'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 April.Alameda@mbc.ca.gov.  Check here for details on Internet access to the meeting.

California's Stem Cell Agency Touts Its "Incredible Ecosystem" of Research

The California stem cell agency today added more of its comments to last week's evaluation of its work by the San Franciso Chronicle in a lengthy piece that said the agency had not measured up to voter expectations.

The forum was The Stem Cellar, the blog of the California Institute for Regenerative Medicine (CIRM), as the nearly 14-year-old agency is formally known. 

Maria Millan, CIRM photo

CIRM CEO Maria Millan, in a Q&A, elaborated on the value proposition offered by the agency and some of the points that were raised in the Chronicle article. Here are two excerpts from the blog. 

"Q: There have been many critics who say it’s taking too long for CIRM to deliver cures, and they expected more. What is your response to these people?

"A: Many of us can relate that relief cannot come quickly enough for our relatives and friends who suffer from debilitating and devastating medical conditions— I believe that is why many of us are at CIRM, an organization whose mission is to accelerate stem cell treatments to patients with unmet medical needs. Through the years, we have enabled the creation of an incredible ecosystem of top scientists and researchers and partnered with patients and patient advocates to pursue this mission. We continually strive to improve and to become more efficient and we share the sense of urgency to harness the potential of stem cell biology to deliver relief to those in need.

"Q: Given all of the differences between CIRM and the NIH (National Institutes of Health), why do you think the reporter compared CIRM to the NIH?

"A: The NIH is the largest health research funder world-wide, has been around a lot 
longer, has a much larger budget >$30B this past year alone and the NHLBI
(National Heart, Lung, and Blood Institute) alone has a $3B annual budget—NHLBI is just one of the 27 NIH Institutes. The reason that CIRM was formed is that the advocates of Proposition 71 wanted to make sure that scientists and developers can pursue vital research opportunities that may not have access to funding by traditional funders, including the NIH. CIRM has a total budget of $3B available to fund research and support operations and we have been managing that budget since the passage of Proposition 71 in 2004. If we consider the number of stem cell trials for given available budget, CIRM has funded a disproportionately higher number of translational and clinical programs in stem cell and regenerative medicine. In fact, the NHLBI has entered into a collaboration with CIRM on their Cure Sickle Cell initiative because of CIRM’s specialization in funding and enabling cell-gene regenerative medicine research. I take this as a validation of CIRM’s value proposition in this new area– acceleration, translation, and clinical trials."

See here for more on the sickle cell initiative.

CEO Millan Makes the Case for California's $3 Billion Stem Cell Research Program

Maria Millan, CEO of CIRM, at state Assembly hearing last week

The top executive at California $3 billion stem cell agency, which expects to run out of cash for new awards next year, outlined for state lawmakers last week the benefits of the nearly 14-year-old program.

Maria Millan, president and chief executive officer of the California Institute for Regenerative Medicine (CIRM), as the agency is officially known, laid out how the agency funds "the most innovative, high risk but high reward" stem cell research in the state.

Millan highlighted some of the 49 clinical trials backed by CIRM. She said,

"Every single trial represents a tremendous breakthrough approach that was unimaginable and even the subject of science fiction just years ago."

She also discussed the agency's relationship with the stem cell industry in backing research during a risky financial stage called "the valley of death." She said,

"CIRM supports and de-risks through this stage, where industry and traditional investors are not yet ready to come in. By accelerating development and allowing these early stage trials to proceed with CIRM funding, we have enabled the projects to gain visibility and interest leading to an increase in industry investments."

Her presentation was generally well received by the five lawmakers who were in attendance at various times during the meeting of the Assembly Select Committee on Biotechnology, chaired by Kevin Mullin, D-San Mateo.

The full hearing can be seen here and downloaded with closed captioning. An audio file is also available at the same URL. Tomorrow the California Stem Cell Report will carry the text of remarks by Art Torres, vice chairman of the agency and a former state legislator.  This writer was also invited to appear at the session. My remarks can be found here.

Here is the full text of Millan's comments.
Remarks by Maria Millan, President of CIRM, To California Assembly's Select Committee on Biotechnology 2018... by DavidJensen on Scribd

California: Stem Cell Capital of the World? The Perspective from Oakland

The California Stem Cell Report recently spent an hour chatting with Maria Millan, the president and chief executive officer of the $3 billion California Institute for Regenerative Medicine (CIRM), as the 13-year-old, state stem cell agency is formally known. 

Maria Millan, CIRM photo

The session at CIRM headquarters in Oakland, Ca., covered a wide range of topics as the agency was releasing its annual report. One question raised by this publication was whether the Golden State has reached the sort of critical mass that would make it "the stem cell capital of the world."

Here are some lightly edited excerpts of Millan's response to that question.

"I don't know if we've reached the critical mass, but I think we are probably the furthest to (it). I remember joining (the agency)....and actually some of our programs were geared toward how can we bring in more business into California. What I'm seeing right now is a lot of players who don't actually need to come to California.  They're fine. They've got funding. They've got trials. They already have a plan to bring their products to market in the U.S., but they're coming to California, so that's telling."

"California is expensive, so we have to think about that piece of it. These cell therapies are manufacturing intensive. One of the things we are setting up is the translating center (a $15 million project at Quintiles -- now IQVIA -- in San Diego) to address that need because the process and the manufacturing have to occur close to the site of delivery, and the scientists are here, the developers are here.

