$5.5 Billion Stem Cell Ballot Measure: California Backers Taking First Formal Step This Month

CIRM graphic

Backers of a California ballot initiative to provide $5.5 billion more for the state's cash-strapped stem cell agency say they will take their first official step by the end of this month. 

That's when they will submit the proposed measure to California election officials and trigger a many-months-long process. The effort is aimed at ensuring that the nearly 15-year-old research effort survives in a meaningful way beyond next year.

The stem cell agency, officially known as the California Institute for Regenerative Medicine (CIRM), expects to  run out of money for new awards as early as late October.  It has already cut off grant applications except for a joint sickle cell effort involving the National Institutes of Health. 

Robert Klein, a Palo Alto real estate investment banker, and his stem cell advocacy group, Americans for Cures, are leading the way on the initiative. Melissa King, executive director of the organization, told the California Stem Cell Report last week that the wording of the initiative is still being crafted.

King said, however, that she expected the measure to be submitted to the California secretary of state before October. She said that one area still being drafted involves providing assistance for low income patients and their families in rural areas who do not have the wherewithal to travel to and participate in clinical trials.  

The initiative filing will trigger a lengthy process to gather the  633,212 signatures of registered voters necessary to qualify the  proposal for November 2020 ballot. Gathering those signatures is a task performed by specialized firms. It could cost something in the neighborhood of $5 million or more, which Klein will have to raise on his own.

Many more signatures than the minimum are gathered because significant numbers of the signatures are commonly determined not be valid.

The ultimate deadline for qualification is June 25 of next year, 131 days before the Nov. 3 election. But the signatures will have to be submitted well before then.

The stem cell agency was created in 2004, also by a ballot initiative, with $3 billion in state bond funding. The new initiative will likewise use bond funding, which roughly doubles the cost to taxpayers because of interest expense on the borrowed cash.

The 2004 ballot campaign was supported by Hollywood stars and Nobel Prize-winning scientists. It generated grand expectations that stem cell therapies were right around the corner. While CIRM is currently involved in 56 clinical trials, it has yet to back a therapy that is widely available. Clinical trials are the last step before a medical treatment is approved for widespread use and have a high failure rate. 

Klein led the campaign in 2004 and became the first board chairman of the agency. Klein has been gathering information, statistics and support for the effort at his Palo Alto offices. 

In June, he told the California Stem Cell Report:

"This medical revolution holds the promise of restoring health and quality of life for many of California’s individuals and families suffering from chronic disease and injury.

"However, the last tactical mile to bring this broad spectrum of therapies to patients will require more funding and the thoughtful support of California’s public as the human trials and discoveries are refined and tested, overcome numerous obstacles or complications, and ultimately serve to improve the life and reduce the suffering of every one of us."

Pressures for Stem Cell Profits and Cures: A Case from Japan with Implications for California

A stem cell treatment in Japan for spinal cord injury is raising a ruckus about ethics, efficacy and billion-dollar searches for cures and profits.

The matter involves a therapy called Stemirac and Sapporo Medical University. The treatment is now available to the public in Japan with most of its $140,000 cost covered by Japan's national health insurance program. 

The most recent overview of Stemirac came yesterday on a site called "Undark" in an article written by Amos Zeeberg. In the piece, Arnold Kriegstein, director of the stem cell program at UC San Francisco, called Stemirac  "essentially an unproven therapy."  Bruce Dobkin, a UCLA neurologist, was reported as saying "the results briefly reported in the media may suggest the treatment doesn’t even work."

(Undark's site says it is a "is a non-profit, editorially independent digital magazine exploring the intersection of science and society."  Its publisher is Deborah Blum, a former colleague of this writer at The Sacramento Bee. Blum is now director of the Knight Science Journalism Program at MIT.) 

