Stem Cell Researcher on $5 Billion Retooling of California's Stem Cell Agency: Learn from Experience, 'Do This Better'

California stem cell scientist Jeanne Loring has endorsed an insider's critique of a proposed refinancing plan for the state's stem cell agency, calling the analysis "logical" and coming "from the heart."

Loring's statement this week to the California Stem Cell Report addresses the 3,300-word evaluation by Jeff Sheehy, who has served on the agency board since its inception in 2004. 

In her comments, she endorses his analysis as logical and detailed. Among other things, she supports a reduction in the size of the agency's board from 29 to seven or 11 and stronger leadership of the panel. 

Loring is an award-winning scientist who has received more than $20 million in grants from the agency, officially known as the California Institute for Regenerative Medicine (CIRM). She is a co-founder and chief scientific officer of Aspen Neuroscience of San Diego, Ca., and professor emeritus at Scripps Research in La Jolla.

The CIRM board is known as the Independent Citizen's Oversight Committee and has called a Nov. 15 meeting to consider the ballot initiative. Its sponsor has until Nov. 18 to submit changes in the proposal. 

The California Stem Cell Report more than welcomes additional comment on this important subject, both pro and con. You may comment at the end of this item. Or send your commentary to djensen@californiastemcellreport.com. It will be carried verbatim.

Here is the full text of Loring's comments: 

"I’m a stem cell researcher who has benefited greatly from California’s investment in human pluripotent stem cells. I’ve been attending the Independent Citizen’s Oversight Committee (ICOC) meetings since 2005; I’ve been to more than 50, which is more than many of the current ICOC members have attended. I am writing because my own observations support Jeff Sheehy’s insightful critique of the 2020 ballot measure. I know that it is likely that he will be criticized for raising his concerns, but there is no stronger supporter of CIRM’s regenerative medicine mission than Jeff Sheehy. With all due respect to the other 28 members of the ICOC, I’ve seen that Jeff has always been the best prepared for their meetings. 

"I urge the promoters of the new initiative to not confuse criticism with anti-CIRMism. Jeff’s assessment is logical, detailed, and comes from the heart; his goal is to de-politicize the agency and make it more accountable to the people of California.

"There is tremendous concern that the decisions of the ICOC, both on policy and in deciding what grant applications to fund, are politically and selfishly motivated. This concern is warranted, as manifest in some of the wasteful decisions the ICOC has made. This can be fixed. I would like to see stronger leadership of the ICOC, in the form of a fully committed chairman, and that the committee take its responsibilities more seriously. I agree with Jeff that the ICOC should have fewer members, 7 or 11 instead of 29 or more, so that no member can escape his or her responsibility.

"The ICOC is supposed to make judgements that assure that the funding provided by CIRM is consistent with CIRM’s mission for the people of California. Unfortunately, I have seen too many situations in which it is clear that the ICOC members too often defer to the grant reviewers, who are all from outside California, and have no stake in the outcome of their decisions. The ICOC has too often approved grants of tens of millions of dollars without spending any time investigating the applications and deciding whether the money will be used in the best interest of Californians.

"I fervently hope that Jeff’s specific suggestions about the ballot measure be considered by ICOC members, politicians, and voters. There is a chance now to learn from experience and do this better, and all that will take is to engage the critics who know what they are talking about.

"Anyone who has an untreatable condition should be aware of the practical value of regenerative medicine. We in California have an opportunity that the rest of the world can only wish for - to make lives fundamentally better for those who suffer now and will suffer in the future from diseases and injuries that only regenerative medicine can help."

Skeptical Look at $5.5 Billion Plan to Refinance California's Stem Cell Research Program

A longtime critic of California's $3 billion stem cell agency this week chalked up pluses and minuses of the research effort, which it said is "essentially broke," and sounded a skeptical note about a plan to refinance it with $5.5 billion more.

The Center for Genetics and Society (CGS) "essentially" agreed with what it described as a "scorching" critique of the proposed ballot initiative to provide the agency with additional billions. In addition to the cash, the initiative would make major changes in the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

In a post on the CGS blog, Pete Shanks, a consultant with the Berkeley organization, briefly recounted the history of CIRM and some of the past criticism of it. But he focused primarily on the critique last week by longtime CIRM board member Jeff Sheehy. Shanks said,

"Sheehy’s range of concerns is wide. He convincingly criticizes proposed changes in the board structure, in handling conflicts of interest, and in the way CIRM addresses ethical concerns. He objects to the measure’s failure to maximize financial returns to the State from any successful therapies. He is also apparently baffled by the strange requirement that CIRM must expand its (apparently successful) 'Alpha Stem Cell Clinical Program' — seemingly a clear example of fixing what ain’t broke."

