Yes or No on More Money for California's Stem Cell Program? Your Chance to Vote Today

It's time to vote -- sort of -- on whether to kick in $5 billion more for California's $3 billion stem cell research program.

Recent results from poll on CIRM

I say "sort of" because the voting consists of a poll being conducted by UC Davis researcher Paul
Knoepfler on his blog, The Niche. Initial results are already in. Sixty-three percent of those responding say they would definitely vote for a ballot measure to "refund" the agency.  (That figure jumped to more than 67 percent by Sept. 1,  three days after this item appeared. You can vote by clicking on this link.)

The poll, of course, is non-scientific, and the readers of Knoepfler's blog are hardly representative of the state's voter profile. Presumably those who seek out his blog are more informed about and more interested in the California stem cell agency than the average California voter . Nonetheless, it is an interesting exercise.

The question posed by Knoepfler's poll is not trivial. The agency, formally known as the California Institute for Regenerative Medicine(CIRM), expects to run out of cash for new awards at the end of next year. It hopes voters will approve a proposed ballot measure in November 2020, giving it another $5 billion.

If you would like to know more about the case to be made for the agency, search this blog (the California Stem Cell Report) using the term "assembly biotech hearing."  That will take you to seven brief brief articles related to a legislative informational hearing on the work of the agency over the last 13 years.

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

Pawash Kashyap and son, Ronnie

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

"It was a blessing day for us," said Pawash Kashyap, who appeared before the Assembly Select Committee on Biotechnology,  which held a hearing Aug. 15 dealing with the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. Kashyab referred to the occasion when he and his wife, Upasana, received a call from UC San Francisco warning him of the immune deficiency that afflicted their baby, Ronnie.

Researchers told them that the child could well die if he suffered from even a slight infection. The immune deficiency is sometimes known as the "bubble baby" disease, which has sometimes meant children were encased in plastic bubbles to stave off infections.

The problem was detected by routine newborn screening. Kashyap told legislators of their concern after learning the bad news.

"We Googled it, and nothing good was coming out of it."

Ultimately, the Ronnie was treated successfully. His father said that Ronnie has experienced potentially infectious crowds in malls and elsewhere and that he is doing "fantastically well."

Ronnie is now the "cover baby" on CIRM's annual report, which was praised by one lawmaker, Assemblyman Todd Gloria, D-San Diego. He said the report told the nearly 14-year, CIRM story in understandable and compelling language.

The full hearing can be seen here and downloaded. Kashyap's brief remarks begin at 55:36 into the video (he spoke without a prepared text). An audio file is also available at the same URL.

Here are links to the remarks at the hearing by CIRM CEO Maria Millan, CIRM Vice Chair Art Torres, patient advocate Don Reed, Jan Nolta, head of the UC Davis stem cell program, and David Jensen, publisher of this web site.

Article Postponed on Folsom baby and the Stem Cell Agency

Due to circumstances beyond the control of the California Stem Cell Report, the promised article on a legislative appearance by a Folsom father of an infant involved in a stem cell agency clinical trial has been delayed until Monday.

The father appeared at a legislative hearing earlier this month, along with his wife and child. The baby was afflicted with an immune deficiency disorder that would have been fatal. But he was treated in a clinical trial backed by the stem cell agency and is now healthy.

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

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

That came from the Biopolitical Times, a blog operated by the Center for Genetics and Society of Berkeley, a longtime foe of the agency. 

In a piece written by Pete Shanks, the agency was taken to task for a number of reasons. And he argued that it should not receive additional funds as it is presently constituted.

Shanks wrote, 

"At one time, CIRM had a deserved reputation for funding buildings , some of them at private universities, and was heavily criticized for that, but the $270 million “major facilities” budget approved in 2008 has all been spent. Some of the conflict of interest scandals are largely in the past, though ripples persist , and some of the institutional ones remain; several universities that receive large grants are still represented on the board . But there has been a new regime in place (“CIRM 2.0”) for several years.

"Things have improved, though not enough."

Shanks noted that the agency has failed to finance any therapies that are available for widespread use. He noted that the interest expense on state bonds that support the agency boost the cost to taxpayers to $6 billion from the $3 billion in awards.