In the piece yesterday about Stemirac, Zeeberg wrote, 

"It’s arguably the world’s most ambitious approved stem cell treatment and should have been a cause for celebration: a long-awaited breakthrough for the field of regenerative medicine — using modern biological tools to repair the body — and a harbinger of more impressive medicines."

He continued, 

Arnold Kriegstein
UCSF photo

"Instead, the therapy has been met with a heated debate. On one side, many experts have slammed Stemirac’s approval in uncommonly direct terms, saying there isn’t enough evidence to show it is effective or even safe. The treatment went through an expedited approval unique to Japan: After short, small clinical trials that suggest safety and efficacy, regulators can approve stem cell treatments on a conditional basis — allowing use of the treatments for seven years, while sponsors gather additional evidence to support a full approval.

"Critics also say Japan’s approach is far too soft — that early approvals allow patients to take experimental therapies that could be ineffective or dangerous, at a high cost to both patients and insurance providers. 'This is essentially an unproven therapy,' said Arnold Kriegstein, a stem cell researcher at the University of California, San Francisco. 'I’m very surprised this is happening in a country like Japan.'..."

The Undark article continued, 

Bruce Dobkin
UCLA photo

"What’s more, UCLA neurologist Bruce Dobkin told Undark, the results briefly reported in the media may suggest the treatment doesn’t even work. Dobkin pointed to previous trials testing other potential treatments for spinal cord injuries, and the Stemirac findings 'are exactly the results we found in patients in randomized controlled trials — in the control groups,' he said. That is, patients injected with Stemirac seemed to do as well as patients who got placebos in these earlier trials. He says people who have recently suffered spinal cord injuries, like Kusachi, the injured high diver (mentioned in Zeeberg's piece), and the others in the Stemirac trial, often have significant natural improvement over the next several months — exactly the period covered by the trial. It’s possible the patients were simply healing naturally, he says, but without a control group and double-blinding, it is hard to tell."

Undark's article captures many of the issues surrounding the development of stem cell therapies including the pressure to generate results for patients and profits for companies -- not to mention prestige and praise for researchers.

California's $3 billion stem cell program feels that pressure as well. It is running out of cash and would more than welcome a breakthrough in one of its 56 clinical trials -- one that would stimulate California voters to provide more billions for the state's nearly 15-year-old stem cell research effort.

'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.

Coming Up Next Month: Final Award Decisions by California's $3 Billion Stem Cell Agency?

The California stem cell agency, which is running out of cash for new awards, has cancelled a meeting this week at which it was scheduled to approve funding for more clinical stage research.

The action sets the stage for a full discussion and decisions by the agency board in late September on its last likely research awards unless it receives a substantial infusion of cash. Known formally as the California Institute for Regenerative Medicine (CIRM), the agency was created by voters in 2004 with $3 billion. That has dwindled to $71 million, although $28 million is committed to sickle cell anemia.

CIRM is pinning its hopes for survival on a possible ballot initiative in November 2020 to provide it with another $5.5 billion. 

In response to a question about the status of the meeting once scheduled for this Thursday, Kevin McCormack, CIRM senior director of communications, noted that the last of the clinical stage applications are being reviewed this month. He said,

"There may be more projects to fund than money."

McCormack said,

"We will be bringing all meritorious projects to the board in September to make funding decisions.  In addition, the full board (not the Application Review Subcommittee) must decide whether or not to redirect money to the Translation bucket in order to fund  those that were not funded last month."

CIRM's funding squeeze generated considerable discussion at the July meeting, including appearances by scientists seeking awards. The full transcript of that session can be found here. 

The application review subcommittee is a subset of the full 29-member board, which includes a number of representatives from institutions that receive awards. The subcommittee was created to avoid the appearance of conflicts of interests on the part of the CIRM board. 

September's meeting will be based at the Oakland headquarters of the agency. It is also scheduled to be audiocast live with Internet access. Additional physical locations are expected to be announced for teleconference access. The public can participate in the meetings via the Internet and at the teleconference locations. 

