Showing posts with label Prop. 71. Show all posts
Showing posts with label Prop. 71. Show all posts

Thursday, November 19, 2020

'Demeaning and Uncalled For:' A CIRM Complaint About the Use of the Word 'Obscure'

The California stem cell agency today sharply criticized an item on the California Stem Cell Report as "demeaning and uncalled for" because it described the four-person Citizens Financial Accountability and Oversight Committee (CFAOC) as "obscure."

The offending word was used in an article yesterday that previewed the first meeting of the committee in nearly 15 months. The CFAOC was created as part of the initiative that created the stem cell agency in 2004, which is known officially as the California Institute for Regenerative Medicine (CIRM)

The CFAOC reviews CIRM financial documents that have already been reviewed in public by CIRM's 29-member governing board, which is officially known as the Independent Citizen's Oversight Committee (ICOC). The CFAOC has no legal authority over CIRM beyond making possible recommendations.

The article in question began like this:

"One of the more obscure entities in state government is scheduled to meet this Friday to review the financial affairs of California's stem cell agency, a matter that now runs to nearly $12 billion.

"The panel is the Citizen's Financial Accountability Oversight Committee (CFAOC), which was created in 2004 along with the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known. The CFAOC is charged with reviewing the financial practices of the stem cell agency. The CFAOC is the only state panel legally charged with that function. The governor and the legislature have no authority to do so."

Here is the text of the email expressing CIRM's displeasure with the use of the word "obscure." The email was written by Kevin McCormack, senior director of communication at CIRM.

"I think your characterization of the CFAOC in your latest column as 'One of the more obscure government entities' was demeaning and uncalled for. It may not have a high profile but it certainly has an important role. It was created by the voters in 2004 for a very specific purpose, to make sure we are held accountable, every year, for our financial performance. As one of the common, if erroneous, complaints about CIRM is that we are not accountable to the legislature or any other legislative body in Sacramento I would have thought the CFAOC should be praised not dismissed.

 

"This is a group of people who come together with the sole goal of making sure the state’s money is well spent. The members cover a broad range of professions but have one thing in common, they have vast experience in their area. They are appointed by the Controller, the Treasurer, the President pro Tempore of the Senate, and the Speaker of the Assembly (and one by CIRM). These are nobody’s fools, they are diligent in the way they work, meticulous in their analysis of how we work, and forceful in making recommendations. Which we then follow.

 

"At CIRM we are used to people criticizing us, more often than not incorrectly. The CFAOC deserves better."

As the author of the article in question, I have high regard for the CIRM staff and McCormack as well as the persons on the CFAOC. Nonetheless, it is an obscure state entitity, as are many state departments. More than 200 exist and most of them are obscure. Consider the State Allocation Board, for example, or the First 5 California


The count of 200 does not include state advisory bodies such as the CFAOC. If it did, the number would likely run into the thousands. Such bodies can do important work, such as was done by the Little Hoover Commission, an obscure state entity whose analysis and recommendations concerning CIRM won significant praise by the Institute of Medicine (IOM), which studied CIRM for months. 


What all these bodies have in common is that they are virtually unknown to the public, even to persons who consider themselves well-informed. 


The California Stem Cell Report writes about the CFAOC because of its connection to CIRM and the fact that both were created by the same 2004 initiative -- a measure criticized as ballot-box budgeting and that sometimes has also created impediments for CIRM. (See the IOM findings and recommendations.)


My bottom line? For a state body to be considered obscure is not a mark of shame. It is just a matter of fact.  


(Editor's note: A very early version of this item incorrectly said that Friday's meeting of the CFAOC will be the first in 19 months. The correct figure is 15 months.)

 

Monday, October 26, 2020

Proposition 14: First CEO of California Stem Cell Agency Says $5.5 Billion Stem Cell Measure Not Needed

Zach Hall, UCSD photo
The first president of the California stem cell agency, Zach Hall, says that he would vote against the $5.5 billion ballot measure to save the research enterprise from financial extinction if he still lived in California.

Hall, now retired and living in Wyoming, says a justification for agency existed in 2004 when it was created by voters via another ballot measure, the $3 billion Proposition 71. 

But, according to the new book, "California's Great Stem Cell Experiment," Hall says "that the rationale and need are not so evident today for a state-supported agency dedicated to stem cell research."  

The creation of induced pluripotent stem cells has largely supplanted the use of cells derived from embryos, Hall said. The Bush Administration restrictions on human embryonic stem cell research were major drivers for Proposition 71, but those have now been lifted.