He said the hearing last week was largely "a promotional vehicle." (Shanks' piece was published on Aug. 14, the day before the hearing by the Assembly Select Committee on Biotechnology.)

Shanks concluded:

"Going forward, there are two separate questions to consider: Is continued state funding of stem cell research at a rate of roughly half a billion dollars a year the best use of state funds?

"If it is, should those funds be spent through CIRM as it is presently constituted?
Is continued state funding of stem cell research at a rate of roughly half a billion dollars a year the best use of state funds? If it is, should those funds be spent through CIRM as it is presently constituted? 

"The first question is debatable; the second deserves a flat “No.” There is something obviously wrong when an agency is funded by public money but never has to submit a budget to the legislature, and can even go 13 years without appearing before an oversight committee. Two major reports, in 2009 by the Little Hoover Commission and in 2012 by the then Institute of Medicine (now part of the National Academies of Sciences, Engineering, and Medicine), both concluded that the governance structure of CIRM is seriously flawed."

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

UC Davis graphics and captions

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

Jan Nolta, the stem cell chief at Davis,  told the Assembly Select Committee on Biotechnology last week that the demise of the agency would lead to what some in her program are calling "cirm-amageddon," a play on the word armageddon and the initials of the stem cell agency's formal name..

The nearly 14-year-old agency is officially known as the California Institute for Regenerative Medicine (CIRM). It expects to run out of cash for new awards at the end of next year. CIRM is pinning its hopes for survival on yet-to-be-written ballot measure that would give it another $5 billion in state bonds if voters approve it in November 2020.

Nolta told lawmakers about the specialized facilities that are needed to deliver "living medicines" and highlighted the growth of the UC Davis stem cell program, which was virtually non-existent in 2004 when the agency was created.

Since then Davis has received $138 million from CIRM and has 24 stem cell trials underway "thanks to CIRM."

Nolta was lured to California after the passage of the ballot measure creating the agency. She said that the agency's programs have been a big draw for scientists from throughout the nation.

"If you want to do stem cells, this is where you come."

The full hearing can be seen here and downloaded. Nolta's remarks begin at 1:26:19 into the video (she spoke without a prepared text). An audio file is also available at the same URL.

Here are links to the remarks at the hearing by CIRM CEO Maria Millan, CIRM Vice Chair Art Torres, patient advocate Don Reed and David Jensen, publisher of this web site. Tomorrow the California Stem Cell Report will carry an item on the remarks of the father of an infant whose life was saved as the result of a clinical trial supported by CIRM. (This story has been postponed until Aug. 27.)

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

Don Reed 

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

He said,

"Today, thanks to the 7.2 million voters who authorized the California Institute for Regenerative Medicine, or CIRM, we have something better than hope; we have results, accomplishments, people made well—and a systematic way to fight chronic disease."

Reed was among the persons testifying last week before the state Assembly Select Committee on Biotechnology.

He  is vice president of Americans for Cures, a nonprofit group founded by Robert Klein, a real estate investment banker and who ran the ballot initiative campaign that created the $3 billion California stem cell agency. Klein was also the agency's first chairman and may lead another ballot initiative in November 2020 to renew the agency with an additional $5 billion.

Here are links to the remarks at the hearing by CIRM CEO Maria Millan, Art Torres, vice chairman of the CIRM board, and David Jensen, publisher of this web site.  Tomorrow the California Stem Cell Report will carry a look at the remarks by Jan Nolta, head of the stem cell program at UC Davis.  

The full hearing can be seen here and downloaded with closed captioning. An audio file is also available at the same URL. Here is the text of Reed's remarks.
(Some readers have reported that they cannot download Reed's comments shown below. However, I cannot detect any problem with that process. If you would like a PDF of Reed's remarks, please email me at djensen@Californiastemcellreport.com.)

   2018-08-15 Reed Assembly Biotech Hearing by DavidJensen on Scribd

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

Art Torres at Assembly hearing last week

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

Art Torres, a former state legislator and now vice chairman of the agency's board, kicked off the proceedings Aug. 15 with a brief overview. He also said that agency-backed projects have "brought in over $2.7 billion in investments to those projects from other sources (industry partners, co-funding and additional follow-on funding such as non-CIRM grants or philanthropy."

