The California Stem Cell Agency: 'Envy of the World, ' Hopes Too High?

The prestigious journal Nature yesterday published a piece about California's $3 billion stem cell agency that spoke of voids, envy and "double-edged swords."

The opinion piece was written by Jeanne Loring, a San Diego area

Jeanne Loring

researcher who has followed the agency for years and has been one of its beneficiaries($17 million in awards).

Reflecting on the agency's importance, she wrote,

"For the past dozen or so years, stem-cell researchers in California have been the envy of the world."

Creation of the agency, known formally as the California Institute for Regenerative Medicine (CIRM), "essentially guaranteed that the state would become the center of innovation in the field," Loring declared. Its demise would leave a major void, she said.

Loring continued,

"Although its intentions were laudable, CIRM raised the hopes of the public too high. It needed catchy advertising to gain voters’ support. One of its campaign slogans was 'Save lives with stem cells.' Effective advertisements often focus on a promise and downplay shortcomings, such as the time and resources required to advance a stem-cell therapy through clinical trials to market approval. No CIRM-supported therapy has been approved by the US Food and Drug Administration (FDA), resulting in dashed expectations.... 

"Still, fulfilment of the campaign promise is under way. CIRM has granted funding for 56 stem-cell-based clinical trials."

At the same time, dubious and unregulated clinics that peddle stem cell "snake oil" have proliferated across the country, leading the FDA to attempt a belated takedown of some of the enterprises.

The growth of those clinics is part of "the double-edged sword that is CIRM’s legacy,"  Loring said.

"The agency has enabled fundamental science and helped to establish know-how for rigorous assessment of stem-cell therapies. Earlier this year, my colleagues and I started a biotechnology company, Aspen Neuroscience in La Jolla, California, and are raising funds for a clinical trial of a neuron-replacement therapy for Parkinson’s disease. Without the work that CIRM has done to educate investors and researchers, this would have been very difficult. 

"But the agency’s work has inadvertently helped to boost unregulated, for-profit ‘clinics’ claiming, without sound evidence, that cells derived from fat, bone marrow, placenta and other tissues can cure any disease."

Loring said,

"CIRM has regularly denounced these clinics, which existed before the institute’s creation and will persist as long as they can make money. Still, it is easy to understand how public enthusiasm would spill over to those offering quackery."

Loring noted that the agency, which expects to run out of cash for new awards this year, is hoping that voters will give provide $5 billion more via a ballot initiative in November 2020. 

Loring urged rhetorical caution in the ballot campaign.

"We must strike a balance between future potential and current reality when we talk to the public. Researchers should emphasize that even when therapies show promise in mice, they often fail to work in humans. The only way to find out — and to check for safety — is rigorous scientific testing in clinical trials."

"We need to temper public hope," Loring wrote, while regulators, including the FDA and the California State Medical Board, bring the bad actors under control. 

Sacramento Bee, Royalties and California Stem Cell Payoff

The Sacramento Bee this morning carried an article by this writer on the California stem cell agency and its first $190,345.87, royalty check. The article is aimed at a more general audience including policy makers in the state Capitol. Here is a link to the piece, which carries this headline on The Bee web site:

 "Will California's $3 billion in stem cell spending pay off? First royalty check arrives"

The full text of comments summarized in the piece will be carried later today on the California Stem Cell Report. 

California Stem Cell CEO Millan on What's Missing in CIRM News Coverage

California's $3 billion stem cell research program suffers from the same sort of problem that arises at other state departments, ranging from Fish and Wildlife to Pesticide Regulation.

They are all struggling to gain the public's attention, tell their story and create support for their activities. The big difference is that Fish and Game and Pesticide Regulation are not likely to go out of business in two years. The stem cell agency, however, could well be on its way to closing its doors by then because it is running out of cash.

The California Stem Cell Report recently talked with Maria Millan, CEO and president of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. One of the topics was news coverage and how the agency is perceived, especially considering that voters may be asked to give the agency another $5 billion.

Millan was asked: What is the most "uncovered" story about CIRM, what is the "least written about?" Here is how she responded,

"I don't think people really have an understanding for what the value proposition of CIRM is as an agency. They know we're a funding agency, and I think it's best recognized for money."

Millan said that a lot of news coverage involves such things as "are you spending that money well, do you have enough oversight, do you have enough things in place to make sure that that's being done responsibly? There's a lot of focus on that."

"I think we've solved a lot of those issues and have things in place to catch issues and deal with them as they come up.... Part of it is that some of these assets didn't exist before and now we have them, and part of it is that the field has matured, and we've been positioned well to drive it."

Millan said that CIRM is "the model" for stem cell research and its funding. She said, 

"It's well-recognized that we do this very, very well. The NIH (National Institutes of Health) recognizes that we've been able in the space of stem cell regenerative medicine to do this extremely well. We have a portfolio in terms of development and our way of doing things that's unparalleled. 

