Save the Goose: California's Stem Cell Story and the 'Army of 900'

Is the stork bringing stem cell billions?

More post-election coverage is trickling out regarding the activities of the $12 billion California stem cell agency, including a lengthy and compelling piece in the Los Angeles Times, the state's largest circulation newspaper. 

The hitch is that the Times piece does not mention the stem cell agency until the 21st paragraph, a fact that carries more weight than the 40 words in the paragraph itself. 

The article in the Times, written by columnist Sandy Banks, was not really about CIRM per se. But it was a story that might not have been told without a $13 million check from CIRM. 

The piece dealt with sickle cell disease. Banks' entry point was a patient named Evie Junior, whose harrowing experiences are certain to resonate with nearly all the paper's readers. 

Banks wrote,

"As a child, (Junior) grew accustomed to frequent hospital stays. But the disease got progressively worse as he moved through his teens; the bone pain was so disabling, he often had to be sedated with heavy-duty opioids."

Banks continued,

"At one point, the bones in both his legs were so damaged by the disease, doctors thought he might not ever walk normally again. Junior battled back."

Banks wrote,

“'I want to be cured of this disease,' he said. 'And I wish the world was more understanding about the people who are struggling with it.'”

Junior is a patient in a UCLA clinical trial led by Donald Kohn, whose research has been supported by a total of $52 million from the stem cell agency -- a figure not noted in the story. 

The stem cell agency comes into the story ever so briefly at paragraph 21. "The trial is funded by the California Institute for Regenerative Medicine (CIRM), the stem cell agency created by voters in 2004 and infused with $5.5 billion in new research money by voters who narrowly approved Proposition 14 this fall," the Times reported.

That is the first and last mention of California's stem cell program in the Times' story. However, that is better treatment of CIRM than is found in most stories in the media over the last few years -- the ones that deal with accomplishments tied to the agency's cash. (See here and also here.) The articles rarely mention where the money comes from. It could be flown in by stork, for all the readers know. 

Why is this important? The answer has to do with CIRM's reliance on tenuous, ballot-box financing and the sustainability of the California stem cell program.  The situation should be of interest to a little known state entity, which is the only state panel that has a legal charge to review the stem cell program.  

The panel is the Citizens Financial Accountability and Oversight Committee (CFAOC). It was created by the same ballot initiative that established the stem cell agency 16 years ago. Last month, at the CFAOC's once-a-year meeting, its members urged the agency to make itself better known. 

Proposition 14 saved the agency from financial extinction. But the measure was a squeaker, and it set the stage for another life-or-death ballot initiative. The money from Proposition 14 runs out in about 11 years. No other source of funding is provided. If CIRM goes to the ballot again in less than a decade or so, it will need a strong base of support from California voters, 8.2 million of whom voted last month to cut off funding for CIRM.

Building a solid base takes years. It also requires something of a change involving the scientific community and science journalism, which is a big ask. The tradition in scientific journals regarding funding is to relegate mention of the sources of money to a tiny footnote as if it were not the lifeblood of research.

Science writers -- the few that now exist in the mainstream media -- follow that ancient and tired public tradition of ignoring what makes research happen. They focus on "exciting" results from the bench and clinic -- not the "filthy lucre" that pays scientists, their institutions, the post-docs who labor behind the microscopes, the equipment suppliers and keeps the lights on.

Recipient institutions write the news releases -- the starting point for most journalists -- without mentioning the many, many billions that taxpayers (state and federal) have provided. Even the stem cell agency itself does not mention dollar amounts in its blog items dealing with significant results from CIRM research. (For an example, see this item from the agency's blog on the UCLA/Kohn sickle cell trial, which CIRM is funding with $13.1 million. That figure is not directly revealed in the blog item.)

Like most state agencies, CIRM is ignored by the media with only occasional exceptions. Its activities are not well known. That is not likely to change over the next decade despite the best efforts of CIRM's tiny staff. The exception would be a major or even minor scandal or development of a major breakthrough that would resonate with the people of California.

The folks in the media have a vast array of topics to cover, all of which CIRM competes with for attention. The news industry is, in fact, overwhelmed by matters that need public scrutiny: schooling for children, jobs for parents, affordability for housing, vaccines for Covid-19, homelessness, climate change, wildfires and much, much more. Meanwhile, the industry is struggling with its own sustainability issues, laying off thousands of reporters, closing outlets and desperately searching for new business models.

Breaking through this wall of issues to generate favorable, regular coverage of the stem cell agency's good works is a herculean task. And so far, the reality is that CIRM's good works are quite minor in terms of how they affect the lives of the vast majority of the people in California. 

The CIRM team, from top to bottom, has provided a prodigious amount of positive information that can resonate with the public. Building positive relationships with voters, however, is a long-term task that requires commitments that go well beyond CIRM's stalwart staffers, who currently weigh in at only 33 for a multibillion dollar program. 

