Thursday, September 19, 2019

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.
Sphere: Related Content

Wednesday, September 18, 2019

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. 
Sphere: Related Content

Monday, September 16, 2019

'Regulated, Reputable, Reliable:' A California Call for More Regulation of Dubious 'Stem Cell' Clinics

The president of the $3 billion California stem cell agency, Maria Millan, this week is recommending a new approach to state regulation of rogue "stem cell" clinics that are preying on desperate patients.

In a presentation prepared for a meeting Wednesday of the
Maria Millan
CIRM photo
stem cell task force of the State Medical Board, Millan listed standards for patient care, declaring that the field should be "regulated, reputable and reliable."

Millan said patients are being harmed by unproven treatments at clinics in California and elsewhere. The chairman of the board of her agency, Jonathan Thomas, has called the treatments "snake oil." (The agency is formally known as the California Institute for Regenerative Medicine (CIRM).)

A small task force of the State Board is laying the groundwork for additional regulation of the industry, which has grown rapidly in recent years. Estimates are that 1,000 clinics exist across the country with the largest number in California. 

Earlier this year, a measure to set standards for the clinics died in a legislative committee. The board itself initiated its task force more than a year ago. This week is its first public hearing. 

Millan's slide presentation for the meeting did not go into details. But she called for improved informed consent from patients and raised the following considerations:
  • Creation of publicly available registries of stem cell treatment options  
  • Creation of a method for patients to self-report outcomes or harm from unregulated regenerative medicine treatments 
  • Requiring "clear visibility" for patients of the treatment team's credentials
  • Creation of a "mechanism for investigating those holding healthcare  licenses suspected of violating professional        standards when providing unproven stem cell interventions,  particularly those outside their scope  of training" 
  • Registration of treatments that involve human cellular and tissue products along with a clear distinction between federallly authorized treatments and practice of medicine
The public can file comments on the matter by emailing them to  Check here for details on Internet access to the meeting. Sphere: Related Content

Friday, September 13, 2019

$1 Million StemGenex Bankruptcy: Only $300 in the Bank

The tale of a La Jolla firm called StemGenex appears to be winding down with a bankruptcy filing that says it owes more than $1 million and has assets of less than $156,000.

The current state of the "stem cell" enterprise, which is facing a patient lawsuit and federal scrutiny, was disclosed this week in the Los Angeles Times and on the blog of UC Davis stem cell researcher Paul Knoepfler

Knoepfler has followed the firm for years. In his posting Wednesday, he said the company's bankruptcy filing shows that it owes money to its landlord, some former employees, some of its attorneys and has only $300 in the bank. 

Yesterday, Michael Hiltzik of the Los Angeles Times, who also has followed the company for some time, wrote, 
"The filing opens a window into the scale of StemGenex’s business. It discloses revenues of more than $8.2 million dating back to Jan. 2, 2017. Based on the firm’s standard fee of about $14,900 per treatment, suggesting it may have had as many as 550 customers over that period; some have said they had more than one treatment, for which they were charged separate fees."
Hiltzik said that company officials could not be reached for comment and that the firm's attorney did not respond to a query. The company's web site no longer shows a phone number and identifies the firm as only an "educational stem cell resource."

Hiltzik reported that the lead attorney in the patient class action lawsuit says he will continue to press the company, including physicians formerly associated with it. 

A task force of California's state Medical Board next Wednesday is scheduled to begin hearings in Sacramento on possible regulations dealing with operations of dubious stem cell clinics and physicians associated with them.    Sphere: Related Content

Wednesday, September 11, 2019

Meeting Delays and the California Stem Cell Agency: A Matter of Many Millions of Dollars

For the second time in two months, the governing board of the $3 billion California stem cell agency has postponed important meetings which had been expected to deal with its final research awards and its declining finances. 

The latest postponement involved a Sept. 25 meeting. On the table would have been redirection of tens of millions of dollars and the question of financing awards already approved by the agency's reviewers.

Last month, an Aug. 22 session involving major awards was  cancelled. As of today, the next meeting of the agency's board will not come until Oct. 31.

The governing board of the agency, known formally as the California Institute for Regenerative Medicine (CIRM), has before it far more reviewer-recommended awards than it can fund. Agency officials hoped that funds received from cancelled research projects would help fill the gap.

CIRM is financed with $3 billion in state bonds but has no source of significant cash beyond that. It expects to run out of money for new awards -- most likely by the end of next month -- depending on how it deals with the applications in its award pipeline. Those decisions are now likely to be made at the meeting currently scheduled for Oct. 31. 

Queried about the reason for delaying this month's meeting, Maria Bonneville, executive director of the board,  replied, 
"There were some scheduling issues and, as you know, getting the whole board together isn’t easy. (The) soonest I could get a quorum was Oct 31."
CIRM's future depends on a proposed, $5.5 billion ballot measure in November 2020 that would allow it to continue at the pace of the last 14 years. The agency also has been engaged for some time in an effort to raise privately more than $200 million to bridge the funding gap between now and the 2020 election. 

