Halloween Meeting Set for $3 Billion California Stem Cell Agency: Last Research Awards Expected to be Made

California's $3 billion stem cell agency next week is expected to give away the last of its funds for new research awards and hear a report on private donations that it may have received in the recent past or perhaps expects in the future. 

The donations report is something of a mystery, based on the agenda, but perhaps appropriate for the governing board's meeting, which comes on Halloween, Thursday Oct. 31. 

More details are likely to be posted online in the next week or so and reveal more clues about the direction -- over the next 12 months -- of the agency, formally known as the California Institute for Regenerative Medicine (CIRM).

The agency expects to run out of money for new awards at the end of this month unless a there is a surprise announcement that a private fund-raising effort has produced significant results. CIRM was created in 2004 by voters, who allotted it $3 billion in state bonds. No other source of cash was provided by the ballot initiative. 

In addition to consideration of applications for clinical and translation stage awards, also on next week's agenda are:

• "Use of public funds in connection with ballot measure presentation"
• Last week's report on the economic impact of the agency 
• Changes in the "administrative funds donor agreement and disclosure of funds received from donors." (Those changes were not available on the agenda but are likely to be available in the next few days.)

The ballot measure discussion involves the appropriate and legal role of the agency in connection with the $5.5 billion, proposed ballot initiative to provide additional support for CIRM. The measure is now before state officials for certification prior to being circulated for the 600,000-plus signatures needed to qualify for the November 2020 ballot. 

State law prohibits the use of state funds to support ballot campaigns. The agency has been careful to adhere to the law. But it is always a touchy area. A number of gray areas exist involving the agency's additional legal requirements to respond to questions and inform the public about the conduct of the taxpayer's business.

The public can comment and recommend changes in the proposed initiative between now and Nov. 12.  Suggestions for changes will be relayed to the backers of the measure.

Regarding the currently cryptic Halloween agenda, look for more details on the California Stem Cell Report between now and Oct. 31. We will be filing fresh items as the information emerges.  

Next week's meeting will be held at CIRM's Oakland headquarters. The public can attend and also participate via the Internet. Remote telephonic locations that are open to the public can be found in La Jolla, San Diego, Fresno and San Francisco. Directions and instructions can be found on the agenda.

California's 15-year Stem Cell Journey: Where to After 1,000 Research Awards, 56 Clinical Trials, $904 Million for Basic Research?

BURLINGAME, Ca. -- California's $3 billion stem cell research agency this year is nearing only its 15th birthday, but tomorrow it will wrestle with the question of whether there is life after 2020.

The agency, known formally as the California Institute for Regenerative Medicine (CIRM), is convening a passel of out-of-state scientists here to address the very real matter of what to do next year and thereafter. 

CIRM expects to run out of cash before the end of this year. It is hoping that California voters will approve a $5.5 billion bond measure in 2020 to continue its work. 

The scientists arriving for tomorrow's public meeting are non-California folks who have evaluated some of the more than 3,000 applications from California scientists for cash from CIRM.  The agency is barred by law from financing work outside the Golden State.

A presentation prepared by the agency for the session asks:

• "What should CIRM be thinking about now to prepare for a possible life beyond 2020?
• "How can CIRM deliver the greatest impact in the future?
• "What opportunities might CIRM seize to accelerate the field?• 
• "What challenges must be addressed?
• "What types of CIRM programs should be sustained or expanded?
• "What is missing, or needs more support?"

The day-long program also includes a 49-page, information package dealing with CIRM programs, ranging from basic research to clinical activities. It is the most comprehensive, recent, public look at CIRM's efforts that the agency has prepared.

Here are some of the highlights of the fresh data overview from CIRM: 

• 56 clinical trials
• More than 1,000 awards
• More than 1,200 patients in CIRM-initiated Alpha stem cell clinics
• $904 million for basic research
• Stimulation of $3.7 billion in outside investments
• 2,700 alumni from CIRM training programs 

The session begins at 8:30 a.m. PDT. It will be available also on online. Instructions for access can be found on the meeting agenda.

Meeting Delays and the California Stem Cell Agency: A Matter of Many Millions of Dollars

For the second time in two months, the governing board of the $3 billion California stem cell agency has postponed important meetings which had been expected to deal with its final research awards and its declining finances. 