The "Hard Case" of Stem Cell Sound Bites and a Campaign to Raise Billions

The journal Nature today reported on the $5.5 billion plan to save what it called the "struggling" California stem cell agency, which is fast running out of cash for new research awards. 

In an article by Jonathan Lambert, Nature caught up with news that it is not so new to readers of this web site. 

The piece carried information from Robert Klein, who expects to lead a new ballot initiative effort late next year to provide the billions more for the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. 

Nature also had this from a man who served on the Institute of Medicine team that evaluated the California program in 2012.

Aaron Levine, Georgia Tech photo

"Aaron Levine, a science-policy researcher at the Georgia Institute of Technology in Atlanta, says that CIRM has put California at the center of stem-cell research worldwide. 'CIRM has been really important in driving stem-cell research forward, especially in the preclinical and proof-of-concept space,' he says. 

"But he isn’t sure whether that will convince voters to keep supporting the agency." 

The Nature article continued,

"'It will be interesting to see if the campaign is one of promised cures, or something a bit more nuanced,' says Levine. 'It takes time for a whole new field of research to result in cures, but that’s a hard case to make to voters in short sound bites.'"

Nature said Klein told it that he will form a non-profit lobbying arm in October or November to help support the campaign next year. 

The journal also said changes in the stem cell research program were anticipated in the new ballot initiative.  

"These include creating a dedicated staff of 10–15 people who would work with insurance companies and patients to improve access to clinical trials and future therapies."

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."

A Stem Cell Crossroads in California: The Viewpoint From USC

The University of Southern California, co-sponsor of a meeting this week in Los Angeles of 4,000 stem cell researchers and others, has offered up a perspective on its program and the crossroads facing California. 

The lengthy piece by Gary Polakovic captured more than the work being done USC, which has received $111 million in funding from the state stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM). 

He touted the program at USC but also looked at the state of stem cell affairs in the Golden State.  

Keying off the annual meeting of International Society for Stem Cell Research, Polakovic, research communications manager at USC, wrote, 

"California has proven fertile soil for stem cell research. The state has assumed a leadership role in stem cell science since voters approved Proposition 71 in 2004, which seeded the industry with $3 billion in bond funds. The program is administered by CIRM, which contributes about 30 percent of USC stem cell funding."

The article continued, 

"With progress comes growing pains, and California’s stem cell program is at a crossroads.

"On one hand, gains in the lab have moved stem cell therapies closer to making a significant impact on medicine. Yet, the complexity and cost of cellular medicine has proven a big challenge. Scientists acknowledge it will be difficult to cure major diseases with stem cells. The gap between hype and hope has narrowed, but not closed.

"'Hype can be right, but it’s the time frame when people

Andrew McMahon, USC photo 

expect things to happen that can be wrong,' (Andrew) McMahon (director of the USC stem cell program) said. 'Curing cancer, diabetes, Alzheimer’s and other diseases is taking longer and involves a lot more complication and funding. The progress has been astounding — but it’s never fast enough.'"

Polakovic also tackled the difficult financial condition of the state stem cell agency. He wrote, 

"State funding for stem cell research under CIRM is expected to run out this year. The $3 billion ballot initiative that voters approved — Proposition 71, the California Stem Cell Research and Cures Act — is substantially depleted. Other sources, such as federal funding, private investment and philanthropy, are available but not necessarily dedicated to statewide research. CIRM funds have played a big role in creating and sustaining the USC stem cell initiative.

"Researchers are hopeful California voters will have an appetite to continue funding. Backers of Proposition 71 are planning a $5 billion measure for the November 2020 ballot. With research gains and clinical trials underway, backers are hopeful California will continue to support progress for another decade.

"Yet, voter perception of stem cells could be colored by rogue clinics peddling dubious wonder cures like snake oil. Those businesses operate outside the realm of leading research institutions such as USC. More than 100 such stem cell clinics operate in California alone. The Food and Drug Administration is stepping up enforcement actions against clinics offering unapproved stem cell products that endanger the public.