CIRM has major budget and legal limitations, courtesy of Proposition 14. Its outreach team is small, again an outgrowth of ballot initiatives. Nonetheless, CIRM has a potential army of more than 900 available plus more in the future, given the agency's expansive, new responsibilities authorized by Proposition 14. And that doesn't count the patient advocates or their allied organizations.

The army of 900 consists of the researchers and institutions that have received cash from CIRM -- all potential missionaries who can carry the CIRM message from Yreka in the north to Calexico in the south. 

The institutions could highlight CIRM funding in their news releases, instead of burying or omitting it. Researchers could reach out regularly to the various affected communities to help keep the cash flowing. Grantees could pony up $2,000 each and send it off to a new, broad-based nonprofit that might be called something like "Friends of CIRM."

But more immediately, the army of 900 could -- one by one -- resolve to make six new contacts in 2021 with the public or media to tell the CIRM story. Doing so would serve science and CIRM not to mention themselves.

The next statewide election best suited for approval of another bond measure is fast approaching. It is only eight years away. That is much less time than it takes to complete the preclinical work and clinical trials needed for a federally approved stem cell treatment, which is, ultimately, the only thing that voters really want. 

If the army of 900 needs an inspirational motto, it could be something like "Save The Goose."  You know, the one that lays the Golden Eggs. It is better than relying on the stork. 

'Demeaning and Uncalled For:' A CIRM Complaint About the Use of the Word 'Obscure'

The California stem cell agency today sharply criticized an item on the California Stem Cell Report as "demeaning and uncalled for" because it described the four-person Citizens Financial Accountability and Oversight Committee (CFAOC) as "obscure."

The offending word was used in an article yesterday that previewed the first meeting of the committee in nearly 15 months. The CFAOC was created as part of the initiative that created the stem cell agency in 2004, which is known officially as the California Institute for Regenerative Medicine (CIRM). 

The CFAOC reviews CIRM financial documents that have already been reviewed in public by CIRM's 29-member governing board, which is officially known as the Independent Citizen's Oversight Committee (ICOC). The CFAOC has no legal authority over CIRM beyond making possible recommendations.

The article in question began like this:

"One of the more obscure entities in state government is scheduled to meet this Friday to review the financial affairs of California's stem cell agency, a matter that now runs to nearly $12 billion.

"The panel is the Citizen's Financial Accountability Oversight Committee (CFAOC), which was created in 2004 along with the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known. The CFAOC is charged with reviewing the financial practices of the stem cell agency. The CFAOC is the only state panel legally charged with that function. The governor and the legislature have no authority to do so."

Here is the text of the email expressing CIRM's displeasure with the use of the word "obscure." The email was written by Kevin McCormack, senior director of communication at CIRM.

"I think your characterization of the CFAOC in your latest column as 'One of the more obscure government entities' was demeaning and uncalled for. It may not have a high profile but it certainly has an important role. It was created by the voters in 2004 for a very specific purpose, to make sure we are held accountable, every year, for our financial performance. As one of the common, if erroneous, complaints about CIRM is that we are not accountable to the legislature or any other legislative body in Sacramento I would have thought the CFAOC should be praised not dismissed.

 

"This is a group of people who come together with the sole goal of making sure the state’s money is well spent. The members cover a broad range of professions but have one thing in common, they have vast experience in their area. They are appointed by the Controller, the Treasurer, the President pro Tempore of the Senate, and the Speaker of the Assembly (and one by CIRM). These are nobody’s fools, they are diligent in the way they work, meticulous in their analysis of how we work, and forceful in making recommendations. Which we then follow.

 

"At CIRM we are used to people criticizing us, more often than not incorrectly. The CFAOC deserves better."

As the author of the article in question, I have high regard for the CIRM staff and McCormack as well as the persons on the CFAOC. Nonetheless, it is an obscure state entitity, as are many state departments. More than 200 exist and most of them are obscure. Consider the State Allocation Board, for example, or the First 5 California. 

The count of 200 does not include state advisory bodies such as the CFAOC. If it did, the number would likely run into the thousands. Such bodies can do important work, such as was done by the Little Hoover Commission, an obscure state entity whose analysis and recommendations concerning CIRM won significant praise by the Institute of Medicine (IOM), which studied CIRM for months. 

What all these bodies have in common is that they are virtually unknown to the public, even to persons who consider themselves well-informed. 

The California Stem Cell Report writes about the CFAOC because of its connection to CIRM and the fact that both were created by the same 2004 initiative -- a measure criticized as ballot-box budgeting and that sometimes has also created impediments for CIRM. (See the IOM findings and recommendations.)

My bottom line? For a state body to be considered obscure is not a mark of shame. It is just a matter of fact.  

(Editor's note: A very early version of this item incorrectly said that Friday's meeting of the CFAOC will be the first in 19 months. The correct figure is 15 months.)