Torres said the add-on funding was the product of the $2.6 billion that has been actually awarded during the nearly 14-year lifespan of the agency, known formally as the California Institute for Regenerative Medicine (CIRM). The agency was provided with $3 billion in state bond funding in 2004. Cash for new awards is expected to run out by the end of next year.

Here are links to the remarks at the hearing by CIRM CEO Maria Millan and David Jensen, publisher of this web site. Tomorrow the California Stem Cell Report will carry the text of remarks by Don Reed, a longstanding patient advocate for stem cell therapies. 

(Toda,y, Aug. 21, the stem cell agency reprinted Jensen's remarks on its blog, noting that "at CIRM we know that not everyone agrees with us all the time, or supports all the decisions" of the agency.) 

The full hearing can be seen here and downloaded with closed captioning. An audio file is also available at the same URL.

Here is the text of Torres' remarks.

   California stem cell agency: Testimony by Vice Chair Art Torres to state Assembly Biotech Committee 2018-08... by DavidJensen on Scribd

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

Maria Millan, CEO of CIRM, at state Assembly hearing last week

The top executive at California $3 billion stem cell agency, which expects to run out of cash for new awards next year, outlined for state lawmakers last week the benefits of the nearly 14-year-old program.

Maria Millan, president and chief executive officer of the California Institute for Regenerative Medicine (CIRM), as the agency is officially known, laid out how the agency funds "the most innovative, high risk but high reward" stem cell research in the state.

Millan highlighted some of the 49 clinical trials backed by CIRM. She said,

"Every single trial represents a tremendous breakthrough approach that was unimaginable and even the subject of science fiction just years ago."

She also discussed the agency's relationship with the stem cell industry in backing research during a risky financial stage called "the valley of death." She said,

"CIRM supports and de-risks through this stage, where industry and traditional investors are not yet ready to come in. By accelerating development and allowing these early stage trials to proceed with CIRM funding, we have enabled the projects to gain visibility and interest leading to an increase in industry investments."

Her presentation was generally well received by the five lawmakers who were in attendance at various times during the meeting of the Assembly Select Committee on Biotechnology, chaired by Kevin Mullin, D-San Mateo.

The full hearing can be seen here and downloaded with closed captioning. An audio file is also available at the same URL. Tomorrow the California Stem Cell Report will carry the text of remarks by Art Torres, vice chairman of the agency and a former state legislator.  This writer was also invited to appear at the session. My remarks can be found here.

Here is the full text of Millan's comments.
Remarks by Maria Millan, President of CIRM, To California Assembly's Select Committee on Biotechnology 2018... by DavidJensen on Scribd

A California Stem Cell Tale: The Search for a Treatment for Paralyzing Spinal Injuries

Ed Wirth of Asterias, photo by Gabrielle Luri, SF Chronicle 

The San Francisco Chronicle, in a lengthy and dramatic article this week, reported on the story of the first clinical trial in the United States for a human embryonic stem cell treatment, a tale that continues today and well into the years ahead. 

The story involves paralyzing spinal injuries, incurable afflictions, walking rats, Geron, Asterias Biotherapeutics and BioTime and the economics of development of a stem cell therapy. That is not to mention the California stem cell agency, which has pumped more than $20 million into the effort.

The story was written by Erin Allday and is the third installment in a series involving stem cell research and treatments, both legitimate and illegitimate.

Her story began like this:

"Amid the controlled chaos of the operating room, Edward Wirth stood to the side, watching the surgeon slice open the back of the young man on the table....

"The surgeon nudged aside skin and a thin layer of muscle and clamped them out of the way. He chipped away bony vertebrae, exposing the shiny, smooth rope of the spinal cord. His scalpel slid into the membrane surrounding it.

"Wirth stepped forward, just beyond the surgeon’s shoulder, close by for these last steps. The surgical team moved a robotic arm holding a syringe into place. It was loaded with millions of immature support cells that had been meticulously crafted from stem cells, the cells that are the foundation of the human body, able to transform and reproduce indefinitely."

Wirth is a scientist who has been involved with the research since its early days at Geron of Menlo Park, Ca.. He has put in more than 20 years on the effort. Asterias Biotherapeutics of Fremont, Ca., which is associated with BioTime of Alameda, is currently handling the effort to create a treatment for spinal cord injuries.

