California's Big Research Tent: Beyond Stem Cells and Into VROs

California's $3 billion stem cell research program is unprecedented in state history, and it is now ready to mark another first: Backing a couple of clinical "VROs"  with $13.5 million.

VRO is a term that only a policy wonk could love or perhaps a researcher seeking funding under the rubric. It is not exactly posted on the home page of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. It is nestled comfortably among the 10,000 words of the 2004 ballot initiative that created CIRM.

VRO stands for vital research opportunity. It is fair to say that few persons are aware of the language or know that CIRM's funding authority is something of a big tent that can go well beyond stem cells. Or that CIRM's program could be more expansive under a new, proposed $5.5 billion ballot initiative.

CIRM's governing board set the stage for the awards last November when it approved a VRO process for gene therapy research that did not involve stem cells but involved an aspect of regenerative medicine.

According to the meeting transcript, an application for an award would be considered a VRO if, among other things, "the approach is intended to replace, regenerate, or repair the function of aged, diseased, damaged, or defective cells, tissues, and/or organ. This basically constitutes the definition of regenerative medicine and brings that as a requirement."
 
A VRO designation also requires a two-thirds vote of the grant reviewers, who conduct their meetings behind closed doors.

On Thursday, the board is expected to approve its first two VRO proposals. One for $8 million targets Parkinson's disease. The summary of the review said the research has "the potential to slow disease progression and provide amelioration of motor symptoms."

The other is a $5.5 million award for treatment of a rare autoimmune disease called IPEX. The summary of the review said the research offered "a valuable alternative to the current standard of care options, which have significant toxic side effects."

VRO awards are permitted under the 2004 ballot measure, which says:

"The institute shall have the following purposes: (a) To make grants and loans for stem cell research, for research facilities, and for other vital research opportunities to realize therapies, protocols, and/or medical procedures that will result in, as speedily as possible, the cure for, and/or substantial mitigation of, major diseases, injuries, and orphan diseases."

Currently on file with state election officials is a proposed ballot initiative that would provide an additional $5.5 billion for the agency, which is soon expected to run out of cash for new awards.

The measure would make a number of changes in CIRM, including permitting the agency to venture even farther afield than permitted under the 2004 language.

The measure would impose a new requirement that the agency support training programs for "careers in stem cell research and other vital research opportunities." It would establish experience in "other vital research opportunities" as acceptable criteria for the selection of governing board members and the chair of CIRM. The criteria would apply also to the grant review group and appointments to a new scientific advisory board.

The proposed ballot measure also defines a VRO as including personalized medicine, genetics and aging. Here is the proposed, new language:

"Vital research opportunity means scientific and medical research and technologies, includinq but not limited to qenetics, personalized medicine, and aqinq as a patholoqy, and/or any stem cell research not actually funded by the institute under paragraph (3) of subdivision (c) of Section 125290.60 which provides a substantially superior research opportunity, vital to advance medical science as determined by at least a two-thirds vote of a quorum of the members of the Scientific and Medical Research Funding Working Group (reviewers) and recommended as such by that working group to the ICOC (the governing board),or as determined by the vote of a majority of a quorum of members of the ICOC. Human reproductive cloning shall not be a vital research opportunity."

The language still can be modified by the initiative backers prior to being certified for the November 2020 ballot. Here is how that mechanism works. 

The 'Permissible' and 'Impermissible:' Admonitions Involving the California Stem Cell Agency and a $5.5 Billion Proposal

The 29 directors of the California stem cell agency are hearing a warning this week that certain types of their possible activities on behalf of a proposed $5.5 billion ballot initiative could lead to a criminal investigation by state or local law enforcement agencies. 

While that would seem to be an unlikely event, it has caught up another California public enterprise (the Bay Area Rapid Transit District). That's because state law restricts the use of public funds in connection with ballot campaigns. It is a complex subject, however, and laden with lots of gray areas. 