"At the same time, the momentum toward stem cell therapies at USC and other universities is undeniable. On the trail to finding breakthroughs for big diseases, basic research has unlocked a host of co-benefits — many unforeseen when California embarked on its stem cell program 15 years ago — that are valuable to medicine."

The ISSCR, California Stem Cell Financing and Silence

It comes as no surprise that the largest organization of stem cell scientists in the world is in favor of "rigorous funding" for stem cell research and warns of the perils of decreased financial support. 

That organization is International Society for Stem Cell Research (ISSCR), which is meeting in Los Angeles later this week and expects 4,000 persons to attend. 

California's stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), has long been a supporter of the ISSCR. In 2009 it contributed $200,000 to help out with the ISSCR's annual conference. 

Times have changed, however, since those halycon days 10 years ago. It is running out of cash for new awards. This year CIRM contributed "only" $50,000 to help stage the group's annual meeting. A few days ago it cut off applications for new research awards beginning next Monday. It needs support for $200 million in private "bridge" funding to continue its program while it awaits what it hopes will be voter approval in November 2020 of re-funding the agency. 

What does the ISSCR have to say about the state of the California stem cell agency?

Anne Nicholas, director of communications for ISSCR, was asked about the situation last week by the California Stem Cell Report. She replied,

 "We don’t have anything to add to your story at this point."

Turning Off the California Stem Cell Spigot: Will Private Donors Step Up?

Benchmarks are important to the $3 billion California stem cell research program. When scientists fail to achieve them, the flow of cash from the agency disappears. 

Last week, the stem cell agency quietly announced something of a funding benchmark for its own, 14-year-old efforts.

The bad news? In just six days, the agency will shut off  applications from California stem cell scientists and companies for multimillion dollar awards.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), last Thursday said that its dwindling finances forced the closure. 

CIRM has only $33 million left for new awards and already has requests in the pipeline for $88 million. Private funding is a possibility, but major donors have not yet surfaced publicly.

Immediate reaction to the announcement was muted but ranged from dismay to tributes to the agency and the work it has financed. 

One scientist, Jeanne Loring, chief scientific officer at Aspen Neuroscience in San Diego, said the action was like "a rug being pulled out from under you." Loring also said in an email that the agency has built an "enormous resource in stem cell expertise in California" and played an important role in her own work at Scripps Research. 

Loring's work has received $17.4 million from the agency since 2005. (See the full text of her remarks here.)

Steve Peckman, deputy director of the Broad Stem Cell Research Center at UCLA, said the agency has chalked up "impressive success" and made California an international leader in the field. (See the full text of his remarks here.)

UCLA has received $289 million from the agency. 

Robert Klein, who led the ballot campaign in 2004 that created the agency, said the application shutdown will create a gap that will hold back development of critical therapies. Klein is expected to lead another ballot initiative in November 2020 to provide $5.5 billion for CIRM. (See the full text of his remarks here.)

Klein is chairman and founder of Americans for Cures of Palo Alto, Ca. He was also the first chairman of CIRM. 

CIRM has provided funds to about 600 researchers and 128 institutions and companies. The researchers run labs that vary in the number of employees, but the total would include  hundreds more stem cell workers. 

The agency is currently trying to raise $200 million privately to continue its awards programs between now and the fall of 2020. No philanthropic gifts have been announced. Queried last week by the California Stem Cell Report, the agency said that it had nothing new to report in the fundraising effort that began last year.  

It is unclear how the application shutdown will affect the fundraising effort. It may serve as a prod, however, for some potential donors and help to crystalize decision-making as CIRM executives stress the importance of the agency.

CIRM's announcement left open the possibility of re-opening applications come September. The agency expects to have a better handle then on how much cash might be returning to CIRM from awards that have missed benchmarks. The amount is not expected to be huge. 