 

California's $5.5 Billion Stem Cell Campaign and Covid-19: How Science and Politics Marry

Editor's note: The following item written by the producer of the California Stem Cell Report appeared yesterday on the Capitol Weekly online news service. 

**********

The California stem cell agency has just finished pumping $5.3 million into the fight to save the lives of COVID-19 victims. And — in a ballot-box bonus — its efforts are already surfacing in the ballot campaign to rescue the agency from its own demise.

The agency is running out of money. It will begin closing its doors this fall without major financial support that it hopes will come from Proposition 14, a $5.5 billion bond measure on the November ballot.

The $5.3 million in the fast-track, Covid program is tiny by comparison. But the awards are likely to play a heftier role than might be anticipated based on their size. Stories about the Covid awards have appeared on the campaign website in a move to tap into the deep public concern about the coronavirus.

The Covid research piggybacks on the federal “warp speed” drive to develop Covid treatments and is aimed at demonstrating that the agency is part of what CIRM officials describe as a worldwide, “all-hands-on-deck” effort.

Three clinical trials have been funded, which involve actual patients as opposed to laboratory research. If the trials are successful, patients who have benefited are likely to surface in campaign video advertising aimed at refinancing the agency, formally known as the California Institute for Regenerative Medicine (CIRM).

CIRM sorely needs research results that will resonate with voters. The 2004 ballot campaign that created the $3 billion program raised expectations that stem cell therapies were right around the corner. But the agency has yet to produce a stem cell treatment that is widely available to the public.

The agency launched its Covid round on March 27 when CIRM board chairman Jonathan Thomas declared, “The coronavirus is creating an unprecedented threat to all of us, and, as one of the leading players in regenerative medicine, we are committed to doing all we can to develop the tools and promote the research that will help us respond to that threat.”

Indeed, failure by CIRM to respond to the COVID-19 threat — it could be argued — would be a breach of CIRM’s public responsibilities.CIRM’s Covid research financing is a case where an urgent medical need marries nicely with ballot-box politics. The agency is forbidden by law to campaign for the ballot measure. But its charter also allows it to pursue a wide range of research possibilities.

Late last month, CIRM directors capped off the round by approving two awards and adding nearly $300,000 to the original authorization of $5 million. All of the awards have been made under an emergency process that requires awardees to be ready to begin research within 30 days and achieve “a clear deliverable within six months.”

The final awards went for basic level research. Earlier, CIRM approved funds to assist in the three clinical trials, which are the final stage before a treatment can be approved for widespread public use. The trial awards range from $1 million to $701,000 each, a bargain price for joining a trial. Most of CIRM’s previous clinical trial awards run into many millions.

CIRM now counts a total of 64 clinical trials that it is helping to finance. Two of the Covid trials are taking place at the University of California, San Francisco-UC Davis and the City of Hope in the Los Angeles area. The third involves a New Jersey firm called Celularity, Inc., which has failed to respond to multiple questions about the California location of its trial. The agency is limited to funding activities within the state. CIRM directors approved the Celularity award on June 26, more than 30 days ago.

The Celularity award has another interesting angle. It involves a treatment supported by President Trump’s personal attorney, Rudi Giuliani. 

You can find more information about all the awards in a series of items on CIRM’s blog, The Stem Cellar, using the search term “covid.”   Five CIRM/Covid stories, including CIRM blog items about the Covid round, have been placed on the campaign website by the campaign staff. 

*********

​Read all about California's stem cell agency, including Proposition 14,  in David Jensen's new book. Buy it on Amazon:  California's Great Stem Cell Experiment: Inside a $3 Billion Search for Stem Cell Cures. Click here for more information on the author.

Covid-19 and Stem Cell PR: Informing Voters, Loosening Their Pursestrings

The California stem cell agency often has trouble making it into mainstream media coverage, but last week it popped up in a piece involving its special Covid-19 round. 

Some of the "normal" grants from the agency run upwards of $20 million. But in this case, it involved only $701,049. The award carried extra news heft for the San Francisco Chronicle because it went to a local institution, UC San Francisco, and was connected to the hottest medical story in the world. 

Michael Matthay of UCSF was the recipient of the cash on May 15 from the California Institute for Regenerative Medicine (CIRM). Matthay has a phase two clinical trial 

Michael Matthay
UCSF photo

underway for acute respiratory distress syndrome, which can kill patients with severe cases of Covid-19. He is using intravenous doses of mesenchymal stem cells (MSC) to help damaged lungs repair themselves. Results will not be known for probably about a year. 

The award will largely be used to expand the clinical trial to UC Davis. 

In a letter to the CIRM board, Matthay said, 

"UC Davis serves a population that includes underserved patients, and therefore adding UC Davis as a clinical site will serve the CIRM mission well. Also, please note that our UCSF Alpha Stem Cell application was focused on underserved patients in the East Bay (Sickle Cell Disease) and in San Francisco (Zuckerberg San Francisco General Hospital)."