"They recognize that. We're working with them to generalize this and it'll just be a two-way street in terms of benefiting for both sides of relationships."

Millan also talked about the value that her agency brings to the state, enhancing its position globally in biomedical research. She said,

"My goal is to make sure that we're responsibly sharing the knowledge, bringing things forward, because it's going to benefit California. I know that we want to make sure that we're responsible for optimizing our funds that they really support California directly, but there are a lot of things that can happen outside that will feed into us if we enable them."

Significant coverage in the mainstream media of the activities of the agency, created by voters in 2004, is rare nowadays. However, CIRM recently received some widespread attention within the state and nationally as the result of a lengthy assessment of its efforts on NPR and KQED's web sites.

The November 2020 ballot, which will include a presidential election, is the target date for the proposed $5 billion bond measure to keep CIRM alive.  Look for more intense coverage of the agency as that date nears.

(Look for more on the California Stem Cell Report on our conversation with Millan in the coming weeks.)

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.)

California Stem Cell Spending: Priming a $1.5 Billion Research Influx

California's $3 billion stem cell agency this week reported on "what you may not know" about the unusual, 13-year-old research organization. 

Its "disclosure" did not deal with such matters as the fact that it is the first such enterprise in state history or that it operates outside of the purview of the governor and the legislature. Rather it dealt with, among other things, leverage -- a term used often in the real estate business to describe, for example, using  $10 to create $1,000.

In the case of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known, it said it has created $1.5 billion in leveraged funds. It also noted that it has served as a "validator" for a significant amount of research, meaning that its awards re-assure the nervous nellies of finance about making an investment in a particular project. 

CIRM's seal of approval has led to $528 million in additional "partnership" funding for specific research, the agency said, along with more than $395 million in awards from other agencies that were built on CIRM-backed research. . 

News about the agency often focuses on stories about people and their afflictions. Those emotional tales are powerful ways to tell the CIRM story, but its less well known ventures into leverage and validation also speak to its impact, both short term and long term. 

Kevin McCormack, CIRM's senior director of communications, wrote about all this earlier this week with specific numbers and details in the agency's blog, The Stem Cellar, in a preview of the agency's annual report.

"Our goal is to do all we can to support the best science and move it out of the lab and into clinical trials in people. Obviously, providing funding is a key step, but it’s far from the only step. For us, it’s really just the first step."

CIRM does report its impact in the best possible light. However, there is little doubt that it has played an important role in "de-risking" much stem cell research and creating a friendly environment that is more likely to attract additional financing for a still young field. 

UC Davis Stem Cell Program Cited in Sacramento Bee

Here is one of the photos run by The Bee today with a story
about the UC Davis stem cell program.

The Sacramento Bee today published an article on the rise of UC Davis as a major stem cell research center, largely with a $130 million assist from California’s stem cell agency.

The news article was written by the editor of this blog, David Jensen, on a freelance basis. At the time of this writing, the article ranked No. 1 out of 81,000 results on a Google news search using the term “California stem cell.” 

While the piece focuses on UC Davis, a similar impact has been felt at UC Santa Barbara, whose stem cell program has also benefited greatly from CIRM largess.

Creating Critical Mass at a California Stem Cell Consortium

News about the California stem cell agency is dominated by such matters as $70 million Alpha stem cell rounds and clinical trials but other significant activities occur below that high level surface.

One such event is the creation of a state-of-the-art imaging facility at the Sanford Consortium in the San Diego, a facility that was built with the help of a $43 million award from the stem cell agency.

According to Terri Somers, a spokeswoman for the imaging facility,

“It is the only commercial location within California where these high-powered imaging modalities are available to researchers under one roof, along with pharmacology expertise and a deep reference library.”

The facility was opened last fall by Molecular Imaging, Inc., of Ann Arbor, Mich. While it provides services to the stem cell researchers at the consortium, its services are also available to researchers throughout the state, both private and academic.

Edward Holmes, president of the Sanford Consortium, said in a statement,

“We believe that in vivo imaging will play a critical role in tracking the effects of stem cells, and accelerate first-of-their-kind therapies to treat and cure some of the world’s most debilitating diseases.”

The facility includes one of the world’s most powerful MRIs, a PET, SPECT, CT, ultrasound and other molecular imaging technologies. It additionally provides as access to hundreds of disease models and a decades-deep expertise in pharmacology, according to the company.

Somers said,

“These technologies have been developed and leveraged in academic settings. Some global pharmaceutical companies have embraced the technologies as well, creating core-imaging facilities. However, drug companies don’t have this capacity on all their campuses, and none are in Southern California, making access for researchers here problematic.”

With the facility at Sanford, these services are more readily available to cash-strapped biotech companies on the entire West Coast.

Backers of the stem cell agency argue that one of its benefits has been to help build the critical mass in California that is necessary to support and attract stem cell research. The addition of the Molecular Imaging Center at the Sanford Consortium appears to be part that continuum. 