The $3 billion California Institute for Regenerative Medicine (CIRM), as the agency is formally known, has provided a total of $20.7 million for the effort, first with Geron and now with Asterias.

Allday detailed the ups and downs, financial and scientific, of the research. She peered into the future and wrote that "it will take years to produce a marketable, federally approved therapy -- assuming their research makes it that far."

Nonetheless, she reported,

"Clinical trials like the one run by Asterias are scientists’ most ambitious attempts to harness the potential of what remains an elusive medical marvel. And the work at Asterias exemplifies both the incredible progress that’s been made in stem cell research and the great distance the field has yet to go before life-changing therapies are widely available.

"Riding on Asterias’ success isn’t just the fate of a single company or the careers of scientists like Wirth. Potentially, tens of thousands of patients every year could benefit directly from its therapy — and millions more if its research leads to further advances."

The fourth and final article in the Chronicle series is now scheduled to appear on Sept. 6  and will take a look at the California stem cell agency.

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

A committee of the California Legislature today examined the state's $3 billion stem cell agency. Officials of the agency and others presented their perspective. Here is the text of prepared remarks by David Jensen, publisher of this blog, who appeared at the invitation of the Legislature.

(On Aug. 21, the stem cell agency reprinted the text below on its blog, noting that "at CIRM we know that not everyone agrees with us all the time, or supports all the decisions" of the agency.) 

   2018-08-15 Jensen Assembly Biotech Committee Testimony by DavidJensen on Scribd

ACT to Astellas: A 'Looping' Tale of the Stem Cell Industry and Research

Ever wonder what happened to Advanced Cell Technology, Inc. (ACT), the Massachusetts stem cell company that migrated -- with some considerable ballyhoo -- to California in the wake of the creation of the Golden State's $3 billion stem cell agency?

ACT landed in the San Francisco Bay area in 2006 with hopes of snagging millions from the agency, which had been created two years earlier. At one point in the company's life, Gov. Jerry Brown even approvingly toured the company's operation in Alameda. But little has been heard from the company in recent years.

Now comes UC Davis stem cell researcher and blogger Paul Knoepfler. Last week, he published a brief update on the doings of the company once known as ACT and its changing corporate identities.

Michael D. West, AgeX photo

But first a little more on ACT's history and the company's not-so-good times in California. It is a tale  that loops around Geron, Inc., which launched and then abandoned the first U.S. clinical trial involving human embryonic stem cells, and companies called
Asterias and BioTime. One thread that strings them all together is a stem cell pioneer named Michael D. West.

When ACT announced its move to the Bay Area, visions of millions of research dollars danced in the heads of its executives. The move was heralded by the state's governmental leaders, Then State Treasurer Phil Angelides said,

"California voters overwhelmingly approved Proposition 71 (which created the stem cell agency) to ensure that California remains the hub of groundbreaking scientific innovation that has the potential to cure and treat debilitating and life-threatening ailments. Advanced Cell Technology's move to California sends a powerful message that this promise can be realized - bringing high-skilled jobs and revenues to our state, and most importantly, offering hope to millions of patients and their families."

West, then president of ACT, said the company was committed to being a world leader in regenerative medicine.

That was in 2006.  Eight years later, ACT, once the only company in the United States with an ongoing clinical trial involving human embryonic stem cells, changed its name to Ocata Therapeutics.  In 2016 it was purchased by Astellas Pharma of Japan for $379 million. By then, ACT had abandoned its then nominal operations in California after its multiple applications for California state funding failed to win approval (see here and here).

West left ACT in 2007. He is now co-CEO of BioTime, Inc., of Alameda, and is a member of the board of directors of Asterias Therapeutics, Inc., of Fremont, Ca. Both companies are faring much better with the state stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM).

In 2013, Asterias picked up the human embryonic stem cell trial that Geron gave up on in 2011. West founded Geron in 1990 but left the company in 1998. Asterias has received $14.3 million to support the spinal injury trial that Geron abandoned.

BioTime also had better luck than ACT. It received a $4.7 million award from CIRM for work on human pluripotent stem cells. 

Last week's report from UC Davis scientist Knoepfler said that Astellas began a phase 1B trial in July to continue the macular degeneration work of ACT/Ocata. Knoepfler described the latest effort as a "small bit of encouraging news."