The latest postponement involved a Sept. 25 meeting. On the table would have been redirection of tens of millions of dollars and the question of financing awards already approved by the agency's reviewers.

Last month, an Aug. 22 session involving major awards was  cancelled. As of today, the next meeting of the agency's board will not come until Oct. 31.

The governing board of the agency, known formally as the California Institute for Regenerative Medicine (CIRM), has before it far more reviewer-recommended awards than it can fund. Agency officials hoped that funds received from cancelled research projects would help fill the gap.

CIRM is financed with $3 billion in state bonds but has no source of significant cash beyond that. It expects to run out of money for new awards -- most likely by the end of next month -- depending on how it deals with the applications in its award pipeline. Those decisions are now likely to be made at the meeting currently scheduled for Oct. 31. 

Queried about the reason for delaying this month's meeting, Maria Bonneville, executive director of the board,  replied, 

"There were some scheduling issues and, as you know, getting the whole board together isn’t easy. (The) soonest I could get a quorum was Oct 31."

CIRM's future depends on a proposed, $5.5 billion ballot measure in November 2020 that would allow it to continue at the pace of the last 14 years. The agency also has been engaged for some time in an effort to raise privately more than $200 million to bridge the funding gap between now and the 2020 election. 

No specific progress has been publicly announced concerning that effort. However, the financial plight of the agency has received more public attention in recent months. That attention may have generated the loosening of the purse strings of some potential donors. 

The agency also may well have made a pitch to California Gov. Gavin Newsom for some sort of interim funding, possibly payable back with 2020 bond proceeds. Newsom was an important supporter of the agency in 2004-05 and engineered a $17 million package to lure its then headquarters to San Francisco when he was mayor of that city.

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.

California's $76 Million Bet: Something Called CAR-T, Cancer and Promises

CIRM-funded researcher Saul Priceman of the City of Hope discusses the CART-T frontier, April 2019. Video by American Association for Cancer Research

California's state stem cell agency has now invested $76 million in treatments sometimes described as "miraculous," whose costs can run upwards of $1.5 million. 

The push into the much-heralded CART-T therapy field is part of the agency's effort to fulfill its 15-year-old campaign promise to state voters to turn stem cells into cures. 

The California Stem Cell Report this week took a look at the  CAR-T slice of the agency's $3 billion research pie in the wake of news that Medicare would now cover major portions of the cost of the cancer-fighting technology.

The decision applied to only two specific therapies. But Seema Verma, the top Medicare executive, said CAR-T was
"an important scientific advancement" and would provide help for "some patients who had nowhere else to turn."

Maria Millan, president of the stem cell agency, called the announcement a big step forward. In an email to the California Stem Cell Report, she said,

“We are encouraged by the announcement that the Centers for Medicare and Medicaid will provide coverage for hospital costs related to CAR-T therapies. This is a major step to providing access to these life-saving treatments for patients in need.”

The CAR-T therapies approved by Medicare can run close to $500,000 plus hundreds of thousands more for related costs. One estimate is that total cost of a CAR-T treatment could hit $1.5 million. 

Their medical promise of CART-T is that it's a cure -- not an ongoing treatment, which also can raise total costs exponentially. The idea behind the therapy is that it powers up a patient's immune system to destroy cancer cells in a targeted fashion.  

"Drugmakers note CAR T-cell therapy is designed to be given just once and to be a potential cure for patients who have run out of other options. But not all patients benefit from it and because it is so new, it’s too soon to know whether it will deliver long-term cures," Laurie McGinley wrote in the Washington Post last week. 

California's stem cell agency, formally known as the California Institute for Regenerative Medicine(CIRM), has approved 11 awards involving CAR-T therapies ranging from basic research to clinical trials. The amounts of the awards run from less than $2 million to nearly $20 million, which went to Poseida Therapeutics, Inc., of San Diego, Ca.

The company has received a total of nearly $24 million from CIRM for its CAR-T work. In May, the Food and Drug Administration approved Poseida's CIRM-backed product as an orphan drug treatment for multiple myleoma, a disease that affects 300,000 Americans every year. The company reports that its Phase Two clinical trial for the treatment is now underway.

