California Stem Cell 'Renewal:' The Search for More Billions -- And Credit for Results

A KTVU video connected to CIRM's research support

California's 13-year-old stem cell agency, which is facing its demise as it lingers in a dim media shadow, last week snagged some credit for the work it has helped to finance in saving the life of a five-year-old girl. 

The news story appeared on San Francisco Bay Area television station KTVU. The piece involved Evangelina “Evie” Padilla Vacarro, who was born with what is known as the "bubble baby" disease. That is a genetic affliction that compromises the immune system so severely that it is nearly impossible for a person to survive.

Evie's story is powerful and well-known among those who closely follow the $3 billion California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known. But it has not seeped in the consciousness of California voters, who are likely to be asked to cough up $5 billion more for CIRM.

The agency expects to run out of cash by the end of next year. It is pinning its survival hopes on a private fundraising drive underway this year and passage of a proposed bond measure in November 2020. However, many competing priorities exist for that sort of funding. Plus the agency has yet to fulfill voter expectations that creation of the agency would lead to a stem cell treatment that would have widespread use.

In Evie's case, the treatment is available under exceedingly limited circumstances, like other treatments that the agency is supporting. They are still being tested before the federal government approves them for wider use.

The positive results that are, in fact, surfacing often do not mention CIRM's substantial backing, or they bury that fact so deeply it is all but invisible. (See here, here and here.) That is occurring despite the fact that many of those stories emerge from institutions that have received tens and tens of millions of dollars from the agency.

The fresh news peg for the KTVU story last week was the launch of a book, “California Cures: How the California Stem Cell Program is Fighting Your Incurable Disease," by longtime stem cell patient advocate Don Reed. The station reported,

"'Lives have been saved, and suffering eased, because California stood up for stem cells in 2004,' Reed said. 'Now as we approach the end of that (2004) voter-approved program, it is vital that everybody knows the story of (the institute) and why we must renew its funding.'"

Whether that "renewal" actually takes place may depend on whether the researchers and institutions that have benefited from CIRM's largess do a much better job of selling what they believe are the benefits and importance of the agency's programs and cash.

CIRM Board Member Sheehy Exiting as San Francisco Supervisor; Says SF Voters Favorable on Stem Cell Work

Jeff Sheehy, a member of the governing board of the $3 billion California stem cell agency, will be leaving his fulltime post as a member of the San Francisco board of supervisors later this year, but he says his unsuccessful election campaign disclosed favorable perceptions of stem cell research.

Sheehy disclosed his plans not to seek re-election in the media in the Bay Area. In a statement, he said it was "immensely rewarding" to have served the city. He also had this to say yesterday to the California Stem Cell Report,

"I have been asked about the implications for CIRM (the stem cell agency) and, based on polling around my bio that my campaign conducted, stem cell research remains very popular.

"When we asked voters about what prior work of mine resonated with them, the tagline, 'Fights for stem cell cures for deadly diseases and conditions as a founding board member of the California Institute for Regenerative Medicine,' was one of, if not the most popular items listed in my background.

"I had multiple conversations with voters about our work at CIRM and they were very supportive and very impressed with the what we have accomplished."

Sheehy is a patient advocate member of the stem cell board and has served on it since its inception in 2004. He is also chair of the Science Subcommittee of the board, participates in closed door reviews of applications for funds by scientists and oversees board discussion of the reviewers' decisions.

Sheehy's term on the 29-member board expires in 2020. The agency expects to run out of cash for new awards by the end of next year, 2019.

CRISPR, the Cancer Question and the California Stem Cell Agency: $13.5 Million Investment in Eight Projects

CRISPR was in the news this week, and it wasn't about how you like your French fries cooked.

CRISPR, for those of you who don't know already, is a relatively easy way to edit genes. The technique is used in at least eight research projects backed by the California stem cell agency, totalling about $13.5 million.

What made CRISPR generate the headlines was research -- as Business Insider reported -- that the "blockbuster gene-editing tool has been linked to cancer." The assessment was described as hype by some scientists.