Running the Davis site will be Rachael Callcut, who led the site at San Francisco General until joining Davis in March, and Tim Albertson,  chair of internal medicine at UC Davis. 

Rachael Callcut
UC Davis Photo

In  Matthay's letter he defended his application (CLIN2COVID19-11823) against criticism by the anonymous reviewers who evaluated it for CIRM. The application was initially not approved by the reviewers, who gave it a score of 72 out of 100. The score was so low that it fell outside the range of normal funding. Among other things, the review summary said that reviewers disagreed about the rationale of the research and expressed concerns about whether enough Covid-19 patients could be enrolled. 

Matthay said in a letter to the CIRM board that 11 Covid patients were already enrolled. As for the rationale, he said, 

"There is substantial pre-clinical data that MSC therapy can decrease lung injury from bacterial and viral pneumonia and hasten lung repair. Our group and other investigators have reported several pathways by which MSCs can reduce inflammation in the lung." 

After receiving the letter and hearing from Matthay via telephone at the meeting, the board overrode the reviewers' action and approved the proposal. 

While Matthay's work received favorable notice in the Chronicle article (written by J.D. Morris), for CIRM it was a mixed bag. The first mention of the state funding said, 

"California has invested billions of dollars in public funds to advance stem cell therapies over the last 15 years or so and, while researchers have made some important progress, a 2018 Chronicle series found that the advancements did not live up to the original promise of the 2004 ballot measure that funded the scientific endeavor."

CIRM fared somewhat better in the next mention in Morris' story: 

"Matthay has no guarantee that the stem cells he’s studying will be useful in treating COVID-19. But it’s worth a shot, said Jonathan Thomas, the board chairman of the regenerative medicine institute. 

"'Given that we’re dealing with something the world’s never seen before, it behooves everybody who has potential ideas to be heard,' Thomas said. 'The notion of stem cell-related treatments ... is something that at least we have to give the opportunity to have a full consideration.'"

Why does the play in the story, its use of photos (three in all) and wording matter?  CIRM is all but unknown to California voters today.  As the proposed, stem cell ballot initiative gains more attention, ongoing media coverage will become increasingly important. It will shape the perceptions of voters who are going to be asked in November to provide $5.5 billion more for CIRM, which is running out of money. The agency has handed out nearly all of the $3 billion that voters provided it in 2004. 

The CIRM story has plenty of plenty of pluses and minuses. CIRM and its backers hope that voters will see more pluses than minuses in the media and vote to continue to finance the agency's search for stem cell treatments for everything from cancer to incontinence. 

A Call for $5.5 Billion More and 'Silence' from Scientists

The story of jCyte, Inc., the California stem cell firm that notched a $252 million deal last week, contains a chapter that might be titled  "Golden Eggs and Buttering Your Bread."

What makes that chapter significant is that it involves steps taken by jCyte to give some credit to California's stem cell 'goose,' so to speak: The creature that has laid $2.7 billion in the laps of hundreds of scientists and institutions over the last 14 years. JCyte readily acknowledges and publicizes the importance of the $34 million that the California stem cell agency has provided directly and indirectly to the firm and its founders. 

"What?" you might ask incredulously. "Isn't that to be expected?" 

Well, no actually, based on the performance of institutions and researchers over the past few years. When they issue news releases about major developments in research funded by the agency, most often the state's beneficence is omitted or buried.  And that means that the state stem cell agency's financing is also not mentioned in the subsequent news stories about the research.  (See here and here also.)

The California Stem Cell Report holds no brief for or against the agency and its quest for $5.5 billion more to save it from financial extinction. But should the multibillion-dollar initiative reach the ballot in November, California voters deserve a full and robust exploration of the pluses and minuses of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. 

If the seemingly tongued-tied institutions and researchers do not speak up about whether they think CIRM is worthy, they do not serve the public well or their own self-interest. The same applies to CIRM's opponents, who owe it to themselves and to voters to make a well-reasoned, fact-based case for letting the agency slowly expire. 

The stem cell agency is all but invisible to the voters of the Golden State. The media rarely write or broadcast stories about it nowadays, which has been the practice for the past decade or more. And CIRM is certainly a much different creature than it was 10 or so years ago.

JCyte deals with rare eye diseases. It is also rare among CIRM recipients in that it clearly recognizes the critical role that the agency has played in its short life. The firm's web site attests to that impact on its "missions" page.  Its news release on the $252 million deal demonstrates that as well. And it was demonstrated again yesterday morning when its president, Paul Bresge, was quoted on the CIRM blog as saying, 

“jCyte is extremely grateful to CIRM, which was established to support innovative regenerative medicine programs and research such as ours.  CIRM supported our early preclinical data all the way through our late stage clinical trials.  This critical funding gave us the unique ability and flexibility to put patients first in each and every decision that we made along the way. In addition to the funding, the guidance that we have received from the CIRM team has been invaluable. jCell would not be possible without the early support from CIRM, our team at jCyte, and patients with degenerative retinal diseases are extremely appreciative for your support.”