James Harrison
Remcho Johansen & Purcell photo

James Harrison, the former and longtime general counsel for the state stem cell agency, is scheduled to brief the board Thursday on the subject of "the permissible" and "the impermissible." Harrison is  well-known and respected for his work on election law and initiatives. 

He was deeply involved in drafting the $3 billion, 2004 ballot initiative that created the agency, known formally as the California Institute for Regenerative Medicine (CIRM). Harrison is also immersed in the proposed $5.5 billion measure for the 2020 ballot. It is designed to re-fund the agency, which expects to run out of cash this year for new awards. 

Harrison's memo to the CIRM board and his slide presentation on the use of public funds have been posted by the agency on its web site. In short, he says the key principles are:

"Communications about a ballot measure should be delivered through CIRM’s ordinary communication methods, like its website, blog, newsletter, emails to interested persons, and public meetings, in the style CIRM normally uses to communicate other information. CIRM should avoid passionate or inflammatory language and modes of communication that it does not regularly employ, and should not encourage voters to vote in a particular manner. For example, CIRM should not 'borrow the voices and the sympathy' of individuals who could benefit from the passage of the stem cell funding measure when discussing the measure, as BART did for Measure RR. CIRM should also take care to state past or present facts in a fair and dispassionate manner, and avoid using graphics, text, or video aesthetics that are similar to campaign advocacy (and different from CIRM’s normal practices). Taking steps like these will help ensure that the FPPC (Fair Political Practices Commission) or a court will conclude that CIRM is engaging in information sharing, rather than overt campaigning."

Harrison's presentation specifically discusses the BART case and others. The memo contains a list of specific permissibles and impermissibles. One permissible allows the board to vote on whether it supports the initiative. Another allows the agency to "prepare staff reports and other analyses to assist decision-makers in determining the impact of the measure and what position to take." 

Impermissibles include gathering signatures on the initiative and preparing materials for the public to use in support of the agency's position on the initiative. Working for an initiative on state time is barred, but CIRM employees can work for the campaign on their own time. 

Given the history and nature of California ballot campaigns, the state stem cell agency is certain to see public complaints that it is using public resources to support the initiative. The key, Harrison is telling CIRM directors, is to think about the context and content of proposed activities. 

His admonitions also include:

• "Ask legal counsel when you are not sure about a proposed activity.

• "Remember that even minimal use of public funds related to a measure can lead to investigation and litigation."

California Stem Cell Agency Posts $54 Million in Good News for Eight Researchers

Eight California researchers may be able to breathe a sigh of relief this morning with the news that the California stem cell agency has enough cash on hand to fund their applications to develop therapies for afflictions ranging from Parkinson's to eye diseases.

All eight of the applications have been approved already by the agency's reviewers. The agency's  governing board very rarely reverses the reviewers' actions. 

Until a day ago, it was uncertain whether the agency had the money to fund all the proposals. The good news for the scientists came in the form of a posting late yesterday on the web site of the agency, formally known as the California Institute for Regenerative Medicine (CIRM). The posting said that the agency has $54.2 million available for new awards as of Wednesday of this week (Oct. 23). The total for the eight application is about $52 million. 

Of course, it is possible that the board could move in a different direction based on considerations not readily apparent from the agenda for its meeting next Thursday. 

If the agency has $2 million or so left after next week's meeting, it is also unclear what it will do with the cash. One possibility is that it could retain the cash for awhile and combine it with funds that might be recovered from awards that have not met milestones.  So far this year, the agency has recovered $30.3 million.

CIRM has expected to run out of cash this year for new awards after dispensing $2.7 billion over the last 15 years to 1,017 recipients. It is hoping voters will approve $5.5 billion more via a ballot initiative in November 2020. 

Thursday's meeting does have another financial fillip. Listed on the agenda is a line that says a report will be made on private donations. That could be good news or not-so-good news depending on how successful its private funding efforts have been over the last couple of years. Results of that drive are yet to be publicly announced. 

(Editor's note for careful readers: The CIRM document that contains the $54.2 million figure also lists $57.2 million in applications. One of those applications, a $5 million proposal, was not approved by reviewers, however.) 