"It would be great if there was a way that we could continue to help the field to address this need because that is what's going to be required in terms of getting these therapies really to patients and (meeting) the manufacturing challenges. 

"We're doing what we can with the expertise and what we currently have in place. We need to do that in partnership with other entities, so there's a lot of interest in that. The NIH and the FDA at a regenerative medicine forum identified this as a clear need -- standardization in manufacturing...."

"Another thing that is important in terms of recognition, the space of stem cell regenerative medicine does not follow the classical pharma model of drug development at all. Even the CAR-T therapies and the SPARC, those were all developed in academia, they were spun out to smaller, nimble organizations. These projects are highly risky. The de-risking role that CIRM has is critical, (reflecting) a recognition that there's now probably a new dance that occurs to getting these types of treatments developed and out there and commercialized....

"We have played a critical role in being kind of not just a funder, not just a match-maker, but an active participant in connecting the dots and bringing this conversation forward."

(Look for more on the California Stem Cell Report from Millan's additional comments throughout the month.)

CEO Millan on the Prospects for California's $3 Billion Stem Cell Research Effort

Maria Millan, the president of the $3 billion California stem cell agency, this week discussed the future of the nearly 13-year-old effort, which expects to run out of cash for new awards in 2020.

Her remarks are contained in a Q&A interview on RegMedNet, "a networking site that unites all members of the diverse regenerative medicine community," according to a statement on the site.  

The piece contains no big surprises but is worth examining if you want to get a sense of her background and where she is headed. Here are two excerpts.

Question: "What will happen once CIRM has given out all $3 billion allocated to it by Proposition 71?"

Answer: "We are working with our board to pursue sustainability options to ensure that the products of the Proposition 71 investment have the best chance of reaching the patients.  CIRM has played an essential role in growing the stem cell/regenerative medicine field by bringing in the critical mass of resources, top notch researchers, rigorous basic and translational research and building the most robust late development and clinical stage portfolio to accelerate novel treatments to patients with unmet medical needs. With the CIRM 2.0 processes that drove operational excellence and acceleration, there is incredible potential to build upon the recent early successes that led to projects that are nearing FDA marketing approval and to drive more of these successes to the benefit of healthcare and patients."

Question: "What are your plans for CIRM over the next few years?" 

Answer: "We are fully committed and motivated to executing on the 5 year strategic plan that we launched in January 2016. When we proposed these strategic goals, they were 'stretch' goals and now it looks like we will achieve these goals. This strategic plan is driven by our mission 'to accelerate stem cell treatments to patients with unmet medical needs.'"

'Right Person at Right Time:' Maria Millan Named as CEO of California Stem Cell Agency

Maria Millan at CIRM board meeting today, following her appointment as new president.
CIRM photo by Todd Dubnicoff

OAKLAND, Ca. -- Maria Millan, who joined the California stem cell agency five years ago, this morning was named as president of the $3 billion enterprise as it moves into what may well be its last three years.

Millan, who was serving as interim president,  was unanimously approved by the governing board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

CIRM Chairman Jonathan Thomas said he was enthusiastic about Millan when she was named interim president in June, when Randy Mills left. Thomas said he was "doubly enthusiastic" today and  described Millan as the "right person at the right time."

Millan was vice president for therapeutics and her appointment was expected. She will preside over  the agency in what some have called the "final stage" of the organization. It has projected it will run out of funds for new awards in mid 2020.  The agency is looking at various alternatives for future funding ranging from another bond issue, the agency's current financing, to possible some sort of merger or acquisition.

Millan this morning said the agency had $414 million in uncommitted funds after awarding $2.34 billion.

Millan's salary will be $550,000 retroactive to the beginning of July, when she assumed her new duties.

New President Coming Up for California Stem Cell Agency: Maria Millan Likely Choice

By this time next week, California's $3 billion stem cell research agency is expected to have a new president to preside over what may be the last act for the nearly 13-year-old effort.

Item Five on the agenda for this Thursday's meeting of the governing board of the agenda says:

"Consideration of appointment of new president, including compensation package."

Preceding that is an executive session of the board to discuss the matter behind closed doors. 

The California Stem Cell Report is predicting that Maria Millan, currently interim president, is likely to get the job. Millan was named as the interim CEO after Randy Mills left the agency at the end of June. Mills publicly recommended Millan to replace him as head of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. 

A subcommittee of the governing board has met only once -- also behind closed doors -- to consider a replacement for Mills. The board made no public comment about the outcome that July meeting.

Millan was vice president for therapeutics for the agency.  A news release from CIRM said in May that Millan "was instrumental in developing and implementing the current strategic plan."

The news release continued, 

"She received her general surgery training and transplant immunology postdoctoral fellowship at Harvard Medical School and trained in solid organ transplantation at Stanford University School of Medicine, where she went on to serve as associate professor of surgery and ultimately director of the Pediatric Organ Transplant Program. Maria received her bachelor’s degree from Duke University and her M.D. from the University of Medicine and Dentistry of New Jersey."

She joined the agency in 2012 after leaving a position as vice president at StemCells, Inc., which has received $9 million from the agency. 

CIRM has projected that it will run out of cash in mid 2020. Its governing board is currently studying a wide range of options for life after 2020.