Bresge clearly understands where his bread is buttered, so to speak, and what produces the golden eggs. We do not intend to say that in any kind of demeaning way. The financial component of scientific research is far below the radar of the overwhelming majority of voters. It is up to those who have something at stake to strive diligently to explain how the money works and sustains science. No cash. No research. 

The deal with jCyte has big numbers attached. Not all research results can say that. Most are considerably less dramatic as far as the public is concerned. But researchers -- if they are to well serve themselves and their institutions -- should be clear with the public that they would not be doing much research in any area if state and federal funding did not exist. Informing voters is an incremental task and slow. One bit of information on top of another.  It requires perseverance and patience. Just like culturing something in a petri dish. Not just ballot campaigns every 15 years.

Quarter-of a-Billion Dollar 'Salute' to California Stem Cell Agency; Japanese Firm and jCyte Sign Big Licensing Deal

A California stem cell company with major backing from the state's stem cell agency received a $252 million boost today when a Japanese firm cut a deal to develop the firm's therapy for an eye disorder that affects nearly 1.9 million people globally. 

The California company is jCyte, Inc., of Newport Beach. It has received $15 million from the California stem cell agency over the last few years to develop a treatment for retinitis pigmentosa. Its co-founder, Henry Klassen of UC Irvine, has received an additional $19 million from the agency for his work that led to creation of jCyte. The Japanese firm is Santen Pharmaceutical Co. Ltd., a publicly traded firm. 

In a comment for the California Stem Cell Report, Maria Millan, CEO of the agency, formally known as the the California Institute for Regenerative Medicine (CIRM), said, 

"This is exciting news for everyone at jCyte. They have worked so hard over many years to develop their therapy and this partnership is a reflection of just how much they have achieved.

"For us at CIRM it’s particularly encouraging. We have supported this work from its early stages through clinical trials. The people who have benefitted from the therapy, people like Rosie Barrero (see video above), are not just patients to us, they have become friends. The people who run the company, Dr. Henry Klassen, Dr. Jing Yang and Paul Bresge, are so committed and so passionate about their work that they have overcome many obstacles to bring them here, an RMAT designation from the Food and Drug Administration, and a deal that will help them advance their work even further and faster. That is what CIRM is about, following the science and the mission."

In its news release, jCyte said, 

"The treatment is a minimally-invasive intravitreal injection, which can be performed in an ophthalmologist’s office with topical anesthetic. The entire procedure takes less than 30 minutes. The principal mechanism of action is the release of neurotrophic factors that may rescue diseased retinal cells. jCell therapy aims to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated."

The company's statement continued, 

"Under the terms of the licensing agreement, jCyte will receive $50 million in upfront cash, $12 million in a convertible note offering, and $190 million in clinical and sales milestones based on regulatory approval and initial sales in Europe, Asia and Japan. The total deal is valued at up to $252 million. jCyte is also entitled to receive tiered, double-digit royalty payments on net sales of jCell therapy once commercialized outside the U.S."

Currently there no federally approved treatments for the rare, genetic affliction. In its clinical trials, jCyte has treated more than 100 patients,, and the federal government has given it a regenerative medicine advanced therapy designation based on the early results. That designation is aimed at speeding further federal review.  The company says the treatment could have application in other degenerative retinal diseases, including age-related macular degeneration and diabetic retinopathy.

In another rare instance, jCyte featured CIRM's role in funding the work. The agency was cited in the fifth paragraph of the company news release. The firm's web site also contains a glowing acknowledgement of the importance of CIRM support. In virtually all news releases from businesses or academic institutions, the role of CIRM's funding, which totals $2.75 billion over the last 14 years, is omitted. 

The deal with jCyte undoubtedly will be cited during possible efforts this fall to keep CIRM operating. It is running out of cash and is hoping a proposed $5.5 billion ballot initiative will be approved by voters to keep the agency operating.

Klassen co-funded the company along with Yang, also of UC Irvine. The cells used in the treatment are manufactured at a facility at UC Davis that was financed with funds from the stem cell agency. 

Kaiser Healthline: California's Stem Cell Program Short on Cash and Cures

An overview by Kaiser Healthline of California's nearly 15-year-old stem cell research program received attention in California and nationally last month. 

"Despite Failed Promises, Stem Cell Advocates Again Want Taxpayers To Pony Up Billions" said the headline on the article by Ana Ibarra.

Her piece carried both positive and negative comments on the performance of the $3 billion agency, which is running out of cash for new awards. The enterprise hopes voters will approve in November 2020 another $5.5 billion to continue its work.

The article quoted Marcy Darnovsky, executive director of the Berkeley-based Center for Genetics and Society, a longtime critic of the agency, which was created through a ballot initiative campaign in 2004. 