Want to Speak to the Directors of the California Stem Agency? Here is Your Chance.

The $3 billion California stem cell agency meets Oct. 31 to consider applications for $52 million in research funding. Also on the table are issues ranging from its finances to an initiative that would re-fund it with $5.5 billion. The agency expects to run out cash for new awards by the end of the month.

The meeting is open to the public with its main location at its headquarters in Oakland. Here is the current list of locations, which may change. The public can also participate via the Internet as the meeting is streamed live. See the agenda for instructions. 

Here are links to items dealing with issues before the board. "Last Research Awards...." "Competition for $52 Million..."

CIRM HQ: 1999 Harrison Street Suite 1650, Oakland, CA 94612

Other locations, which are usually hosted by a member of the agency's governing board: 

10901 N. Torrey Pines Road, La Jolla, CA 92037
4005 N Fresno St., Fresno, CA 93726
765 Market Street, #31D, San Francisco, CA 94103
9500 Gilman Drive #0602, La Jolla, CA 92093-0602
291 Campus Drive, Stanford, CA 94305
Medical Sciences 1C, Room 110, Davis, CA 95616

California Stem Cell Researchers Compete Next Week for $52 Million in Awards

Directors of the California stem cell agency will make yes-or-no decisions next week  on eight applications for $52 million to seek cures for afflictions ranging from Parkinson's to an incurable eye disease called retinitis pigmentosa.

All eight have been approved behind closed doors by the agency's grant reviewers, but it is yet to be determined whether the agency has enough cash to ratify that action.

Prior to voting on the applications, the 29-member board is scheduled to discuss how the agency's final funding will be parceled out. The agency expects to run out of cash for new awards at the end of the Oct. 31 meeting. However, also on its agenda are a few words promising a report on the status of its search for private donations. 

Below is a table on the applications. It includes links to the review summaries prepared by the agency, which is formally known as the California Institute for Regenerative Medicine. Links to the review summary can be found on the application numbers.

Some of the applicants have filed additional letters with the board seeking to bolster their pitches. The letters can be found by clicking on the researcher's name. Some of the applicants are expected to address the board personally Oct. 31 in Oakland.

The agency has a non-embarrassment policy of not disclosing the names of applicants and their institutions until a vote by its board.  However, when they write the board, their names become a public record. The names of applicants who fail to win approval are never disclosed by the agency.

Look for more stories in the upcoming days from the California Stem Cell Report on the agency's Halloween meeting.

Application Number	Amount In millions	Institution	Principal  Investigator	Target
CLIN1-11591	$5.5	Stanford	Rosa Bacchetta	IPEX
CLIN1-11472	$6.6	Not disclosed	Not disclosed	Retinitis pigmentosa
CLIN2-11620	$10.5	Not disclosed	Not disclosed	Retinitis pigmentosa
CLIN2-11661	$8.0	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	Not disclosed	Not disclosed	Ovarian cancer

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 Stem Cell Health Dividends: A Whopping $900 Billion? Maybe Only $175 Billion?

A team of University of Southern California researchers said today that work by California's stem cell agency could pay off with nearly $900 billion in "health dividends" by 2050 by treating or curing afflictions ranging from diabetes to heart disease.

The study, commissioned by the agency, acknowledged the difficulty in forecasting the benefits of research backed by the the state program, which is unprecedented in California history. But the report said,

"We find that nearly half of Californians aged 50 and older will develop diabetes during their lifetime. Furthermore, more than one-third will experience a stroke, and between 5 and 8 percent will develop either breast, colorectal, lung, or prostate cancer.  

"Taking into account the high prevalence and the social and economic burden of these diseases, an intervention that reduces the incidence of these selected cancers, diabetes, and stroke by 50 percent would generate almost $900 billion in social value between 2018 and 2050.  

"A more modest 10 percent decline in incidence translates to $175 billion in social value during the same period. Put in this context, the CIRM investment would be worthwhile if it increased our chances of success even modestly. Against the billions of dollars in disease burden facing California, the relatively small initial investment is already paying dividends as researchers work to bring new therapies to patients."