The agency has reported that it has enough money to sustain a wind-down of the agency and to administer remaining multi-year grants, should the yet-to-written ballot measure fail. 

Klein is optimistic, however, regarding the prospects for a bond measure 16 months from now.  He told the California Stem Cell Report that unspecified polls show that 70 percent of voters support re-funding the agency when they learn of the "remarkable progress" that has been achieved as a result of CIRM-backed research. 

CIRM Application Shutdown: Text of Robert Klein's Comments

Here is the text of comments by Robert Klein, chairman of Americans for Cures, on the shutdown of applications at the California stem cell agency. Klein was responding to questions from the California Stem Cell Report.

"When Californians learn of the remarkable progress from the California funded stem cell and genetic therapies FDA approved human trials in restoring major function to paralyzed patients, to save the lives of children with severe immune diseases, to restoring sight to patients that are blind, the support for stem cell research and therapy development exceeds 70% in recent polls.

"California’s leadership in cellular and genetic therapies, through its state funded research and human trials, currently supports 51 human trials for a range of chronic disease and injuries, and 24 more human trials are in progress by biotech companies based upon California’s funded research.

"The life-changing and life-restoring work of California’s scientists and physicians will predictably earn the chance for renewed funding. Polls indicate that California voters want the opportunity to vote in 2020 on continuing this visionary California initiative, originally funded in 2004 through Proposition 71, the California Stem Cell Research and Cures Act. The gap in available state funding from the fall of 2019 through November 2020 will hold back additional critical human trials for new life saving and/or disease mitigating therapies; but, I have faith that Patient Advocates and California voters will back new funding at the polls in 2020 and continue California’s remarkable contribution to this medical revolution that effects every one of our lives.

"In advancing the research and therapies, the California research funding agency has also gained broad financial support for its portfolio of research and human trials based on the strength of more than 2850 peer reviewed published medical discoveries and the 75 human trials directly funded or separately funded supporting the California funded discoveries. The matching funds from donors, institutions, private companies and non-profit organizations have already exceeded $3,250,000,000, more than a 100% match on the $3,000,000,000 originally approved in state funding.

"This medical revolution holds the promise of restoring health and quality of life for many of California’s individuals and families suffering from chronic disease and injury. However, the last tactical mile to bring this broad spectrum of therapies to patients will require more funding and the thoughtful support of California’s public as the human trials and discoveries are refined and tested, overcome numerous obstacles or complications, and ultimately serve to improve the life and reduce the suffering of every one of us."

California Stem Cell Agency Pitches for Support; Los Angeles Event Scheduled Tuesday

Just one day before the California stem cell agency announced it was cutting off applications for more research funding, it made an ardent pitch to the public for increased support. 

The appeal concerned a public event that the agency, formally known as the California Institute for Regenerative Medicine (CIRM), is staging on Tuesday in Los Angeles. 

In an item on the agency's blog, The Stem Cellar, Kevin McCormack, senior director of communications, briefly recounted the $3 billion agency's work. And then he wrote,

 "We still have a lot to do. The problem is we are quickly running out of money. We expect to have enough money to fund new projects up to the end of this year....Some may get funding from other sources, but many won’t. We don’t want to let that happen."

Stem researchers, CIRM leaders and others will be there. McCormack said,

"It’s going to be an opportunity to learn about the real progress being made in stem cell research, thanks in no small part to CIRM’s funding."

On the web page for the event, there was also this:

"You'll hear from a woman whose infant daughter was cured of a fatal immune-system disorder, from the doctor who developed that treatment and from stem cell champions who are worried what will happen to the most promising research if CIRM no longer exists."

 The free, public event will be in the Los Angeles Convention Center, 1201 South Figueroa St., Petree Hall C. The event begins at 6 p.m. but McCormack said CIRM staffers will be there at 5 p.m. to answer questions.

Interested persons can RSVP here. But it is unlikely that anyone will be turned away.

