Tuesday, September 11, 2018

SF Chronicle Backs Away From More Cash for California's Stem Cell Agency

The San Francisco Chronicle, which has the largest circulation of any newspaper in Northern California, today said the results generated so far by state's $3 billion stem cell agency "don’t argue for expanded public spending."

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, expects to run out of cash for new awards at the end of next year. It is seeking to raise privately $220 million to tide it over until November 2020, when it hopes that voters will approve a $5 billion bond measure for continued stem cell research.

However, in a formal unsigned editorial published on its website, the Chronicle said,
"The results to date don’t argue for expanded public spending. The science of stem cell work will need to evolve before more money is provided."
The editorial followed a four-part series  in the Chronicle looking into the state of stem cell research. The final installment last week said the agency had fallen short of the high voter expectations when they approved creation of the agency in 2004. The agency has not yet backed development of a therapy that is available for widespread use, although it currently has investments in 49 clinical trials.

The Chronicle, which says it has a readership of more than 500,000, said,
"California’s pioneering decision to spend $3 billion on stem cell research isn’t producing cures after 14 years of work. Instead, it’s generating a widening scientific field that shows potential but not results.
"It’s a frustrating shortcoming, especially as supporters of the state-sponsored research weigh another bond measure to continue the work. Promised breakthroughs used to sell Proposition 71 in 2004 aren’t panning out, a Chronicle investigation found."

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.

Thursday, September 06, 2018

San Francisco Chronicle: California's $3 Billion Stem Cell Program Does Not Measure Up to Voter Expectations

The San Francisco Chronicle, in a long and penetrating look at California's $3 billion stem cell agency, today said the research program has fallen "far short" of the promises made by its backers during the ballot campaign that created the effort.

Written by Erin Allday and Joaquin Palomino, the article said the agency, created by Proposition 71 in 2004, "can take credit for some notable progress," including saving the lives of children with rare immune deficiency diseases. Such efforts have been well supported by the agency, formally known as the California Institute for Regenerative Medicine (CIRM).

"But as thrilling as such advances are, they fall far short of what Prop. 71’s promoters promised." Allday and Palomino wrote.

"Not a single federally approved therapy has resulted from CIRM-funded science. The predicted financial windfall has not materialized. The bulk of CIRM grants have gone to basic research, training programs and building new laboratories, not to clinical trials testing the kinds of potential cures and therapies the billions of dollars were supposed to deliver."

Allday and Palomino worked on the CIRM overview for months, along with three other major pieces on stem cell therapies, both unregulated and those backed by the stem cell agency.  They reviewed the nearly 1,000 grants awarded by the agency and tracked the results, interviewing researchers and patient advocates and quantified the results.

The Chronicle series appeared as the agency nears its financial demise. It expects to run out of cash for new awards next year. The agency hopes that voters will approve a yet-to-be-written, $5 billion ballot measure in November 2020.

The Chronicle noted, however, that much of the research financed by the agency is not likely to resonate with voters.

Nonetheless, the article today contained ample information from the agency about its efforts, including its 49 clinical trials and some high profile results from those trials.  The piece posed the question of whether the nearly 14-year-old program has paid off. And it said,
"It’s not a question that can be answered simply. Science often can’t be measured in quantifiable outcomes. Failures aren’t just common, they’re necessary — it’s impossible to expect every dollar invested in research to lead down a traceable path toward success.... 
"It has helped make California a global leader in the field that’s come to be known as regenerative medicine. Anywhere significant stem cell research is taking place in the state, it almost surely has received support from CIRM."
The Chronicle quoted a member of the CIRM board who has been with it since its first days.
"'What was promised was not deliverable,' said longtime CIRM board member Jeff Sheehy, a former San Francisco supervisor. 'However, I would distinguish the promises from the impact and value. We have developed a regenerative medicine juggernaut.'"
The Chronicle also spoke with Bob Klein, a Palo Alto real estate investment banker who led the 2014 campaign.
"Klein...is unapologetic about the campaign he led. Indeed, as he lines up advocates and testimonials for the coming campaign, his message is familiar: Fund this research and we will save lives. Slow it down and the consequences will be grave.
"'Do you want your son to die? Are you going to wait?' Klein asked recently. 'Is that the price you are prepared to pay?'"
Today's Chronicle piece, roughly 5,000 words long, raises a host of important issues and deals with them in a nuanced and thoughtful manner. It is must reading for all those interested in California's stem cell research effort. 