Poseida's work has attracted the interest of Big Pharma. In March, Novartis pumped $75 million into the company in a fund-raising round that totalled more than $142 million.  (See here for more information on Poseida.)

Last month, in another CART-T venture, the stem cell agency added more millions to an effort at the City of Hope by Saul Priceman, a breast cancer researcher. He told the CIRM board,

"For the past 20 years, City of Hope has focused intensely on developing CAR-T cell approaches for treating the most intractable solid tumors. In the early 2000s, we were the first to demonstrate solid tumor CART-T cell therapy treatment in patients. And recently we were the first to report our remarkable response for patients with glioblastoma."

CIRM directors added more than $9 million to the $1.4 million already provided to Priceman.

In announcing the award, CIRM CEO Millan said, 

“When a patient is told that their cancer has metastasized to other areas of the body, it can be devastating news. There are few options for patients with breast cancer brain metastases (the target of Priceman's research).

"Standard of care treatments, which include brain irradiation and chemotherapy, have associated neurotoxicity and do little to improve survival, which is typically no more than a few months.  CAR-T cell therapy is an exciting and promising approach that now offers us a more targeted approach to address this condition.”

CAR-T is a gene therapy, which has raised some questions about whether it fits within the scope of the stem cell agency, which was created by a ballot measure in 2004, Proposition 71.  

Kevin McCormack, senior director of CIRM communications, said in response to a query, 

"The programs we’ve funded fit into Proposition 71 because (1) HSC (hematopoietic stem cell) derived CAR-T, (2) CAR T’s enriched for TSCM (stem cell) or gene-engineered T cells qualify as a 'vital research opportunity' as outlined in the proposition." 

Here are links to the CAR-T awards by their CIRM application number, which is very useful in searching for additional information on each grantee's research.

Tran1-10258, $5.6 million, Ezra Cohen, UC San Diego; Clin2-10395, $19.8 million, Matthew Spear, Poseida; Clin1-10999, Devon Shedlock, Poseida; Clin2-10248, $12.8 million, Christine Brown, City of Hope; Clin2-10846, $11 million, Crystal Mackall, Stanford; Clin1-11223, $3.8 million, Xiuli Wang, City of Hope; Disc2-11107, $1.4 million, Saul Priceman, City of Hope; Clin2-11574, $9.3 million, Priceman, City of Hope; Disc2-10748, $1.7 million, Scott Kitchen, UCLA; Disc2-11157, $1.4 million, Lili Yang, UCLA, and Clin2-11380, $4.7 million, Theodore Nowicki, UCLA.

City of Hope video

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."

Unregulated Stem Cell Treatments Targeted by California Medical Board; Hearing Next Week

"Snake oil" stem cell clinics in California and their physician operators are on the agenda next week of state medical regulators as deep concerns are being raised in Congress about the slow pace of federal action against the enterprises. 

Last week, a bipartisan group of leaders of a U.S. House of Representatives committee told the Food and Drug Administration (FDA) in a letter, 

"We appreciate the actions taken by FDA, but this pattern of unsafe and unproven therapies remains deeply concerning. Such treatments not only pose public health risks for patients, but also harm the future promise held by the development of stem cell therapy as a field."

California also has been slow in dealing with the issue. State legislation to deal with the matter has been shunted aside.  More than a year ago, the state's Medical Board created a task force to deal with the issue. The panel did not hold its first meeting until June 27 of this year and then with no public notice. 

Experts who follow growth of the dubious clinics say more than 1,000 exist in the United States. The largest number are in California. The treatments are unproven and cost desperate patients thousands of dollars. The clinics have been linked with several cases of blindness and at least 12 serious infections. The FDA has filed lawsuits against two, including one in California, and sent 45 letters to clinics involving their practices. 

The agenda for next week's state medical board meeting says only that an "update" will be delivered by the task force which consists of two members of the board, Randy Hawkins and  and Howard Krauss. No action was specifically listed for the agenda item (No. 21), but the board couches its agenda in such fashion that it could legally act on any matter before it. 