Money Running Out: California's Stem Cell Agency Shutting Down Applications for Research Awards

The $3 billion California stem cell agency, which is running out of cash, today served notice that it would stop accepting applications for more research awards beginning next month.

The low key announcement is another step towards the looming demise of the 14-year-old agency, created by voters in November 2004. Its hopes for continued life are pinned on a proposed bond measure for the November 2020 ballot.

A memo to the governing board from leadership of the agency, formally known as the California Institute for Regenerative Medicine (CIRM), said that it currently has $88 million worth of requests for cash but only $33 million available for awards.  

The memo said, 

"Given the status of applications in the system and expected applications this month, we intend to close application submissions after this month. In the unlikely event that funds continue to be available after processing of all applications in the queue, we may temporarily re-open the submission window as needed."

The matter was taken up this morning at a meeting of the board that approved an $11 million clinical award for a rare disease. 

During a brief discussion, board member Os Steward of UC Irvine said that "shutting off the spigot could be highly disruptive." 

He indicated that he would prefer to call it a suspension of applications. 

Board member Jeff Sheehy said the move is "abrupt but that is kind of where we are."

Under the ballot initiative that created the agency, the only significant source of funding is bonds issued by the state of California. The authority for issuing the bonds is expiring. 

The agency has been trying for months to raise $200 million in private funding. No announcement of any progress, however, was made at today's session. 

The agency has on hand enough funds to handle administration of existing awards for a couple of years. 

Here is the full text of today's memo. 

"For 2019, the ICOC allocated $93 million for clinical program applications (CLIN1, CLIN2, CLIN3) and $30 million for the CIRM/NHLBI collaboration on sickle cell disease. As of June 2019, the available budget for non-sickle cell disease applications is $45 million.  

"Currently, we have one application recommended for funding by the GWG and pending approval this month for $12 million. There are two applications slated for GWG review later this month and three applications that have received a score of “2” by the GWG that are pending reassessment by the GWG next month. We have also received five applications that are undergoing eligibility review. The total request for all clinical applications currently in our system is about $88 million. We are expecting two to three additional application submissions for this month’s deadline, which falls on June 28, 2019. The budget request for these is still unknown. We expect that not all applications will achieve a funding recommendation, but there may be enough to deplete the annual allocation.

"Given the status of applications in the system and expected applications this month, we intend to close application submissions after this month. In the unlikely event that funds continue to be available after processing of all applications in the queue, we may temporarily re-open the submission window as needed. 

"We intend to inform all prospective applicants submitting an application this month that there is no guarantee of an availability of funds and that review of their application may be halted if funds are depleted prior to the completion of their review. We also intend to post notice that application submissions will close after the June deadline until further notice.Ap 

"This action does not affect application submissions for sickle cell disease that are accepted under the CIRM/NHLBI collaborative program."

Stem Cell Researchers Backing Away from Documentary Touting 'Greatest Paradigm Shift in Medical History'

What do Maria Millan, CEO of the $3 billion California stem cell agency, Larry Goldstein of UC San Diego, Thomas Murphy of Harvard/MIT  and Mark Berman of Cell Surgical Network have in common this morning?

They are all loosely listed as "trusted sources" on a website ballyhooing a 10-part documentary involving stem cells. All are identified as taking part in the documentary. Berman's firm, however, is a target of the Food and Drug Administration, which is seeking to halt the enterprise's "unproven and potentially dangerous treatments." 

Erin Allday of the San Francisco Chronicle reported on the connections in a story this weekend that said some of the scientists involved in the documentary, scheduled to be released tomorrow, want out of the online video because it is partly funded by Berman's firm. 

The documentary is titled "The Healthcare Revolution." The Internet address of Berman's firm is stemcellrevolution.com."  It also offers a book called "The Stem Cell Revolution." The business has offices in Beverly Hills and Rancho Mirage, Ca., and is linked to reportedly dozens of similar enterprises.