Darnovsky said that if the agency, known formally as the California Institute for Regenerative Medicine (CIRM), is measured against the campaign promises, “then CIRM has been a flop.”  

The agency, however, pointed to its 56 clinical trials, along with the stories of patients who have benefitted from that research, ranging from children who are alive today as the result of experimental treatment to spinal cord injury victims who are seeing their lives improve. 

Ibarra's article was picked up nationally by Salon. Politico noted it in its California newsletter.  The Sacramento Bee, the only daily newspaper in the state capital, published it as well.  

'Tough Lesson' and Stem Cells: More Time, More Money Urged -- Indirectly -- for California's Research

BioInformant graphic

Time and money, hope and hard work -- not to mention death -- were the topics today on the blog of the $3 billion California stem cell agency. 

The blog item involved more than the demise of patients waiting for a stem cell therapy but also the possible demise of the nearly 15-year-old program, unique in California history. 

The research effort, formally known as the California Institute for Regenerative Medicine (CIRM), is running out of cash. By the end of this year, it expects to have no funds for new awards. 

That situation led to a piece this morning on CIRM's  blog, The Stem Cellar. It was a bit of indirect pitch for continued funding by California taxpayers.

"Time and money are always going to be challenging when it comes to advancing stem cell research and bringing treatments to patients. With greater knowledge and understanding of stem cells and how best to use them we can speed up the timeline. But without money none of that can happen."

The article by Kevin McCormack, senior director of communications for CIRM, recapped the history of the agency, created by voters in 2004 through a ballot initiative that set the state off on the largest scientific research effort of any state in the nation. Indeed, as McCormack pointed out, the funding surpassed research budgets of some nations. The catch in California was that no source of funding for the agency was provided beyond the initial $3 billion. 

CIRM noted the high hopes for quick therapies back in 2004. 

"In the early days there was a strong feeling that this was going to quite quickly produce new treatments and cures for diseases ranging from Parkinson’s and Alzheimer’s to heart disease and stroke. Although we have made tremendous strides we are still not where we hoped we’d be. 

"It’s a tough lesson to learn, but an important one: good scientific research moves at its own pace and pays little heed to our hopes or desires. It takes time, often a long time, and money, usually a lot of money, to develop new treatments for deadly diseases and disorders."

McCormack briefly catalogued some of CIRM's progress and the 56 clinical trials in which it has invested, some of which are in the final stage before federal approval of a treatment. 

But CIRM said, 

"The simple truth is that unless we, as a nation, invest much more in scientific research, we are not going to be able to develop cures and new, more effective, treatments for a wide range of diseases." 

The agency is hoping that voters will approve a ballot initiative in November 2020 that will provide $5.5 billion more for stem cell research. In the meantime, it has $71 million to hand out.

WebMD and the Rising Framework for a $5.5 Billion Stem Cell Request in California

USC researchers Mark Humayun (right) and Amir Kashani,
CIRM-funded scientists. Click here to go to their research video.

WebMD, a heavily used Internet health and medical site, this week assessed the state of the stem cell field in a two-part series that highlighted much of the clinical work backed by California's $3 billion stem cell agency.

The first installment by Kathleen Doheny offered a national overview, declaring that stem cell research has been underway in significant way for three decades. "Where are we now?" was the headline on the article. Perhaps the key sentence declared,

"While proponents say all this groundwork is finally coming to fruition, others call progress slow and plodding."

WebMD is a go-to site for the public when it looks for medical information. In 2016 it reported that it had nearly 180 million unique visitors per month. Today, it says one out of every four Americans uses its site every month.

The series led with work at USC that is being assisted with millions from the California Institute for Regenerative Medicine(CIRM), as the stem cell agency is formally known. It also noted CIRM's 56 clinical trials and its clinical dashboard. 

CIRM grantees were mentioned, including Andy McMahon, Amir Kashani and Mark Humayun, all of USC and recipients of CIRM awards.

The second part of the series, authored by Karen Weintraub, focused on the unregulated and dubious "stem cell" treatments that are the target of both federal and California state regulators.  The headline said, 

"Stem Cell Clinics: Effective or Pricey False Hope?"

One might ask whether readers of the piece are distinguishing between those sorts of sketchy clinics and California's Alpha stem cell clinics, which are very much the pride of the state research effort. 

The WebMD coverage is part of the framework that is taking shape as the California stem cell agency sees its funds coming to an end. It is hoping for a $5.5 billion infusion from voters via a ballot measure in November 2020. How the public perceives stem cell treatments overall and how voters perceive the success of the state effort are likely to be critical in winning approval of more stem cell cash in California. 

Embedding the Power of Stem Cell Therapies: California's Efforts and the Quest for $5 billion More

Everett Schmitt, photo by Meg Kumin

California's $3 billion stem cell agency has financed a lot of research that winds up as dense, inaccessible articles -- at least to the general public -- in costly scientific journals with imposing pay walls.