California Lawmakers Schedule Update Next Week on the State's $3 Billion Stem Cell Agency

The parents of this child will be testifying next week at a state legislative hearing

For the first time in many years, a California state legislative committee is going to take a look at the state's $3 billion, 13-year-old stem cell research program.

An informational hearing is scheduled for next week, Aug. 15, by the Assembly 
Select Committee on Biotechnology, chaired by Kevin Mullin, D-San Mateo. His district includes a number of biotech firms. No legislation is up for action, and the hearing is not intended to necessarily lead to legislation.

Scheduled to appear before committee are Jonathan Thomas, chairman of the governing board of the agency, and Maria Millan, president and CEO of the agency, formally known as the California Institute for Regenerative Medicine (CIRM).  

The last time a CIRM CEO appeared before a state legislative committee was in 2005 when Zach Hall, then interim CEO testified. 

Also on tap is Jan Nolta, head of the stem cell program at UC Davis, which now stands as the No. 5 recipient of CIRM funds with $138 million. From Folsom will come a couple, Pawash Priyank and Upsana Thakur, whose infant child is now alive as the result of experimental research backed by the agency. The publisher of the California Stem Cell Report, David Jensen, is also scheduled to appear.

The stem cell agency is unique in California history and is a global leader in funding of stem cell research. It operates outside of the normal state budgetary process with its state bond funding flowing directly to it without the usual state budgetary oversight exercised by the governor and the legislature. That is all due to the ballot initiative that created the agency in 2004 and altered the state constitution.

The agency is scheduled to run out of cash next year. It is hoping that voters will approve $5 billion more for it in November 2020.

Next week's hearing, which will be in room 444 in the Capitol, will be available live on the Internet on the day of the  hearing via this page.  It will be archived as well and will be found via this link.

(To see the text of testimony at the hearing, search this blog on the term "assembly biotech hearing.")

(Editor's Note: An earlier version of this item misspelled the last name of Kevin Mullin.)

The 'Renegade Outposts' of the Stem Cell Industry: A Sweeping Examination by the SF Chronicle

Chronicle graphic

"Merchants of hope" was the headline today on a lengthy look by the San Francisco Chronicle at what it called the flourishing but costly, unproven and largely unregulated stem cell clinics in the United States and California.

Written by Erin Allday, the piece said,

"They are renegade outposts operating with little legitimacy and oversight at the frontier of what is otherwise a highly promising field of medicine."

It was the second installment in the Chronicle's look at stem cell treatments, both dubious and not-so-dubious. The first was published last month.

Today's piece took a lengthier look at the dubious efforts than has been done in other recent,  mainstream media examinations of the field, which is drawing increasing scrutiny from federal regulators. Allday wrote,

"None of the treatments the clinics offer have been shown to be safe or effective. None have been approved by the FDA. They’re not backed by decades of laboratory and animal studies or by rigorous testing in humans.

"Yet for many patients desperate for care, these clinics fill the void between the long-anticipated potential and the real-world limitations of stem cells. They cater to people whose needs reach beyond the powers of current medicine — people who want to believe in the almost mythic powers of stem cells, who feel corporate health and science have forsaken them.

"People who turn instead to these merchants of hope."

The article also touched briefly on the $3 billion California stem cell agency. Allday said, 

"The California Institute for Regenerative Medicine (CIRM) — the state agency created by Proposition 71 in 2004 to fund stem cell research — has raised concerns about the thriving consumer therapy market. CIRM officials and many of the scientists they fund worry that the selling of unproven treatments could undermine their own work and cast doubt on the enormous potential of regenerative medicine."

Allday continued,

"'It really hurt our recruitment,' said Jane Lebkowski, president of research and development at Regenerative Patch Technologies in Portola Valley. 'We got a lot of questions: What happened in Florida? Am I going to go blind too?'

"Most patients, Lebkowski noted, don’t understand the difference between the fat stem cells that a for-profit clinic is using and the complex product her company is developing, which uses embryonic stem cells to develop a kind of retinal cell that’s damaged in macular degeneration.

"That’s part of the reason why scientists like Lebkowski and organizations like CIRM, which gave $17 million to the Regenerative Patch trial, have pressured the FDA to crack down on the consumer market."

