Thursday, January 25, 2018

California's Stem Cell Story Gains National Attention on NPR

California's $3 billion stem cell research effort broke into national news this morning when National Public Radio (NPR) picked up a lengthy overview of the Golden State's 13-year-old program to develop therapies that could treat everything from cancer to incontinence.

While the story chronicled the pluses and minuses of the work of the California Institute for Regenerative Medicine (CIRM),  as the agency is formally known, overall the piece bolstered the agency's efforts to tell its story to millions of more persons. It could be said to fit into the category of "I don't care what you say about me, just as long as you spell my name right."

NPR says it reaches 99 million people monthly across its platforms.  Roughly 12 million of those persons are likely to be found in California, given that the state has about 12 percent of the nation's population.

The stem cell agency piece first appeared last week in California on the web site of a popular media outlet, KQED, which produces TV and radio news. KQED's syndicated California Report is expected to air a broadcast version of the story and has linked online to the longer version as well.

All of this meets the need of the agency to reach the millions of California voters who are likely to vote on a possible, $5 billion bond measure in November 2020. The agency is slated to run out of money by then and will whither away without additional cash.

While some in the media would be loath to admit it, they tend to run in packs, chasing the same stories for a variety of reasons. The widespread appearances of the article by David Gorn could well stimulate additional media coverage in the shorter term and also help to shape coverage over the next couple of years or so.

Wednesday, January 24, 2018

A Man Named Lucas and the Future of the California Stem Cell Agency

Lucas Lindner, photo by Cait Covers the Bases
The news last fall from a California stem cell firm was leaden and dense. Terms such as "Phase 1/2a SCiStar study" and "Full enrollment of Cohort 3 (AIS-A; 20 million AST-OPC1 cells)" littered the company's statement. 

Today the state's $3 billion stem cell agency turned the jargon into a heart-warming, human story -- the type that is critical to sustaining the life of the 13-year-old research effort, which is running out of cash.

The story involves Asteria Biotherapeutics, Inc., of Fremont, the California Institute of Regenerative Medicine(CIRM), as the stem cell agency is formally known, a clinical trial for a treatment for spinal injury and a young man named Lucas Lindner.

He is one of the patients in Asterias' clinical trial for a human embryonic stem cell therapy for spinal cord injury.  CIRM has funneled $21 million into the research. 

Writing on the agency's blog, Kevin McCormack, senior director for CIRM communications, said,
"On a Sunday morning in early 2016, Lucas Lindner was driving to get some donuts for his grandmother. A deer jumped in front of his truck. Lucas swerved to avoid it and crashed, suffering a severe spinal cord injury that left him paralyzed from the neck down."
But after the treatment, McCormack wrote,
"Lucas can now type 40 words a minute, use a soldering iron and touch his pinkie to his thumb, something he couldn’t do after the accident.
"In August of last year Lucas did something else he never imagined he would be able to do, he threw out the first pitch at a Milwaukee Brewers baseball game. At the time, he said 'I’m blown away by the fact that I can pitch a ball again.'"
Of course, there are scientific caveats and qualifications in the story. But what the tale really can do is resonate with nearly all California voters if the story ever reaches them.

Those voters are key to continuing the work of the agency, which expects to run out of state funds in 2019 and is hoping that voters will pump an additional $5 billion into the agency through a bond measure on the November 2020 ballot. (A private fund-raising effort is underway to bridge the cash gap.)

The agency has largely functioned in obscurity during the last 10 years or so. Its story is not well known by the public. And it has yet to help finance a therapy that is available to the general public. But CIRM's 44 clinical trials, including the one by Asterias, provide hope for patients and the agency.

The key lies, however, in how the story is told and how well CIRM supporters can turn mind-numbing scientific jargon and public policy issues into compelling yarns that will open both the hearts and purses of California voters.

Friday, January 19, 2018

California Makes a $25 Million Kidney Transplant Wager: Its 44th Stem Cell Clinical Trial

The California stem cell agency this week more than doubled down on its bet on a potentially breakthrough treatment for kidney transplants, raising to $25.4 million its support for a project that is entering its final stages.

The hope is that the treatment will not only improve the success rate of kidney transplants but also lead to use in liver, heart and other solid organ transplants. If successful, the therapy would eliminate the need for immunosuppressive drugs in genetically matched kidney transplant patients.


