immune-evasive cell replacement therapy as a potential cure for type one diabetes."

In 2017, CIRM gave ViaCyte $1.4 million for work on the CyT49 line. Maria Millan, president of CIRM, said at the time,

“Development of an immune-evasive cell therapy would increase the chances of engraftment and durable effect of a cell replacement therapy for diabetes."

Investors were not energized by the CRISPR/ViaCyte announcement. CRISPR's stock price closed at $49.40 today, down from a $49.67 close on Monday, the day prior to the announcement. The 52-week high for the firm is $53.97. The low is $22.22. 

CRISPR and ViaCyte announced their $15 million collaboration a year ago. Here is a 2018 overview of Viacyte by Bradley Fikes of the San Diego Union Tribune.