Two California Stem Cell Agency Directors Plump for Proposition 29

Two directors of the $3 billion California stem cell agency have popped up in the battle over the anti-tobacco initiative on tomorrow's ballot in the Golden State.

They are Sherry Lansing and Kristiina Vuori, who were the subjects of a column by Michael Hiltzik of the Los Angeles Times dealing with Proposition 29, the "Son of CIRM" measure that would raise $800 million for research by increasing the price of cigarettes by $1 a pack. In addition to serving on the CIRM board, Lansing heads her own anti-cancer foundation and is chair of the board of the UC regents. Vuori is head of the Sanford-Burnham Institute in La Jolla.

Proposition 29 is patterned after the measure that created the stem cell agency. The organization established by Proposition 29 would also be governed by a board that is run by representatives of organizations almost certain to receive the bulk of the funding, as is the case with CIRM.

In an op-ed piece on Friday, Lansing and Vuori said the Times and Hiltzik had fallen for "a smokescreen" put up by tobacco companies which are spending something in the neighborhood of $40 million to defeat the initiative. Lansing and Vuori said the measure is needed to stop smoking by young people as well as providing cash for research for tobacco-related diseases. Young people are more sensitive to price increases of cigarettes than adults, according to research.

Lansing and Vuori referred to a column in which Hiltzik opposed the measure because it would divert money from more immediate state needs, including health and welfare programs for children, education and the poor. (See here for thecolumn and here, here and here for related items.)

In his most recent column, Hiltzik said,

"The...problem with Proposition 29 is its pigeonholing of the money for cancer research rather than for immediate needs here in California that are absolutely dire. It’s all well and good to say that cancer research benefits everyone, but the real question is whether it should be the absolute top priority for a state that can’t afford to keep its children fed or offer them medical care in the here and now. 

"Lansing and Vuori say the fact that Prop. 29 'fails to provide funding for schools, roads or affordable housing' is irrelevant, because it was 'was never intended to solve these problems.'

"In the context of the state’s needs, this is a rather callous approach to take. Let’s spell out why, so Lansing and Vuori won’t be so inclined to dismiss these necessities of life so casually."

Hiltzik cited a list of state government cuts that have meant the loss of health coverage for 400,000 California children, eliminated welfare benefits for 578,000 poor California families and would mean an end to state college student aid for 72,000 young people from less affluent families.

Hiltzik continued,

"That’s just the beginning of what might be cut because the state needs money—and won’t be able to lay its hands on the hundreds of millions of dollars that Lansing, Vuori, and their research colleagues are angling for. They don’t want voters to be reminded that there are competing demands for the tobacco money, and they do so by failing to mention that they exist, and also by presenting the spending on cancer research as the voters’ only choice. 

"It’s the only choice because the promoters of Proposition 29 designed it that way. Advocates of programs like this love to pass them in via voter initiatives because they leave no room to measure them against alternative needs."

 A final note: The New York Times carried a piece yesterday on Proposition 29 that drew 481 comments. The article said, 

"Organizers argued that the tax would have less chance of passing if voters thought it would go into the state coffers, and said that their only goal here was cutting down on smoking."

 Also yesterday, Willie Brown, the former mayor of San Francisco and a keen observer of California politics, predicted voter approval of the measure along with an increase in cigarette smuggling from adjacent states and the sale of discount smokes at the 58 Indian casino sites in the state. 

Stem Cell Snippets: Surfing the Big Wave to Practicing PR

Trounson's Longings – CIRM President Alan Trounson says he "absolutely" misses being in the laboratory. Trounson (photo at right) made the comment in an interview late last month on Australian radio. He told interviewer Monica Attard on ABC radio that "I still wake up at night thinking I’m in the laboratory." Trounson also talked about how grant money was luring Australian researchers away from their country. "It’s like surfing in the big surf and unless you can catch the wave you’re never going to get the thrill of it. If you are sitting there and waiting for the wave to come and ... you might get on it, you might not because there’s really not much funding...." He went on to say that "a lot of really good scientists have relocated (from various places) to California because of the money and because of the opportunity."

Liberals Criticize Grant to California Stem Cell Firm – The state is New Jersey. The firm is StemCyte of Covina, Ca. The money relatively small – only $589,000. But a New Jersey state grant in that amount inveigled the company to open a New Jersey facility, creating 12 jobs. Questionable financial decision for the state, said the New Jersey Policy Perspective group, while higher education and transportation budgets are being cut in that state.

CIRM PR Contract – The California stem cell agency is looking for some public relations assistance, specifically a communications special projects manager. Under the terms of the RFP, compensation would amount to $90,000 under a 13-month "all inclusive contract including all administrative expenses and travel." The consultant would be required to work no more than 30 hours a week on an as-needed basis. Deadline for applications is May 19 with the award date scheduled for May 26 and possibly earlier. The agency has also posted job openings for a communications manager, IP attorney, scientific officer and two administrative coordinators (one senior).