The agency, known formally as the California Institute for Regenerative Medicine (CIRM), was created by voters in 2004, who provided it with $3 billion. It has yet to fund a product that is widely available to the public. However, the agency is involved in 56 clinical trials, which are the last stage before a treatment is certified for widespread use.

CIRM expects to run out of cash for new awards by the end of this month. It hopes that a proposed initiative for the November 2020 ballot will provide it with an additional $5.5 billion. 

Today's report, titled "Future Health Dividends for California," and its companion study last week on the agency's economic impact are likely to be significant topics during the 2020 campaign. 

The 22-page study came up with a "social value" calculation using the Future Elderly Model -- "a microsimulation model of health and economic outcomes for older Americans." Basically it involves quality of life outcomes as well as more straight forward financial projections. The study also lays out its methodology and limitations including an acknowledgement that the projections are not guaranteed. 

Today's report and last week's economic study cost CIRM a total of $206,000. The work was performed at USC's Leonard D. Schaeffer Center for Health Policy & Economics. The authors are Bryan Tysinger, Karen Mulligan, Henu Zhao, Alwyn Cassil  and Dana Goldman.

Here is a link to an item on the CIRM blog about the study.

Making Changes in a $5.5 Billion Stem Cell Matter: What California Law Permits

Screenshot from state Department of Justice "active measures" website

A  California ballot proposal to spend an additional $5.5 billion over another 10 years on stem cell research is not locked in concrete or in even locked in its dense, legal language.

It can actually be modified between now and Nov. 17 if its sponsors so desire. That may be a big if, but it is possible as a result of 2014 changes in state elections law.

Here is the way it works. Any member of public can comment or suggest changes in proposed ballot initiative between now and Nov. 12 on this state web site.  The Department of Justice will pass along the suggestions to the sponsor of the initiative. The sponsor can make changes in the initiative up to Nov. 17.

Whether changes actually occur depends on pressures brought to bear by various constituencies that have something at stake in this multibillion-dollar game. Those interests include most of the Golden State's major universities and research institutes, the biotech industry, the fledgling California stem cell industry, patient advocate groups ranging from diabetes to cancer and even national regenerative medicine groups, not to mention religious or other groups that may have ethical concerns.

Of course, suggestions do not have to be filed with the attorney general. They can go directly to Robert Klein, who filed the initiative. Klein is a Palo Alto real estate investment banker, who founded the stem cell advocacy group, Americans for Cures. He led the 2004 campaign to create the agency and served as its first chairman. Klein directed the writing of the 2004 initiative as well as the current proposal.

His group also has a scientific advisory board that includes a host of California stem cell luminaries ranging from Irv Weissman of Stanford and Larry Goldstein of UC San Diego to Leslie Thompson of UC Irvine and Arnold Kriegstein of UC San Francisco. Klein has said he consulted with them in drafting the latest ballot proposal. They may well be interested in hearing comments on what is now being offered.

The California Stem Cell Report has already heard murmurings about changes that backers of the agency would like along with similar thoughts from opponents. But the measure is quite fresh and 30 pages long. So it may take a little time for solid suggestions for changes to emerge. 

The governing board of the stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), has scheduled a meeting for Oct. 31, its first since the initiative was filed. It is expected to act on a number of awards, which are expected to be its last new awards until it receives additional funds. 

Asked last Friday whether board might make suggestions for changes in the initiative, Kevin McCormack, senior director for communications, replied via email, 

"Right now the CIRM board hasn’t taken a position on it. At some point in the future the board will discuss the initiative at a public meeting and take a position."

Here is the contact link for Klein's Americans for Cures and the email address for the stem cell agency: info@cirm.ca.gov 

Re the suggestion period and amendments to the initiative, here is the salient language from the California Secretary of State, the state's top elections official.