(Editor's note: An earlier version of this item inadvertently omitted Palomino's name. Allday noted in an email to the California Stem Cell Report: "He played a HUGE role in putting together the CIRM story – he was basically solely responsible for collecting and analyzing the data from CIRM.")

Wednesday, September 05, 2018

'Better Than Hope' to 'No More Cash Without Changes' -- Overviews of California's $3 Billion Stem Cell Program

Looking for a relatively quick, current overview of the activities of the $3 billion California stem cell agency?

You can find it here on the California Stem Cell Report with the links below to recent items growing out of a state legislative hearing.

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

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.

California Stem Cell Agency Touts its Economic Impact on the Golden State

The California stem cell agency last week told its story to a state Assembly committee on biotechnology, including economic figures from a study financed by the agency itself. 

'Better Than Hope' -- An Advocate's View of California's $3 Billion Stem Cell Research Program

Longtime patient advocate Don Reed appealed to California lawmakers last week to support the state's stem cell research effort, which expects to run out of money for new awards at the end of next year.

A Before and After Story of $138 Million in California Stem Cell Cash

The head of the stem cell program at UC Davis says California is leading the way into a "new era of living medicine," thanks to the efforts of the $3 billion state stem cell agency.

State Lawmakers Hear Story of a Life-Saving Treatment and the California Stem Cell Agency

A father from Folsom, Ca., earlier this month told a state legislative committee about how his baby boy was saved through a clinical trial that was being financed by the Golden State's $3 billion stem cell agency.

California's Stem Cell 'Gold Rush:' Nearly 14 Years of Prospecting

A committee of the California Legislature today examined the state's $3 billion stem cell agency. Officials of the agency and others presented their perspective. Here is the text of prepared remarks by David Jensen, publisher of this blog, who appeared at the invitation of the Legislature. This is a third-party overview that deals with pluses, minuses and policy questions.

A Harsher Look at California's Stem Cell Program: No More Cash Without Changes

California's $3 billion stem cell experiment received a host of accolades last week at a state legislative hearing, but one strong, critical voice was not heard in the proceedings.

Monday, September 03, 2018

Exploring California's $210 Million Search for a Sickle Cell Cure, Plus an Online Look at Stem Cell Over Excitement

CIRM mounted another Facebook Live event last week, which has
chalked up nearly 2,000 views.
Hype, hope and sickle cell anemia -- all were part of Internet videos last week involving the $3 billion California stem cell agency.

The San Francisco Chronicle mounted one and the stem cell agency the other. The Chronicle's production was hosted by Erin Allday, the reporter at the newspaper who wrote its ongoing series, The Miracle Cell, dealing with stem cell treatments. (The next installment is scheduled for publication on Thursday and is expected to evaluate the agency itself.)

Allday asked Maria Millan, CEO of the agency, and Paul Knoepfler, a researcher at UC Davis, about the juggling act needed when the benefits and progress of stem cell research are discussed. Both Millan and Knoepfler said it is easy to get overexcited by the progress of stem cell research.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), produced the second video, a Facebook Live event that so far has drawn more than 1,900 views. Similar figures were not available for the Chronicle video.

Featured on the CIRM video are Donald Kohn of UCLA and Mark Walters of the research institute at Children's Hospital Oakland, along with patient advocate Adrienne Shapiro. The agency has invested $210 million into efforts to find a cure for the disease that afflicts 80,000 people nationally.

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