Shanks also wrote,

"The original governance scheme of CIRM has been widely criticized, first at the time of its formation by a number of public interest organizations (including the Center for Genetics and Society) and subsequently by the Little Hoover Commission in 2009 and by the Institute of Medicine in 2012.”

He continued,

"One of the main criticisms of CIRM involved widespread conflicts of interest on its governing board and the lack of effective oversight from the legislature, both of which were built into the structure quite deliberately. The proposition amended the California Constitution by specifically adding the establishment of CIRM and the right to conduct stem cell research as Article XXXV . It also required waiting at least three years, obtaining votes from a 70 percent supermajority of both houses of the Legislature, and the Governor’s signature before any amendment could be made. And it exempted the research it authorizes from “other current or future state laws or regulation.” Unfortunately, the new language not only avoids making many needed reforms, but it may also actually make the structural issues worse."

Shanks noted the hype surrounding the 2004 initiative that created CIRM. He said,

“Advocates for the new initiative have already demonstrated that they plan to pin their case for billions of additional public dollars on a dubiously rosy prospect of medical breakthroughs and cures, as they did in 2004. They would no doubt be delighted if they could also do-over the political argument that animated their first campaign, though to date Donald Trump has shown no interest in the issue. How voters will judge the promises, and how much weight they will give to non-monetary issues — built-in conflicts of interest, lack of oversight, outsourcing decisions about ethical standards, and the like — remains to be seen.”

The CIRM governing board has scheduled a Nov. 15 meeting to consider the proposed ballot measure. Changes in it can be made by its sponsor by the end of work Nov. 18. 

Public Participation Locations for Consideration of $5.5 billion California Stem Cell Finance Measure

Here is the current list of public locations for the Nov. 15, 9 a.m. meeting of the governing board of the California stem cell agency to consider the proposed $5.5 billion ballot initiative that would refinance the agency. 

The measure will also make major changes in the operations of the enterprise. 

The meeting comes only three days before the deadline for making changes in the proposal.

The meeting will be available on the Internet, making it possible for the public to participate remotely. More details are available on the agenda. The list of locations may change. Check the agenda to be sure a location is available. 

The listed locations are generally sites from which a member of the board can participate remotely in the meeting. 

Main meeting site: headquarters of the California Institute for Regenerative Medicine, as the stem cell agency is formally known, 1999 Harrison Street Suite 1650, Oakland, CA 94612

Telephonic locations:
10901 N. Torrey Pines Road, La Jolla, CA 92037
1001 Health Sciences Rd., Irvine, CA 92697
291 Campus Drive, Stanford, CA 94305
8700 Beverly Blvd 2015, Los Angeles, CA 90048
583 Mrak Hall, 1 Shields Ave., Davis CA 95616
100 Kimball Way, South San Francisco, 94080
12-231 CHS, David Geffen School of Medicine, UCLA
4005 N Fresno St., Fresno, CA 93726
837 Health Science Road, Irvine, CA 92697600 16th Street, Genentech Hall, S272D, San Francisco, CA 94158

California Stem Cell Directors to Air $5.5 Billion Refinancing Measure: Some Less Than Pleased

Directors of California's 15-year-old stem cell research program, which is now down to its last few millions, next week will take up a $5.5 billion proposal to refinance the effort, and not all of them like it.

The proposed ballot initiative has been submitted to state election officials. But it still can be changed up to Monday Nov. 18 at 5 p.m. That is three days after the 29-member governing board has scheduled a discussion of the complex, 30-page measure. That meeting is scheduled for Friday Nov. 15 at 9 a.m.

The only person who can make changes in the measure is Robert Klein, the first chairman of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. Klein is the official sponsor of the new initiative as he was on the initiative, Proposition 71, that created CIRM in 2004.  His current measure would go on the November 2020 ballot.

 Klein, a Palo Alto real estate investment banker, is also chairman and founder of Americans for Cures, a stem cell research advocacy group.

During a sometimes testy meeting last week, some CIRM directors said that Klein's new measure contained features that, in the words of one, were "not helpful." Directors did not go into details about the substance of the proposal, but debated mostly about whether it should be publicly reviewed by the board before the deadline for making changes.