"Once a request for a circulating title and summary has been submitted, the Attorney General’s Office will post the text of the proposed initiative measure on their website and facilitate a 30- day public review process during which any member of the public may submit written public comments to the Attorney General’s Office via their website. The Attorney General’s Office will provide any written public comments received during the public review period to the proponent(s). (Elections Code § 9002(a).) During the public review period, amendments signed by all of the proponent(s) may be submitted to the Attorney General’s Office. Any amendments to the proposed initiative measure must be reasonably germane to the theme, purpose, or subject of the initiative measure as originally proposed. An amendment shall not be accepted more than five days after the public review period is concluded. (Elections Code § 9002(b).)"

It's Official: $5.5 Billion Ballot Measure Filed for California Stem Cell Agency

Backers of the financially stressed California stem cell agency yesterday filed their proposed ballot measure to refinance the agency with $5.5 billion if voters approve it in November 2020.

The complex, 30-page initiative would also restructure a number of aspects of the agency and provide for financial assistance for patients and their families who might be involved in clinical trials. The proposal was submitted by Robert Klein, the Palo Alto real estate investment banker who led the ballot campaign that created the agency in 2004. 

Klein was also the first chairman of the agency, known formally as the California Institute for Regenerative Medicine (CIRM).  He is chairman of the stem cell advocacy group, Americans for Cures.

The $3 billion agency expects to run out of cash for new awards at the end of this month.

In addition to stem cell research, the proposed initiative would provide for awards for other "vital research" opportunities. 

Training for professionals would emphasized. The governing board of the agency would be increased from 29 to 35 persons. Representatives of the California state university system would have a greater official role in addition to representatives from the University of California. 

Additional changes are proposed in priorities along with alteration in conflict of interest rules and its public records provisions.

The measure, "Initiative No. 19-0022," will need 633,212 valid signatures to qualify for the ballot. The first step in the months long process is review of the measure by the attorney general's office, which will prepare the official summary.

The initiative said,

"Although California's stem cell research funding institute has made great gains, much work remains to be done. With new federal restrictions on important research, an anti-science agenda on the rise, and threats to reduce federal research and development funding, California once again must take the lead to ensure that this promising area of research continues and to advance projects from the research stage to the clinic. 

"Without additional funding, many of these promising research and development projects will be forced to stop work on potentially life-changing medical therapies. California's stem cell research institute needs additional funding to help bring promising discoveries through the development process, including clinical trials, with the goal of making treatments available to California patients with chronic diseases and injuries"

The public can file comments on the proposal at this page on the web site of the attorney general. 


(An earlier version of this article did not include the paragraphs beginning "Although California's..." and "Without additional...")

Critics Unmoved by Economic Report; Still Say No More Billions for California's Stem Cell Agency

This week's relatively rosy report on the economic impact of California's $3 billion stem cell research program has not convinced longtime critics to change their positions and support giving it an additional $5 billion.

In the eyes of some opponents, the stem cell agency is still a boondoggle, a waste of money and an inappropriate use of state bonds, the borrowed money that is the only significant source of cash for the program. Other critics recommended moving the program to the University of California and restricting it to "breakthrough medicine."

The agency, known formally as the California Institute for Regenerative Medicine (CIRM), expects to run out of money for new awards this month. It is hoping that voters will see value in its efforts and approve -- in November 2020 -- a $5.5 billion ballot initiative that would refinance the agency.

The 2004 initiative that created CIRM was handily approved by 59 percent of the voters following a campaign that created expectations that nearly miraculous therapies were right around the corner. The agency has yet to back a treatment that is widely available to the public.

The California Stem Cell Report queried a smattering of CIRM's critics following the release of the economic study, which said the agency has provided a "handsome dividend" to the state. The report from USC said, among other things, that CIRM-generated benefits exceed $10 billion and have led to nearly 60,000 jobs.

That was not good enough for state Sen. John Moorlach, R-Costa Mesa. He said,

"It was a boondoggle when it started and it still is. It's held up by emotional appeals, sustained by empty promises, and now rationalizing to extend its existence."