The next installment in the series will deal with stem cell research and is scheduled for publication Aug. 16.

California Stem Cell Cybercast: Multiplying the CIRM Story

Here is today's CIRM Facebook event. The number in the lefthand corner is not static.
It changes as more people view the cybercast.

California's 13-year-old stem cell research agency staged another event in cyberspace today, chalking up more than 1,200 online views for its exploration of the search for cures and therapies for Lou Gehrig's disease.

It was the second go at Facebook Live for the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

The online event, which features disease experts and questions from viewers, bypasses traditional media and features a concrete way of measuring the event's reach both today and two weeks from today. It could be a significant communications tool for the $3 billion agency, which expects to run out of funds for new awards by the end of next year. The agency is hoping voters will approve another $5 billion in funding in November 2020.

Today's event garnered 819 viewers during its initial hour. By the time of this writing that figure had climbed to 1,262 as others found the event on Facebook.

The first Facebook event for CIRM, which discussed stroke therapy, has now recorded more than 7,500 views.

Live Online Event Tomorrow from California Stem Cell Agency: Ask the Experts about ALS

California's $3 billion stem cell agency tomorrow will offer up a live, online and interactive look at ALS and the research it is financing to help find a cure for the affliction.

The agency has pumped more than $79.4 million into efforts aimed at ALS, which is often referred to Lou Gehrig's disease. Three researchers funded by the agency will be online tomorrow and will address questions from viewers. Here are more details here about the agency's FaceBook Live event at noon PDT, including instructions on how to view it and take part. 

Solomon and Stem Cells: California's Stem Cell Agency Is Slicing and Dicing Cash and Research Decisions

California's stem cell agency gave away $14 million this month, which could be described as less than a drop in its $3 billion bucket.

But the talk at the agency's awards meeting July 19 was not about largess. Instead it was about the lack of cash, lack of time and the need to split "babies" and "buckets."

"We are going to have to make some hard choices today," said one member of the agency's governing  board, Jeff Sheehy, a patient advocate member of the board for HIV.

"We are coming to the end," said another, Oswald Steward, director of the Reeve, Irvine Research Center at UC Irvine.

Sheehy and Oswald and others referred to the shrinking finances of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known. It expects to run out of cash for new awards by the end of next year. Its leaders are trying to raise more than $200 million privately to tide it over until November 2020. That's when they hope another ballot initiative/bond measure will be approved by California voters and pump an additional $5 billion into the 13-year-old enterprise.

That was the backdrop at the session earlier this month. It was a meeting that illuminated the changing criteria that the agency is beginning to use  -- as the cash squeeze tightens -- to make decisions on who and what to finance. No longer will patient advocates or researchers be able to assume that an excellent scientific score assures that an application will be ultimately approved. Instead, "programmatic" considerations will play an important and perhaps definitive role.

Those considerations range from whether a researcher has received past funding from the agency, whether the application fits well within the agency's mission, whether an application fills a void in the agency's portfolio and just how innovative the research is, plus much more.

The discussion about diminishing resources arose when directors were given the usual list of applications that were approved for funding by its reviewers, who met earlier behind closed doors.  Normally, the reviewer decisions are rubber-stamped. This time, however, the 14 reviewer-approved applications totaled $19 million, but only $10 million was budgeted. That's when some directors brought up the need for Solomon-like decisions that would "split" the babies and the "buckets" of cash.

One upshot was that two applicants, James Hagood of UC San Diego and  Vittorio Sebastiano of Stanford, received only partial funding with the expectation that the board would approve a revision in the budget in late October. Nonetheless, those researchers were better off than six applicants who were also approved for funding by reviewers, but not by the CIRM directors. Final action on their applications was put off, probably also until late October.

The $10 million round involved basic types of research, but with a two-year time table. Directors also took action in another, separate round involving preclinical work. It dealt with only one application for $3.99 million, which was approved with no discussion. It went to Poseida Therapeutics, Inc., of San Diego for research aimed at supercharging  "a patient’s own immune system cells to attack and kill a treatment-resistant form of prostate cancer," according to the CIRM news release on all the awards. Poseida matched the award with $998,023.