Paul Knoepfler, the peripatetic stem cell blogger and researcher at UC Davis, explored the implications of the findings in an item earlier this week. He wrote,

"To say that CRISPR-Cas9 gene editing might 'cause cancer' seems premature to me so there probably were and are better ways for people to phrase the big-picture meaning of these new papers and others like them that may pop up in the future. However, to be clear safety is crucial, so this is an important development and risks of any potential therapy including gene editing-based approaches should be carefully weighed against potential benefits as trials are designed and then progress."

The California Stem Cell Report asked the agency, which monitors the research it funds, for a list of its projects involving CRISPR. Here is what the agency provided, including the disease focus, principal investigator and grant number.

• Sickle cell disease, $4.5 million, Childrens Hospital Oakland Research Institute, Mark Walters, TRAN1-09292
• Muscular dystrophy, $2.2 million, UCLA, April Pyle, DISC2-08824
• Vision loss, $232,200, UC San Diego, Karl Wahlin, DISC1-08683
• Neurological disorders, $1.9 million, Parkinson's Institute, Birgitt Schuele, DISC2-09610
• Blood disorders, immune disease, $984,228, Stanford, Rosa Bacchetta, DISC2-09526
• Blood disorders, $1.5 million, UCLA, Caroline Kuo, DISC2-10124
• Cystic Fibrosis, respiratory disorders, $2 million, Stanford, Matthew Porteus, DISC2-09637
• Blood Cancer, cancer, $235,800, UC Berkeley, Jacob Corn, DISC1-08776

Not a Dr. Ruth Explainer: California Stem Cell Agency's FAQ

Looking for a reasonably straight forward primer on the $3 billion California stem cell agency, a state program that is unique in California history and now in its 13th year of life? 

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), has compiled an FAQ (frequently asked questions) list which is available on its web site. Obviously, some of the material is colored by agency's own perspective, but the basic facts are there in one handy document.

The questions deal with such things as "when will there be cures," "shouldn't private industry fund stem cell research" and "what happens when the money runs out."  It's not exactly a Dr. Ruth's guide to all things CIRM, but many may find the document helpful.

Fire, Fury and $5 Billion: A Mini Preview of a Ballot Campaign for the California Stem Cell Agency

California's stem cell research effort, which is pinning its survival hopes on a proposed $5 billion bond measure in a couple of years, was slammed in a national publication last week as a "multi-billion dollar money suck."

The column in the conservative magazine National Review, which has about 90,000 circulation and a significant online presence, was a tiny preview of the fire and fury that is likely to erupt around the likely pitch to California voters in 2020 to give more cash to the agency.

Formally known as the California Institute for Regenerative Medicine (CIRM), the agency was created by a ballot initiative in 2004 and backed with $3 billion in state funds. It expects to run out of money for new awards by the end of next year.

Wesley J. Smith, a longtime critic of the agency, wrote in the National Review piece,

"The mendacious (2004) campaign promised Cures! Cures! Cures! with embryonic stem cells and therapeutic human cloning — even promising that disabled children would get out of their wheel chairs and walk. Good grief, campaigners also claimed that the money earned from all the coming cures would reduce California’s health-care budget.

"Some $2 billion later, none of it came to pass. Tens of millions were spent on a fancy-dancy building. Conflicts of interested have abounded. But the supposed point of the CIRM was not achieved. There have been extremely few human trials with embryonic stem cells — mostly dealing with eye conditions — and not all were CIRM-funded."

It is fair to say that Smith's characterizations omit much information about what CIRM calls its value proposition. Nonetheless, his points are likely to resonate with a substantial portion of California voters, who have seen little mainstream media coverage of CIRM. 

Even the institutions and recipients of multi-million dollar research awards regularly fail to note CIRM's contributions in their news releases about state-backed scientific discoveries. (See here and here.)

As of today, the agency has invested in 49 clinical trials, the last stage before a therapy is approved for widespread use. A discovery or treatment that would captivate the public could emerge from those trials between now and the election in November 2020. Meanwhile, given the nature of today's financially struggling media and limited science coverage, the agency and its backers are likely to find it tough to break through the news clutter and convince voters to cough up more cash.