He continued,

"This study does not bring to light anything new. Doling out $3 billion to any entity or entities would generate the same economic metrics. The study is a shell."

"CIRM did not produce or sell anything of substance. It was not a stadium that created jobs around it, like restaurants, bars, and hotels."

Marc Joffe, a senior policy analyst at the Reason Foundation, said,

"I oppose the use of state general obligation bonding authority for any purpose other than building well-conceived civil infrastructure projects. The fact that spending bond proceeds generates economic activity is not surprising and not a reason to support a new bond in 2020. Similar studies have been released in support of the ill-conceived high-speed rail project: We might not get a usable system that takes many passengers out of their cars, but at least we created a lot of jobs in the Central Valley! This is not a persuasive argument for imposing more debt on our children, who already have the challenge of paying for Baby Boomer retirements."

Joe Rodota also responded. Rodato has worked for two Republican California governors. He and Bernard Munos, a senior fellow with FasterCures and the founder of the Innothink Center for Research in Biomedical Innovation,
have advocated a change in California's stem cell program that would restrict funding to businesses with a significant California presence and move the effort to the University of California.

Rodota said,

"Under our proposal, in exchange for providing funding to private companies engaged in developing stem-cell therapies, the University of California would receive shares, alongside any shares sold to private investors. Although the report categorizes equity sold to private investors as part of the 'economic stimulus created by CIRM funding,' that equity is held by private investors, not the University of California."

You can read the full text of the critics' remarks here. 

Full Text: Critics on Economic Impact Report on California Stem Cell Agency

Here is the full text of the email responses of stem cell agency critics to the report on the economic impact of the agency over the last 15 years.

State Sen. John Moorlach, R-Costa Mesa, who responded directly to questions from the California Stem Cell Report (CSCR):

CSCR: "Does the USC study bring something to light that is new to you?"
Moorlach: This study does not bring to light anything new. Doling out $3 billion to any entity or entities would generate the same economic metrics. The study is a shell.
"CIRM did not produce or sell anything of substance. It was not a stadium that created jobs around it, like restaurants, bars, and hotels.
"CIRM spent $3 billion and it's costing the taxpayers that much, plus $800 million-plus in interest."
CSCR: "Does it lead you to modify your thinking about the California stem cell program?"
Moorlach: "The new report has not modified my thoughts on CIRM. It was a boondoggle when it started and it still is. It's held up by emotional appeals, sustained by empty promises, and now rationalizing to extend its existence."
CSCR: "What is your overall view of the agency at this point?"
Moorlach: "Stem cell research is important, but best left to the private sector. This was style drift and extremely expensive, including the necessity to raise taxes with Propositions 30 and 55."

From Marc Joffe, senior policy analyst with the Reason Foundation:

"I oppose the use of state general obligation bonding authority for any purpose other than building well-conceived civil infrastructure projects. The fact that spending bond proceeds generates economic activity is not surprising and not a reason to support a new bond in 2020. Similar studies have been released in support of the ill-conceived high-speed rail project: we might not get a usable system that takes many passengers out of their cars, but at least we created a lot of jobs in the Central Valley! This is not a persuasive argument for imposing more debt on our children, who already have the challenge of paying for Baby Boomer retirements."
"Also, for what it’s worth, I don’t oppose the existence of CIRM. If it can be financed privately, that would be wonderful. I just don’t think it should be imposing burdens of future taxpayers."

From Joe Rodota, who worked for Republican Gov. Pete Wilson and Republican Gov. Arnold Schwarzenegger.

"This sentence (from the study) caught my attention 

"The estimates in the report are based on the economic stimulus created by CIRM funding and by the co-funding that researchers and companies were required to provide for clinical and late-stage preclinical projects. The estimates also include:
"Investments in CIRM-supported projects from private funders such as equity investments, public offerings and mergers and acquisitions, 

"Our (Rodota's) proposal, as outlined here, includes this language:
"In exchange for the funds they receive, companies would tender to the University of California shares of their common stock, with an estimated value as determined by the most recent outside valuation or price set by investors. These shares would become part of the UC endowment -- and the University of California be free to sell or leverage these shares, or acquire additional shares, as it sees fit.