Maria Millan, CIRM photo 
Maria Millan, president of the agency, said in a news release:
“These immunosuppressive drugs not only can cause harmful side effects, but they are also expensive and some patients lose their transplant either because they can’t afford to pay for the drugs, or because their effectiveness is not adequate."
The award also could help save the life of the stem cell agency, which is facing its financial demise as its funding runs out.

The award brings to 44 the number of clinical trials being assisted by the $3 billion agency, formally known as the  California Institute for Regenerative Medicine(CIRM). Clinical trials are the last stages of research prior to certification by the federal government of a treatment for widespread use.

The stem cell agency is hoping that one of its trials will soon produce a therapy that will resonate with California voters who may be asked in 2020 to provide $5 billion more. The agency expects to run out of state funding next year and is attempting to raise more than $200 million privately to tide it over until a bond election in  November 2020.

Action on the award was swift on Thursday. It took less than eight minutes for the agency governing board to unanimously approve an $18.8 million award to Medeor Therapeutics, Inc., of San Mateo, Ca. The award comes on top of a $6.7 million investment in the firm's research in 2016.

Steven Deitcher, Medeor photo
The vote simply ratified a decision on the Phase 3 trial that was already made by the agency's reviewers, who gave the research strong support during a meeting behind closed doors weeks earlier.

Steven Deitcher, co-founder and president of Medeor, said in the agency's news release,
"CIRM funding accelerates our timelines, and these timelines are what stand between needy patients and potential transformative therapies. This CIRM award combined with investor support represent a public-private collaboration that we hope will make a difference in the lives of organ transplant recipients in California, the entire U.S., and beyond."

Tuesday, January 16, 2018

A Look at Whether California's Stem Cell Agency Can Carry the Day Two Years From Now

One of California's more respected news operations today posted an overview of the state's $3 billion stem cell research agency, asking the question:
"Should California Voters OK $5 Billion More for Stem Cell Research?"
The piece by David Gorn on KQED's web site recapped the history of the program, its current condition and included comments from Robert Klein, the man behind the $5 billion proposal and who is often regarded as the father of the agency.

Barbara Koenig
 UCSF photo
Also included were comments from folks not always heard from publicly in the discussions about the agency. Quoted was Barbara Koenig, head of the bioethics program at UC San Francisco, who said she supports stem cell research but voted against the ballot measure that created the agency in 2004. She said,
“I didn’t like the overhyping of the immediate idea that there were cures around the corner.  I think we need to be honest about how we’re investing in research.”
Gorn continued,
"Ask Koenig how she might use that proposed $5 billion differently, and she responds with a moment of stunned silence.
"'Oh my, so many things,' she said. 'I would try to figure out how to make sure every child in California has access to basic health services, nutrition, clean water . . . not just make high-priced products, but to improve public health.'
"She said stem cell research privileges these quick-fix biotech approaches, which may make a lot of money but may not benefit the general public.'". 
Gorn's piece had some reach into the general public in the Golden State. In addition to its online news site, the San Francisco-based KQED operates a public TV station including news and  a public radio station with syndicated programming throughout the state. A version of today's story is expected to run next week on KQED's California Report, which the station says reaches 775,500 persons each week.

Gorn's report was pretty much down the middle, exploring the expectations for cures that emerged from the 2004 campaign directed by Klein, who also oversaw the writing of the initiative. His view is that that agency has "out-achieved" the promises of the campaign. Gorn wrote that Klein said that the campaign "never promised cures during the lifetime of the stem cell agency, only progress toward attaining them."

The governing board of the California Institute for Regenerative Medicine, as the agency is formally known, has all but endorsed Klein's current plan to place another initiative on the 2020 ballot, a presidential election year that will generate the large turnout that tends to favor bond measures.

Bonds -- money that the state borrows -- are the only significant source of cash for the agency. Ironically, the measure that created the agency -- also provided the seeds of its death by terminating its bonding authority after a specified period. Today the agency is down to its last few hundred million.

Zev Yaroslavsky, UCLA photo
Bonds additionally are usually used for long-lived assets such as roads and buildings and roughly double the cost of the research to taxpayers because of the interest expense. 

Gorn concluded with a comment from Zev Yaroslavsky, an authority on state politics and government at UCLA, as saying  that even if voter attitudes are highly favorable toward stem cell research that may not be enough to carry the day.

Yaroslavsky said,
"People do see stem cell research as something they have a stake in, but you’re going to have to explain what we got with the first $3 billion. I suspect their case with the voters will be that we need to keep momentum going. But the question is, ‘Will they buy it?’"

Friday, January 12, 2018

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. 

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