The Congressional letter to the FDA not only expressed serious concern about the proliferation of unproven therapies,  but also said, 

"The advertising strategies some of these clinics employ to attract some of the most vulnerable patients are particularly alarming. Some have advertised stem cell treatments to desperate patients with the most serious untreated illnesses, such as spinal injuries, Parkinson's disease, and multiple sclerosis. Some clinics have misled patients into believing that the therapies they offer are FDA approved or that they are being offered as part of an FDA sanctioned clinical trial."

The chairman of the board of California's $3 billion stem cell agency, Jonathan Thomas, has called the treatments "snake oil." The agency, formally known as the California Institute for Regenerative Medicine (CIRM), is running out of cash. It is worried that a move to refinance the agency via a ballot measure next year could be harmed by voter confusion about the stem cell research.

The congressional letter said,

"When providers offer harmful, unproven stem cell therapies outside of the clinical setting, they create confusion among patients and undermine public confidence in treatments that have been proven to be safe and effective through well-designed clinical trials. If we are to realize the potential clinical benefits of stem cell therapies, it is crucial that developers focus their efforts on the use of traditional developmental pathways that yield definitive results rather than promoting products with 'dubious clinical efficacy and possible risks.'"

Next week's meeting is open to the public. Letters to the board can be emailed to executive director, Kimberly Kirchmeyer, at Kimberly.Kirchmeyer@mbc.ca.gov.

California Stem Cell Agency: 'Inarguable' Source of Hope, Says Article in Scientific American

The headline this morning on the Scientific American web site said:

"A Bulwark against Trump's Stem Cell Ban

"California's Institute for Regenerative Medicine, a kind of mini-NIH, does crucial basic research without federal funding"

Authored by Zachary Brown, a researcher at UC San Francisco, the article used as a peg the Trump administration restrictions on fetal research funding. Brown contrasted those restrictions to work being done here in California financed by the state stem cell agency. 

Brown recalled that a major impetus for voter creation of the agency in 2004 was presidential restrictions on federal backing for human embryonic stem cell research. Brown wrote,

"The future of embryonic stem cell research appears uncertain once again, as researchers are forced to scramble to adjust to arbitrarily changing norms uninformed by science."

Brown said,

"Almost 15 years have passed since Proposition 71 became law, and California voters made a three-billion-dollar bet on the promise of stem cell technology. At the time of its passage, the policy proposal was as groundbreaking as it was subversive."

Subversive because it challenged the "very relevance" of the predominant federal funding model. 

Brown continued,

"The measure was not perfect. Robert N. Klein II, one of the largest donors in support of Proposition 71, ended up as head of the governing body for seven years, and questions concerning bias in the disbursement of its ample endowment linger — curiously, more than 90 percent of awards" have gone to institutions with ties to governing board members.

"However, its role as a source of hope, both symbolic and realized, for the field of stem cell research is inarguable. The federal government weakens the image of the U.S. as a hub for discovery and medical ingenuity every time it prioritizes political gain over scientific progress."

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. 

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."

Text of Loring Letter Asking to be Removed from Stem Cell Video

Here is the text of email that stem cell scientist Jeanne Loring sent to Sara Sheehan in connection with the controversial "HealthRevolution" documentary. 

​"Dear Sara:
"When I was interviewed at the World Stem Cell Summit, I was misled about the context in which my interview would be placed. I was told that it would be an educational video about the progress of legitimate stem cell therapies.

"I am a staunch and outspoken opponent of clinics that flout the FDA regulations for stem cell treatments, and have met many people who have paid many thousands of dollars to obtain unregulated (and therefore unsafe) treatments that claim that stem cells will magically cure any disease.

"As I have explained many times, to print reporters, community groups, and in video interviews, as a scientist with a moral compass, I cannot condone exploitation of desperate people who are led to believe that they will be cured or even helped by a clinic or a pill or any purported therapy that is not based in science.

"I know you are young, but this is equivalent in history to the Laetrile scam of the 1970s. Thousands of desperate people emptied their wallets and obtained this alleged therapy before the FDA stepped in and banned it. Many were crushed and bankrupt; some died.

"'Stem cell' clinics are worse, because they can market their useless and dangerous products to a far larger audience.