"Under our proposal, in exchange for providing funding to private companies engaged in developing stem-cell therapies, the University of California would receive shares, alongside any shares sold to private investors. Although the report categorizes equity sold to private investors as part of the 'economic stimulus created by CIRM funding,' that equity is held by private investors, not the University of California."

'Handsome Dividend' -- California Stem Cell Agency and Its Economic Impact on the Golden State

This video was produced by Forty Seven, Inc., a firm in which California's stem cell agency has invested more than $15 million. 

California’s stem cell research program has had a “major impact” on the state’s economy, generating billions in sales revenue and creating tens of thousands of new jobs, according to a study released today.

Commissioned by the $3 billion state stem cell agency, the report comes as the program is hitting a difficult financial patch. Known officially as the California Institute for Regenerative Medicine (CIRM), the agency expects to run out of money for new awards by end of this month. CIRM is hoping that voters will refinance it with $5.5 billion via a ballot initiative in November 2020.

The 84-page study is expected to serve as a rebuttal to critics who have called the nearly 15-year-old agency a boondoggle. The report said, 

“The state’s investment in (the agency) has paid handsome dividends in terms of output, employment and tax revenues for California."

Adam Rose, USC photo

Dan Wei, USC photo

The study and a yet-to-be released companion report were commissioned at a cost of $206,000 by the agency. The economic study was prepared by Dan Wei and Adam Rose of the Price School of Public Policy at the University of Southern California. 

Maria Millan, CEO and president of CIRM, said in a news release that the study reflects the agency's role in building a stem cell "ecosystem" in the Golden State. 

Beyond CIRM's medical and scientific work, she said that the agency is "promoting economic growth in California  by attracting scientific talent and additional capital, and by creating an environment that supports the development of businesses and commercial enterprises in the state."

The report summarized CIRM's economic impact in four points. 

• "$10.7 billion of additional gross output (sales revenue)
• "$641.3 million of additional state/local tax revenues
• "$726.6 million of additional federal tax revenues
• "56,549 additional full-time equivalent jobs, half of which offer salaries considerably higher than the state average."

CIRM's news release on the report characterized the study as "independent." CIRM said it showed that the agency's efforts had a "major impact" on the state's overall economy, which totalled $3 trillion in 2017. 

The agency cited the assistance it has provided to create companies that ultimately will make CIRM-financed therapies available to the public at large.

While the agency's spending has not yet led to a widely available therapy, it is backing 56 clinical trials, which is the last stage before a treatment can be approved for widespread use. About 86 percent of clinical trials fail to result in a product, according to 2018 figures.

As an example of a fruitful collaboration, the agency cited Orchard Therapeutics of the United Kingdom, which plans to seek to qualify soon for speedier federal approval of its treatment for a version of  "bubble boy syndrome," a fatal immune deficiency. CIRM has awarded the firm $8.5 million.

The treatment was developed by Donald Kohn at UCLA with the help of $52 million in CIRM cash.  During clinical trials, it has saved the lives of more than 50 babies. Kohn said in a statement,

"I think one of the greatest strengths of CIRM has been their focus on development of new stem cell therapies that can become real medicines."

Orchard has offices in the San Francisco Bay area and plans to build a 150,000-square-foot manufacturing facility in Fremont. 

Also cited as an example was a company called Forty Seven, Inc., of Menlo Park, Ca., which is developing cancer therapies. Mark Chao, founder of the firm, said CIRM's support was "instrumental to our early successes."

The economic study also explored the "deal flow" funding that has aided commercialization of research. The study said it is expected that 

"...CIRM's past and current funding will attract increasing amounts of industry investment and lead to additional spending injections into the California economic in the years to come."

The companion report to today's economic study involves "health dividends" provided by the agency. That report is expected to be released next week. 

The agency has commissioned other economic studies in the past including one in 2012 that also lauded the agency. The request for proposals to perform that 2012 study said it must execute "a vibrant and aggressive strategy to support the goals and initiatives of CIRM.” 