It was the second CIRM award for Poseida, bringing its total from the agency to $23.8 million.  Here is a link to the summary of comments from reviewers on the Poseida application.

Below is a list of the other approved awards, all of which went to institutions that have links to a CIRM governing board member. Those members were not allowed to vote on those applications. Here is a link to a document which contains summaries of the reviewer comments on each application as well as ones that were not funded on July 

Application number	Title	Institution	Amount	Principal Investigator
DISC2-11131	Genetically Modified Hematopoietic Stem Cells for the Treatment of Danon Disease	UC San Diego	$1,393,200	Eric Adler
DISC2-11157	Preclinical Development of An HSC-Engineered Off- The-Shelf iNKT Cell Therapy for Cancer	UCLA	$1,404,000	Lili Yang
DISC2-11036	Non-viral reprogramming of the endogenous TCRα locus to direct stem memory T cells against shared neoantigens in malignant gliomas	UC San Francisco	$900,000	Hideho Okada
DISC2-11175	Therapeutic immune tolerant human islet-like organoids (HILOs) for Type 1 Diabetes	Salk Institute	$1,637,209	Ronald Evans
DISC2-11107	Chimeric Antigen Receptor-Engineered Stem/Memory T Cells for the Treatment of Recurrent Ovarian Cancer	City of Hope	$1,381,104	Saul Priceman
DISC2-11165	Develop iPSC-derived microglia to treat progranulin- deficient Frontotemporal Dementia	Gladstone Institutes	$1,553,923	Li Gan
DISC2-11192	Mesenchymal stem cell extracellular vesicles as therapy for pulmonary fibrosis	U.C. San Diego	$865,282	James Hagood
DISC2-11109	Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome	Stanford University	$865,282	Vittorio Sebastiano

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California Stem Cell Agency's Latest Online Event to Highlight ALS and Challenges to Research

California's $3 billion stem cell agency will mount its second Facebook Live event next Tuesday, featuring ALS researchers from Cedars-Sinai discussing their work and the challenges confronting the field.

The noon PDT cybercast will feature Clive Svendsen, director of the Regenerative Medicine Institute at Cedars, Robert Baloh, director of neuromuscular medicine at Cedars, and Ralph Kern, chief medical officer of Brainstorm Therapeutics, Inc., of New York City.

Viewers will be able to pose questions to the participants during the event, which has been dubbed "Ask the Experts.".

The stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), has awarded Brainstorm $15.9  million to support a phase three clinical trial work in California dealing with ALS. Svendsen has received $24.9 million from CIRM, including $6.2 million for a phase one/two trial also involving ALS.  Baloh has received a $3 million CIRM award for research involving peripheral nerve disease.

CIRM's first Facebook Live event has drawn nearly 7.500 viewers since it was staged two months ago, a performance that makes it a useful tool in reaching large numbers of people.

Media Series on California Stem Cell Agency Has New Publication Dates

The San Francisco Chronicle has changed the publication dates for its series on stem cells and the California stem cell agency.

The second installment, dubbed "clinics,"  was scheduled to appear today. However, the publication date is now set for Aug. 2, one week from tomorrow. The rest of the schedule calls for the "research" installment on Aug. 16 and "progress" on Aug. 30.

Here is a link to an item on the beginning of the series earlier this month.

California Parkinson's Group Strikes Out on Path to Stem Cell Therapy; Clinical Trial, For-profit Company Goals

A San Diego patient advocate group for Parkinson's Disease is making a major financial move as it heads toward creation of a for-profit company to develop a stem cell therapy for an affliction that affects about one million people in the United States.

The group is the Summit for Stem Cell Foundation, which has been deeply involved in seeking funds from the $3 billion California stem cell agency. The Summit organization supports research being conducted by Jeanne Loring, director of the Center for Regenerative Medicine at the Scripps Research Institute.

Summit says it has raised $5 million as of this spring from a variety of sources. It estimates it will need $8 to $10 million to reach the stage where the federal government approves the beginning of a clinical trial. It also expects to raise more cash by avoiding the high administrative charges involving many research organizations.

