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.