No specific progress has been publicly announced concerning that effort. However, the financial plight of the agency has received more public attention in recent months. That attention may have generated the loosening of the purse strings of some potential donors. 

The agency also may well have made a pitch to California Gov. Gavin Newsom for some sort of interim funding, possibly payable back with 2020 bond proceeds. Newsom was an important supporter of the agency in 2004-05 and engineered a $17 million package to lure its then headquarters to San Francisco when he was mayor of that city. Sphere: Related Content

Tuesday, September 10, 2019

$5.5 Billion Stem Cell Ballot Measure: California Backers Taking First Formal Step This Month

CIRM graphic
Backers of a California ballot initiative to provide $5.5 billion more for the state's cash-strapped stem cell agency say they will take their first official step by the end of this month. 

That's when they will submit the proposed measure to California election officials and trigger a many-months-long process. The effort is aimed at ensuring that the nearly 15-year-old research effort survives in a meaningful way beyond next year.

The stem cell agency, officially known as the California Institute for Regenerative Medicine (CIRM), expects to  run out of money for new awards as early as late October.  It has already cut off grant applications except for a joint sickle cell effort involving the National Institutes of Health

Robert Klein, a Palo Alto real estate investment banker, and his stem cell advocacy group, Americans for Cures, are leading the way on the initiative. Melissa King, executive director of the organization, told the California Stem Cell Report last week that the wording of the initiative is still being crafted.

King said, however, that she expected the measure to be submitted to the California secretary of state before October. She said that one area still being drafted involves providing assistance for low income patients and their families in rural areas who do not have the wherewithal to travel to and participate in clinical trials.  

The initiative filing will trigger a lengthy process to gather the  633,212 signatures of registered voters necessary to qualify the  proposal for November 2020 ballot. Gathering those signatures is a task performed by specialized firms. It could cost something in the neighborhood of $5 million or more, which Klein will have to raise on his own.

Many more signatures than the minimum are gathered because significant numbers of the signatures are commonly determined not be valid.

The ultimate deadline for qualification is June 25 of next year, 131 days before the Nov. 3 election. But the signatures will have to be submitted well before then.

The stem cell agency was created in 2004, also by a ballot initiative, with $3 billion in state bond funding. The new initiative will likewise use bond funding, which roughly doubles the cost to taxpayers because of interest expense on the borrowed cash.

The 2004 ballot campaign was supported by Hollywood stars and Nobel Prize-winning scientists. It generated grand expectations that stem cell therapies were right around the corner. While CIRM is currently involved in 56 clinical trials, it has yet to back a therapy that is widely available. Clinical trials are the last step before a medical treatment is approved for widespread use and have a high failure rate. 

Klein led the campaign in 2004 and became the first board chairman of the agency. Klein has been gathering information, statistics and support for the effort at his Palo Alto offices. 

In June, he told the California Stem Cell Report:
"This medical revolution holds the promise of restoring health and quality of life for many of California’s individuals and families suffering from chronic disease and injury.
"However, the last tactical mile to bring this broad spectrum of therapies to patients will require more funding and the thoughtful support of California’s public as the human trials and discoveries are refined and tested, overcome numerous obstacles or complications, and ultimately serve to improve the life and reduce the suffering of every one of us."
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Sunday, September 08, 2019

Google to Halt Advertising by Dubious Stem Cell Clinics, Cites 'Bad Actors'

Google says it is cracking down on advertising by rogue "stem cell" clinics, which it characterized as a rising tide of "bad actors" who are fleecing patients yearning for cures. 

The announcement came Friday and was quickly picked up by the Washington Post and then numerous other outlets, ranging from Yahoo to the MIT Technology Review. 

Google's move, which takes effect next month, would seem to add impetus to a growing cry for more and faster regulation of the dubious clinics by state and federal regulators. Both the Food and Drug Administration and California have been slow to act although the problem has been known for years. (See here, here and here.)

Injuries, blindness and deaths have been reported globally as the result of the so-called stem cell treatments. No assurances exist that the substances being used are, in fact, stem cells. 

The MIT article by Antonio Regalado noted that Google hauls in more than $110 billion annually in advertising revenue. The first sentence of his piece said, 
"After brazenly taking ad money from health-care scammers, Google is finally saying no to ads for unproven stem-cell treatments."
A UC Davis researcher, Paul Knoepfler, a national leader and pioneer in documenting the reach of the rogue clinics, said on his blog,
"I see the new policy by Google as a positive step and support them taking it. Perhaps some mainstream media outlets will follow suit and also adopt policies whereby they do not run these risky biomedical ads."
He also noted, 
 "These ads and certain particularly risky clinics also are a real threat to the legitimate stem cell and gene therapy fields."
Deepak Srivastava, president of the International Society for Stem Cell Research and head of the Gladstone Institutes in San Francisco, said in a statement
“Google’s new policy banning advertising for speculative medicines is a much-needed and welcome step to curb the marketing of unscrupulous medical products such as unproven stem cell therapies.”
The California State Medical Board has scheduled a Sept. 18 hearing into the clinics with an eye to strengthening regulation of treatments at the clinics.  Sphere: Related Content