Sometimes, however, stories of hope and stem cell progress do emerge that are likely to resonate with the state's citizens. They are the folks who voted to create and finance the agency in 2004 and who are expected to be asked again for more billions next year.

One such example involves the cases of Evangelina Padilla-Vaccaro of Corona, Ca., Ronnie Kashyap of Folsom, Ca. From birth they suffered from what is known as the "bubble boy" disease.

Both children were born with severe combined immune deficiency (SCID) that is ordinarily fatal. But as the result of clinical trials that were backed with millions from the stem cell agency, both are still alive today. (See here and here.)

The agency is formally known as the California Institute for Regenerative Medicine (CIRM). It has a blog called The Stem Cellar. Today it published an evocative piece written by the mother of another child with the "bubble boy" affliction. 

In her case, the work that has saved her son's life was not financed by CIRM. But the agency carried her story as a powerful affirmation of the importance of stem cell research, wherever and however it is supported. 

Kevin McCormack, director of communications for the agency, wrote, 

"CIRM's mission is very simple: to accelerate stem cell treatments to patients with unmet medical needs. Anne Klein's son, Everett, was a poster boy for that statement. Born with a fatal immune disorder Everett faced a bleak future. But Anne and husband Brian were not about to give up. The following story is one Anne wrote for Parents magazine. It's testament to the power of stem cells to save lives, but even more importantly to the power of love and the determination of a family to save their son."

Anne Klein's article is just what supporters hope will be embedded in the hearts of voters come November 2020 when the next round of financing for CIRM will be on the ballot. 

These sorts of stories, however, have received little attention in the mainstream media, whose reporting resources are ever-diminishing. The media are hard-pressed financially and otherwise nowadays as their once highly profitable business models have been hard hit by the impact of the Internet. 

Nonetheless the agency is assiduously pumping out information and stories on what it calls its value proposition. Here is the final line in the item that CIRM published today:

"You can read about the clinical trials we are funding for SCID here, here, here and here."

UC Researcher on Stem Cells and California's Stem Cell Agency

The California stem cell agency recently highlighted a brief look at the field with a Q&A that ranged from the impact of the agency itself to dealing with questions about "miraculous" stem cell "cures."

The item appeared on the agency's blog and originated at UC Davis, which is among the top five recipients of funds from the agency, known formally as the California Institute for Regenerative Medicine (CIRM).

The school has received $143 million, making it fifth on the list of recipients in terms of dollars.

The Q&A involved Jan Nolta, head of the stem cell program at UC Davis. Some excerpts from what Nolta had to say:

Jan Nolta, UC Davis photo

"Perhaps the most promising and exciting research right now comes from combining blood-forming stem cells with gene therapy. 

"Along with treating the famous bubble baby disease, where I had started my career, this approach looks very promising for sickle cell anemia. We’re hoping to use it to treat several different inherited metabolic diseases.... 

"The beauty of this therapy is that it can work for the lifetime of a patient. All of the blood cells circulating in a person’s system would be repaired. It’s the number one stem cell cure happening right now. Plus, it’s a therapy that won’t be rejected. These are a patient’s own stem cells. It is just one type of stem cell, and the first that’s being commercialized to change cells throughout the body."

About the five Alpha stem cell clinics initiated by CIRM, Nolta said,

"These are clinics where the patients can go for high-quality clinical stem cell trials approved by the FDA [U.S. Food and Drug Administration]. They don’t need to go to 'unapproved clinics'a and spend a lot of money. And they actually shouldn’t."

More News Reports on Halt in California Stem Cell Funding Applications

The journal Science this week joined the publications beginning to report on the financial travails of the $3 billion California stem cell agency. 

In a piece by Jocelyn Kaiser, the journal briefly summarized the agency's activities and its outlook for the future. Kaiser wrote, 

"Some researchers who explore the basic science of stem cells had already been looking for other funding sources as (the agency) began to emphasize clinical work and their support wound down. But others, especially those planning clinical trials, will be hit hard.

April Pyle, UCLA photo

"'It’s going to be a huge impact on my lab and many others if they end,” says April Pyle of UC Los Angeles (UCLA), whose 11-person group works on using muscle stem cells to treat muscular dystrophy. Her last CIRM grant ends in March 2020 and although she also has some NIH funding, it does not support the animal testing and other studies needed to move her work toward a clinical trial."

CIRM is the abbreviation of the official name of the stem cell agency, the California Institute for Regenerative Medicine. 

Pyle has received $4.6 million from CIRM and UCLA $289 million, according to the agency's figures.

Kaiser also wrote, 

"Ongoing payments for approved projects continue, but scientists are already tightening their belts for a funding gap. They are also contemplating the end of a boom in stem cell research in the state. California’s voters may be asked to renew CIRM with another bond initiative next year, 'but there’s no guarantee,' says Arnold Kriegstein, who heads a stem cell center at the University of California (UC), San Francisco, and has received CIRM funding in the past."