Klein's original, 2004 measure financed CIRM with the issuance of state bonds, which are now running out. It did not provide other substantial sources of cash. Today the agency is down to roughly $27 million, which is earmarked for sickle cell research. CIRM directors gave away $54 million last Thursday for research into eye disease, Parkinson's and epilepsy, among other afflictions. 

Last Wednesday, CIRM Director Jeff Sheehy, who has served on the CIRM board since its inception, released to the California Stem Cell Report, a 3,300-word critique of Klein's new measure. On Thursday, Sheehy brought up Klein's measure at the board meeting during a discussion of CIRM planning in 2020. CIRM Chairman Jonathan Thomas ultimately said a number of directors had questions about the initiative and proposed a full board meeting before the deadline for changes.

The California Stem Cell Report has asked Klein via email whether he has closed the door to possible changes. Separately Klein has been asked for a written response to Sheehy's analysis, which the California Stem Cell Report will carry verbatim when it is forthcoming.

The Nov. 15 meeting is expected to be available online with public participation via the Internet. Telephonic sites are expected to be available in addition to CIRM's Oakland headquarters. Here is a link to the agenda with details.

(Editor's note: An earlier version did not carry a link to the agenda since it was not posted online at the time this item was written.)

California Stem Cell Agency Chalks Up its 60th Clinical Trial as Funds Dwindle

OAKLAND, Ca. -- The California stem cell agency last week awarded about $54 million for research into afflictions ranging  from eye disease to epilepsy as it edged increasingly closer to running out of cash.

Nine applications were approved including more clinical trials -- the last stage before therapies are approved for widespread use. The actions bring to 60 the number of clinical trials that the 15-year-old agency is helping to finance.

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known,  has about $27 million left, plus any additional funds that the agency might recover when awards are cut short as research fails to meet required milestones. The $27 million, however, is earmarked for sickle cell research in a partnership with the federal government.

In some past years, CIRM handed out as much as $300 million a year.

The agency's sole source of significant funding has been $3 billion in state bonds. It is pinning its hopes for the future on voter approval of a $5.5 billion ballot initiative in November 2020.  Playing a role in that effort is likely to be a CIRM-financed, economic study from USC that bolsters the argument that the economic benefits of the agency more than justify renewing its funding.

Dana Goldman, a professor of public policy, pharmacy and economics at USC and who directed the study, said that the private sector is not going to make the types of investments that CIRM has made in developing new treatments. He told the board last week that it would take only small improvement in success rates in such areas as stroke therapy to more than cover the cost of CIRM's activities. (Here is a link to his slides for the presentation.)

CIRM was created in 2004 by voters but has yet to invest in research that has resulted in a widely available therapy.

Maria Millan, CEO of CIRM. said in a news release:

“Programs, such as those funded today, that were novel stem cell or gene therapy approaches addressing a small number of patients, often have difficulty attracting early investment and funding. CIRM’s role is to de-risk these novel regenerative medicine approaches that are based on rigorous science and have the potential to address unmet medical needs. By de-risking programs, CIRM has enabled our portfolio programs to gain significant downstream industry funding and partnership."

Below is a list of the awards with links to the official summaries of the review of the applications, which can be found by clicking on the application number. Letters by applicants to the board can be found by clicking on the applicant's name. Additional letters of support for the research can be found on the agenda for the meeting. 

Here is the UCLA news release on the awards to three UCLA researchers.

Application Number	Amount  (in millions)	Institution	PI	Target
CLIN1-11591	$5.5	Stanford	Rosa Bacchetta	IPEX
CLIN1-11472	$6.6	UC Irvine/jCyte	Henry Klassen	Retinitis pigmentosa
CLIN2-11620	$10.5	Cedars-Sinai	Clive Svendsen	Retinitis pigmentosa
CLIN2-11661	$5.5	Brain Neurotherapy Bio	Krystof S. Bankiewicz	Parkinson’s Disease
CLIN2-11650	$10.3	UCLA	Sophia Deng	Limbal stem cell deficiency
TRAN1-11536	$4.9	UCLA	Caroline Y. Kuo	Immune deficiency
TRAN1-11555	$3.2	UCLA	Yvonne Y. Chen	Myeloma
TRAN1-11544	$2.9	City of Hope	Karen Aboody	Ovarian cancer
TRAN1-11611	$4.8	Neurona Therapeutics	Nicholas Corey (no letter posted)	Epilepsy