Queried by the California Stem Cell Report, Kevin McCormack, a spokesman for CIRM, said four enterprises were solicited to develop this year's study. Three declined. The contract for the latest study stipulated that USC had control of the content. The latest study also laid out the methodology in considerable detail, something missing from the 2012 report. 

Below is a May 31, 2019, 58-second video of the president of Orchard, Mark Rothera, discussing the company's work. More brief, 2019 videos of Rothera from same interview sequence can be found here, here, here and here. The videos were taken by the Alliance for Regenerative Medicine. 

California's $72 Million Diabetes Wager: ViaCyte Announces Major "Firsts" for Its Stem Cell Therapy

Vox Pop video/Viacyte

One of California's bigger stem cell bets -- $72 million -- turned up this morning with a strong positive report that included a couple of "firsts" in its search for a virtual cure for diabetes.

The announcement came from ViaCyte, Inc., of San Diego. The California stem cell agency has pumped $72 million into the company, making the firm the top for-profit recipient of state stem cell largess. 

The news comes as the agency, known formally as the California Institute for Regenerative Medicine (CIRM), is running out of funds and hoping that voters will give it $5 billion more. A major research score would be a big plus for that ballot initiative effort. 

The agency's president, Maria Millan, described the ViaCyte announcement as important and encouraging. 

ViaCyte is developing a tiny device that is implanted in a person's body and that generates insulin as needed. It is aimed primarily at type one diabetes, which afflicts more than one million Americans 

ViaCyte issued a news release on the developments at major, national stem cell conference in Carlsbad, Ca. The headline on the release said, 

"First demonstration of insulin production in patients from a stem cell-derived islet replacement therapy"

The release said,

"Preliminary data show that implanted cells, when effectively engrafted, are capable of producing circulating C-peptide, a biomarker for insulin, in patients with type 1 diabetes."

Paul Laikind, CEO and president of the firm, declared,

“ViaCyte is the first and only company in human clinical trials with a stem cell-derived islet replacement therapy candidate, and we are now the first to demonstrate production of C-peptide in patients receiving implanted stem cell-derived islets. These data show that our PEC-01 cells are functioning as intended when appropriately engrafted. “While there is still more work to be done, this is an important milestone. We plan to present additional data in the near future.”

Laikind continued,

“ViaCyte has achieved a number of firsts in this field. Now with the first demonstration of insulin production in patients who have received PEC-Direct, we are confident we can be the first to deliver an effective stem cell-derived islet replacement therapy for type 1 diabetes.”

Asked for comment, CIRM's Millan said,

"This is encouraging news. We are very aware of the major biologic and technical challenges of an implantable cell therapy for Type 1 Diabetes, so this early biologic signal in patients is an important step for the ViaCyte program."

ViaCyte is scheduled to present its findings later today at the Cell & Gene Meeting on the Mesa. That session can be seen live on the Internet 1:45 p.m. PDT. 

(An earlier version of this item contained a slightly different quote from Millan. CIRM re-submitted the latest quote, which adds information.)

Measure to Add $5 Billion-plus to California Stem Cell Agency Yet to be Filed

The official kickoff for a proposal to refinance California's nearly 15-year-old stem program with $5.5 billion has been delayed for at least a few more weeks. 

Backers of the proposed bond initiative told the California Stem Cell Report last week that the ballot measure will not be filed with state election officials until later in October. Previously, they had said it would be filed by the end of this month. 

Melissa King, executive director of Americans for Cures, said there was no major reason for the delay in filing the measure. She said it was just a matter of "wanting to get everyone's views heard, etc."

To qualify for the November 2020 ballot, backers will need to gather more than 600,000 signatures of registered voters. 

Americans for Cures is a stem cell advocacy group controlled by Robert Klein, who ran the initiative campaign in 2004 that created the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.
Klein was also the first chairman of CIRM. 

The agency expects to run out of cash for new awards this year and has no further source of major funding.