"FDA leaders Scott Gottlieb and Peter Marks have denounced these unregulated clinics as “bad actors”, and I would go further - I think they are criminals.

"I use pluripotent stem cells to generate real cell types to replace cells that have died in disease. I have not seen any evidence that extracts from fat, or bone marrow, or products derived from placentas and umbilical cords have any medical value.

"You have placed my interview among those of people who are charlatans and thieves, and I request that you remove all reference to me in the video and on your website.

"I understand that you are good and honest film makers, so this should not be a difficult request. Please respond that you have removed me and I will not trouble you further."

"Sincerely,
"Jeanne Loring"

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. 

A Stem Cell Media Story: The Case of the Missing Mention

Storks and stem cell blessings

California's $3 billion stem cell agency, which is fighting to demonstrate its value proposition, this week received what could be considered some favorable attention in the prestigious journal Nature.

The catch is that the 14-year-old research program was not mentioned by name by Nature.

The question is: How does that work and why it is important?

First things first: It is important because the agency, known formally as the California Institute for Regenerative Medicine (CIRM), is hoping that the voters will bless it with another $5 billion come the November 2020 election.

The agency expects to run out of money for new awards this year and is also trying to raise privately more than $200 million to tide it over until the 2020 election. In both cases, it needs clear successes that demonstrate that it has earned its keep and will do even better in the future. 

Unproven Therapies in Japan

The not-so-odd case from Nature involves an editorial that cited a CIRM-funded clinical trial.  Nature praised it as something to be emulated in Japan as an example of good ethics and good science. 

Nature's editorial zeroed in on how Japan has embarked on "a worrisome approach." The headlines said,  "Japan should put the brakes on stem cell sales. Unproven therapies should not be marketed to patients."

Instead, Nature declared that Japan should focus on the approach taken by Asterias Biotherapeutics, Inc., of Menlo Park, Ca., which arguably owes its existence largely to CIRM. (See here, here and here.)  Nature wrote, 

"Last week, a Californian company called Asterias Biotherapeutics released promising results from a 12-month first phase clinical trial, in which embryonic stem cells are converted into oligodendrocytes — cells of the central nervous system that support neurons and can stimulate their growth — and then injected into the backs of people with a spinal-cord injury. The data show that injected cells do stick around at the injury site, and that most patients (21 out of 22) showed improved movement. 

"But these are still early-stage results. It is not clear yet whether the improvements are the result of the cells, or whether something else, such as the body’s own regenerative capacity, was at work. To find out, the company wants permission to move forward with a randomized, controlled phase II clinical trial. That’s the right way to do things: stepping carefully, slowly and rigorously forward."

It's Not the Stork

The problem for the stem cell agency, of course, is that Nature did not identify CIRM as a player in the Asterias effort, although the agency has pumped more than $20 million into the research. The case of the missing mention is not an unfamiliar situation for the agency, which is often not noticed in news and press releases about the scientific accomplishments that it has backed with tens of millions of dollars. 

Of course, CIRM cannot take full credit for the Asterias work. And federal regulators have rules for clinical trials. But CIRM put up the cash for the research after Asterias' predecessor bailed out.  The agency also partners closely with its grantees and has a clear set of pioneering research standards that it worked out more than a decade ago. 

Obviously, CIRM would have been only a brief note, perhaps only a phrase in the Nature editorial if it had decided to include a mention. But these things add up and are needed by CIRM as it tries to tell California voters that it has been worth $3 billion. Their absence can amount ultimately to a substantial negative. 

One does not have to support more cash for CIRM to recognize that California voters need the full array of information about the agency to make an informed decision in 2020. Currently, however, the traditions and practices of scientific journalism regularly omit significant financial information.

When one reads about scientific advances in the mainstream media as well as journals, it seems as if the research magically materializes without a critical insemination of cash -- much like a baby being brought by the stork.

Perhaps it is time for CIRM, other research funding agencies and patient advocates to have a facts-of-life "talk" with the scientific press about the reality of what it takes to give birth to prodigious medical advances.

(Editor's note: Here is a related item by UC Davis stem cell research Paul Knoepfler on the Japanese program.)