Kriegstein has received $4 million from CIRM and UC San Francisco $192 million.

The shutdown of CIRM applications was first reported by the California Stem Cell Report on June 20.

Others have recently followed, in one form or another, including The Scientist, Genome Web, Capitol Weekly, National Review, The Beacon, Spine Review and LifeNews.

Live Online: The 'Inside Scoop' on $3 Billion, California Stem Cell Research Effort?

The headline was provocative, and the question was "now what?"

It is the latest posting on the blog of the $3 billion California stem cell agency, which expects to run out cash for new awards as early as this fall. 

"Getting the inside scoop on the stem cell agency" -- That was the headline for the article, which promoted an online event July 25 involving three of the directors of the nearly 15-year-old agency, formally known as the California Institute for Regenerative Medicine (CIRM). 

"Now what?" is one of the questions they will address during the Facebook Live session. Viewers will have a chance to submit questions during the event and hear the answers. The immediate response is one of the benefits of such an event. Another is that they are preserved online for later viewing, multiplying the potential exposure in a significant way. 

That staying power is a benefit of considerable utility for the agency, which is not exactly a topic at the breakfast table in the homes of California voters. But they are the folks who the agency hopes will approve a proposed ballot initiative in November 2020 for an additional $5.5 billion for the research program. 

CIRM is currently engaged in a bit of an extra effort to educate Californians about the positive aspects of its work. In recent years, it has functioned in the usual obscurity enjoyed by most state agencies. However, unlike most state agencies, it does not survive financially on the usual budgetary process. 

CIR was born in 2004 with $3 billion, but nothing more.  So today the task is demonstrate to the people of California its value proposition. 

Taking up that task online in a couple of weeks will be CIRM directors Anne Marie Duliege, executive vice president and chief medical office Rigel Pharmaceuticals; Joe Panetta, president of BIOCOM, and Dave Martin, chairman and CEO of AvidBiotics. And CIRM is inviting Californians to join in the Facebook Live session "to understand how we got where we are, how the rest of the field is doing and what happens next."

Hard News for Patients, Scientists: California Shuts Down Applications for Its Stem Cell Research Funding

The California's stem agency's rundown on its clinical
trials. Some of the trials have saved lives. 

The $3 billion California stem cell agency today served up the bad news with only a smattering of sugar coating.

No more applications for research funding are being accepted. The cash is running out, perhaps as early as the end of August.

In a posting on its blog, The Stem Cellar, the agency declared,

"It’s never easy to tell someone that they are too late, that they missed the deadline. It’s particularly hard when you know that the person you are telling that to has spent years working on a project and now needs money to take it to the next level. But in science, as in life, it’s always better to tell people what they need to know rather than what they would like to hear."

The news is no surprise to persons who follow the agency. But today brought a more clearly emerging sense of finality.

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, was created in 2004 by voters who also provided $3 billion in bond funding. However, the CIRM ballot measure also contained the seeds of destruction for the agency. No other cash was provided. No other significant means of funding was laid out.

Today, the agency is pinning its hopes for survival on a yet-to-be-written ballot initiative for the November 2020 ballot. To bridge the gap between now and then, CIRM has been attempting for months to raise privately more than $200 million. So far, no results have emerged publicly. 

As of last month, the agency had in its pipeline applications for $88 million in research funding. But it had only $33 million left for new awards. 

CIRM reports it has enough cash on hand to administer its portfolio of awards, which stretch out a couple of years.

Fifteen days ago the agency quietly announced the application shutdown. Little public notice of the action was taken even in California's stem cell community, which has grown mightily over the nearly 15-year life of the agency. 

During that period, CIRM has helped finance 55 clinical trials targeting diseases ranging from cancer and heart disease to diabetes and arthritis. It has served up 1,015 research awards. The scientists it has supported have published more than 3,000 research papers. 

However, CIRM has yet to fulfill the campaign-generated expectations of the 7,018,059 voters in 2004 who voted to create it and who thought they would see new, widely available, miraculous cures. Impressive results, some of which have saved lives, have surfaced from some of the clinical trials. But the elusive stem cell cure that would be ready for the general public is yet to hit the streets.

The Oakland-based agency is not done yet nor is it out of business.  Its reviewers are expected to meet later this month to make the de facto decisions on some of the pending applications. And then again in August. 

More needs to be done in terms of the private fundraising effort. And more needs to be done in crafting a new ballot measure that would bring $5.5 billion to CIRM.  

In the CIRM blog item today, written by Kevin McCormack, senior director of communications, the agency declared, 

"Over the years we have built a pipeline of promising projects and without continued support many of those projects face a difficult future. Funding at the federal level is under threat and without CIRM there will be a limited number of funding alternatives for them to turn to.

"Telling researchers we don’t have any money to support their work is hard. Telling patients we don’t have any money to support work that could lead to new treatments for them, that’s hardest of all."