Allday quoted Berman as saying in an email to patients last week,

“We have a wonderful docuseries coming out. It’s a whole production, not just about us. It’s going to enlighten people."

The website of the documentary features ebullient language about stem cells. "Stem cell technology that is said to be 10, 20 years down the road is actually here --  NOW!" reads one sentence on the video site.  The largest headline on the site said in bold red type, 

"This Must See Documentary Series Unveils the Greatest 
Paradigm Shift in Medical History"

Allday's story said some of the scientists involved in the documentary now say "they weren’t aware of who was backing the project when they agreed to participate...Some scientists said they fear the documentary may promote what they consider junk science."

Allday continued, 

"In fact, many of the scientists listed on the website have said repeatedly that stem cell therapies are still years away from being ready for patients. They have said that the hundreds of providers treating patients at for-profit clinics are selling 'snake oil' products that are worthless and potentially unsafe. No stem cell therapy currently sold at for-profit clinics has been approved by the FDA."

The Chronicle article said the California stem cell agency did not know who was financing the documentary when it agreed to participate.  A spokesman for the agency, known formally as the California Institute for Regenerative Medicine, told Allday that the agency agreed to participate because it would provide an opportunity to promote its work and warn against dubious clinics. 

One scientist who was scheduled to appear in the documentary is Jeanne Loring, professor emeritus at Scripps Research in La Jolla. She told Allday, “I am a stalwart and outspoken critic of unapproved stem cell therapies. I don’t belong in their company."

She said the producers of the video agreed to remove her from the documentary. 

More than 80 physicians and scientists are listed as "trusted sources" or experts on the video website along with a number of major academic institutions, including Stanford, Harvard, John Hopkins, Yale and Oxford.  Also listed is Kristin Comella, chief scientific officer of U.S. Stem Cells, Inc., which earlier this month lost a key ruling in lawsuit filed by the FDA against it. 

Trump Research Restrictions, the California Stem Cell Agency and Moral Obligations

The man expected to lead the drive for $5.5 billion more for California's stem cell agency today said the Trump restrictions on fetal tissue research represent a dangerous precedent that threatens the health of all Americans. 

Robert Klein, who was the first chairman of the state stem cell agency, said that "California has unique opportunity and obligation to maintain the scientific and medical options" that have led to development of the polio vaccine along with many others.

During an interview with the California Stem Cell Report, Klein said the people of California have a "moral" obligation to add more billions to the work of the 14-year-old, $3 billion stem cell agency.

Klein led the 2004 ballot initiative campaign that created the agency, formally known as the California Institute for Regenerative Medicine (CIRM).  The agency expects to run out of cash for new awards by the end of this year. It is staking its existence on a proposed ballot initiative that Klein would carry forward.

Klein's comments came as more reaction surfaced to the Trump action. San Francisco HIV advocate Jeff Sheehy, responding to a question, said in an email,

"Fetal tissue is used to make mice with human immune systems.  Testing new drugs for HIV is just one use--this animal model is used in research across a wide range of diseases to develop and test therapies, including vaccines for infectious diseases.  Stopping this research--which has been taking place for decades--is foolish, anti-science, and a threat to the health and safety of all Americans."

Kaiser Health News reported,

"The Trump administration’s announcement Wednesday about federal cutbacks in fetal tissue research is short of a total ban, but scientists in the field say it is concerning because it could affect work on treatments or preventions for key diseases, such as HIV and Parkinson’s."

Sara Reardon, reporting online for Nature, wrote,

"'It’s a decision that’s going to set back research,' says Andrew McMahon, a stem cell biologist at the University of Southern California in Los Angeles. 

"McMahon is studying ways to grow kidneys from human stem cells. He says that the only way to determine whether he and his colleagues have successfully mimicked natural development is to compare their proto-organs to kidneys in fetal tissue. Although biomedical research is often done using mice as proxies for people, mouse kidneys are too different from human kidneys to use in McMahon’s work."