Reporter Bradley Fikes of the San Diego Union Tribune wrote yesterday about the latest developments. He said Summit is moving out of its space at Scripps as it prepares to apply for funds for clinical research. Fikes wrote,

"The group has leased about 5,000 square feet, doubling its space, and as a parting gift Scripps Research is donating equipment from its stem cell center, saving the foundation nearly $1 million in start-up costs. At the same time, its overhead is dramatically reduced, said Jeanne Loring, the group’s lead scientist and professor at Scripps Research. (Loring is serving as the unpaid director of research for Summit.)

"A $2.4 million grant the group received from California’s stem cell agency ran out earlier this year, Loring said. Now the group can step up fundraising and add staff. It’s also soliciting more donated equipment."

Fikes continued,

"The group also plans to establish a for-profit company to get funding from investors interested in generating returns, Loring said. By drawing investments as well as philanthropy, the project gains access to capital not otherwise available.

"The group was inspired by the example of the Cystic Fibrosis Foundation, Loring said. In 2000, the foundation gave San Diego’s Aurora Biosciences $40 million to develop a cystic fibrosis drug."

Fikes reported that separation from Scripps has financial advantages dealing with what called "indirect costs." 

"Raising (donations) has been difficult, in large part because of the overhead issue, Loring said.

"Scripps Research imposes overhead of up to 94 percent, meaning that every dollar donated to the group required an additional 94 cents for the institute, Loring said. At the new location close by on Torrey Pines Mesa, overhead is roughly 10 percent.

"Lower overhead means the group can now apply for philanthropic grants that previously weren’t practical, she said, because donors prefer as much as their money as possible go directly to the cause."

Here is a video of Loring describing her work. 

A Good Sign for California's Stem Cell Agency? Californians Like to Say Yes to More Bonds

An ambitious campaign to stave off the death of the California stem cell agency with a $5 billion jolt of state borrowing could have an easy ride in a couple of years. 

At least that would seem to be the case if one bets on the odds as perceived in a column carried today in the Los Angeles Times. 

The headline on the piece by John Myers, the Times' Capitol Bureau chief in Sacramento, said,

"California voters almost always say yes to bonds..."

Myers wrote, 

"It’s the closest thing to a sure bet that exists in statewide campaigns, with an approval rate hovering around 90%."

Myers, however, focused mainly on this November's ballot -- not 2020. And he did not comment directly on the plan to present a $5 billion bond measure to voters in November 2020 to keep the stem cell agency alive.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), expects to run out of cash for new awards by the end of next year. Its only significant source of funding is money that the state borrows (bonds), which roughly double the costs of its operations because of interest expense. The borrowing was approved by 59 percent of the voters in 2004.

Myers related the current condition of the Golden State's indebtedness:

"State general obligation bond measures approved since 1986 total more than $167.7 billion. Lenders must be repaid with interest, averaging about 5% a year, over a span of several decades. Most general obligation bond payments come from the same bank account that provides cash for services such as education, healthcare and prisons." 

He continued, 

"A recent report by the nonpartisan Legislative Analyst’s Office estimated that California’s general fund is currently paying off $83 billion in bond obligations. Annual debt payments total about $6 billion. That’s roughly equal to a year and a half in general fund spending on the University of California system, or about triple what the state spends on firefighting."

"'When we make these decisions, we have to look at the big-picture context,' state Sen. Bob Hertzberg (D-Van Nuys) said during a legislative hearing last month on November’s slate of bond measures."

State Sen. Bob Hertzberg, News Growl photo

Hertzberg zeroed in on backers of a clean water measure on the fall ballot. The Times piece reported,

"'You’ve got to tell the truth,' he said to the measure’s proponents. 'You back up an armored car to the treasury, and you can take $430 million out of the back door because you can poll [on] something, spend a few million dollars because it’s an issue that looks sexy to voters, and draw the dough.'"

Myers continued,

"Voters are rarely asked to think about which needs government should meet. In 2004, they agreed to borrow $3 billion for stem cell research. In 2008, voters said yes to $9.9 billion in seed money for high-speed rail. Even though the (rail) bonds have only recently begun being sold to Wall Street investors, it’s debatable whether voters would make the same choice if asked again.

"And campaigns rarely offer enough information to fully consider the pros and cons of a bond measure. Voters must do their own homework, beginning with the understanding that a bond is a mandatory expense, an investment decision that can have profound impacts for more than a generation."