McMahon was the recipient of a $5.7 million CIRM award dealing with kidney problems. A CIRM document filed in connection with his now concluded research said,

"Our analysis of the developing human kidney has provided the first comprehensive insight into developmental processes highlighting molecular and cellular events shared with the well-studied mouse model, but unique human features."

McMahon was recruited from Harvard to USC with the help of the CIRM grant. In response to an email query, he said that it was unclear whether his CIRM research would have become ineligible for federal support, given new Trump review processes. 

Bradley Fikes and Gary Robbins of the San Diego Union-Tribune wrote, 

"The sensitivity of the (fetal tissue research) matter surfaced recently when UCSD drew unwanted attention after one of its employees mistakenly solicited fetal pancreas samples from the Center for Medical Progress (CMP), an anti-abortion group whose surreptitious videos in 2015 galvanized efforts to end federal funding of Planned Parenthood."

Proven Value -- California's Stem Cell Agency Garners Praise in State's Largest Newspaper

California's $3 billion stem cell agency scored a significant media win this morning with an endorsement from one of its harshest critics, a Pulitzer Prize-winning columnist in the state's largest circulation newspaper, the Los Angeles Times.

Michael Hiltzik, who has written two books on massive public projects, wrote in the Times,

"California’s stem cell program has proved its value to the state and to science. It has earned the opportunity to show what it can achieve with an additional 10 years of life and billions more."

Hiltzik's piece appeared this morning in the print version of the newspaper, which says it has a readership of 2.1 million in the print version alone on Sundays. Hundreds of thousands more would be added online, where the column surfaced on Friday. 

Hiltzik began his column with some very bright news for the agency, known formally as the California Institute for Regenerative Medicine(CIRM). He said that Jonathan Thomas, chairman of the agency, told him that a CIRM-funded cure is headed for the marketplace. Hiltzik described it as "a major achievement."

He wrote,

"A stem cell-based cure developed by a team at UCLA for a rare disease — a 'bubble baby' disease that deprives children of a functioning immune system — is on a path toward expected approval by the Food and Drug Administration as early as next year. It would be the first treatment funded by the program to reach the market."

The achievement would come as the agency faces what Hiltzik called an "existential inflection point." CIRM expects to run out of cash for new awards later this year. The agency was created by voters in 2004 with $3 billion that the state borrowed.

The agency's existence depends on whether voters approve an additional $5 billion in additional bonds that would come from a yet-to-be-written ballot measure on the November 2020 ballot. Voters will need to be convinced that the agency has not frittered away its original $3 billion.

Hiltzik noted the agency's "ups and downs," including hype, conflicts of interest concerns and the affordability of stem cell therapies. He said a new ballot initiative offers an opportunity to make improvements in CIRM's operations.

Hiltzik wrote, 

"Klein told David Jensen of the California Stem Cell Report that he is contemplating a provision in the next initiative for “funding the infrastructure to work on access, to work with insurance companies, to work with Covered California, to work with Medicare, to make sure that there’s access, to make certain that public hospitals and public clinics in California get discounted prices.” That would be worthwhile, if it can be done."

Like most state agencies, CIRM labors invisibly. Its story and value proposition receive little public attention despite its attempts at ground-breaking science. Reporters are rarely seen at its meetings. Few articles are written about its affairs. 

That will change somewhat come the 2020 election season. However, media coverage will be dominated by national politics since it will be a presidential election year.  Given the scarce resources of the media nowadays -- along with other, expected, pressing California matters in the 2020 election -- stem cell research is unlikely to be on any front page (print or electronic) except infrequently, at best. 

Nonetheless, Hiltzik's column will be one of the key pieces that other journalists will dredge up electronically next year as they research ballot initiatives. And how the Times and Hiltzik have framed CIRM's work will be an important element in how other news stories are shaped.