Hall said that the National Institutes of Health could likely support most of the stem cell work that is now backed by CIRM.

Proposition 14, the $5.5 billion ballot initiative, would refill the stem cell agency's coffers. The program is running out of the $3 billion provided in 2004 and will begin  closing its doors this winter without major funding. 

Hall was president and CEO of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, from 2005 to 2007 and drew up the agency's first strategic plan. During his long career, Hall was also director of the National Institute of Neurological Disorders and Stroke in the 1990s, executive vice chancellor at UC San Francisco, CEO of En Vivo Pharmaceuticals and a director of the New York Stem Cell Foundation.

Interviewed for the book, which was authored by this writer, Hall said that he has no regrets about serving as its first president, a job he largely enjoyed. 

Hall said that Proposition 71 of 2004 served an important and useful purpose. It helped to re-energize the stem field at a time when it was “disheartened and demoralized” by the restrictions of the Bush Administration.

While the 2004 measure had significant flaws, he said it was very successful at a critical time in attracting stem cell researchers to California.

“The idea that California would make this sort of commitment, I think, had a huge impact on the field,” Hall said.

 “It's certainly true that because of Proposition 71 that California continues to play a stronger role in stem cell research than it otherwise would have. But, contrary to some expectations, it is not the center of the universe of stem cell research in the same way that Silicon Valley is for information technology.

“It is one of many global centers of excellence for stem cell research. One perhaps naïve expectation that has not been met is an explosion of profitable California biotech companies specializing in stem cell research.”

Hall said, however, that Proposition 14 “is searching for a rationale to continue CIRM.” Hall mentioned the “amorphous” research avenues provided for in the measure: mental health, personalized medicine, “aging as a pathology” and “vital research opportunities.”

“You can use the money for almost anything,” Hall said. “This takes off a lot of the brakes on how the money can be spent.”

Given what has been learned over the last 15 years, he said he would have thought a new initiative would have attempted to improve governance and try to make CIRM work better, be more efficient and more strategic. “There's just no sense of thoughtfulness of using the expertise of getting relevant people together to think about it and come up with a plan,” Hall said.

He also said that Proposition 14 does not provide a good or transparent mechanism for making decisions about how the money is going to be spent.

“My guess is that all the board positions will be filled by constituents, people who depend on CIRM money in some way and who will be very pliable about what is to be done. Exactly the wrong way to do it.”

The California Stem Cell Report asked Hall last week if he would like to add anything to his earlier comments for the book. "One thought I might add," he replied, "concerns the idea that Proposition 14 will address the issue of the 'Valley of Death', i.e. the gap between discovering a possible therapeutic and being able to 'de-risk it' enough to attract the interest of big pharma.  What is being proposed, it seems, is that CIRM wants to act as a kind of VC (venture capitalist) with the state's money.  

"In my view, a much better approach to this problem is to find ways to encourage academic and industry scientists (both biotech and big pharma) to work together starting from an early stage of the work. This has been done effectively (and much more cheaply!) by several private philanthropic organizations.  The Michael J. Fox Foundation and Target ALS are two examples that I know of." 

Tuesday, November 26, 2019

Media Coverage: California Stem Cell Agency Receives Slight Whack in Los Angeles Times

California's $3 billion stem cell research program, now down to only $27 million, popped up in the Los Angeles Times this week in a piece that dealt with presidential politics, term limits and dubious ballot measures.

The mention consisted of only two paragraphs. But it demonstrated, at least for the state stem cell agency, the flaws of excessive generalization in P.T. Barnum's famous saying that “Any kind of publicity is good publicity as long as they spell your name right.”

Los Angeles Times columnist Michael Hiltzik wrote yesterday about "crowd pleasing" pitches to voters that actually contain "few virtues."  The starting point was term limits for congressmen and women. Hiltzik noted that such measures, in fact, do not serve the public well because they enhance the power of lobbyists. And then he went on to discuss ballot initiatives in California. 

Hiltzik, a Pulitzer Prize winner and author of "Big Science," wrote, 
"It's not unusual for ballot measures to win the approval of voters despite provisions that work directly against voters’ interests. That includes Proposition 71 of 2004, which created California’s $6-billion stem cell program. (See below for a look at the $6 billion figure and the nominal cost of the stem cell effort.)
"Proposition 71 was sold to voters as a gateway to cures for a host of intractable diseases, a promise hopelessly incompatible with the way science is done. Its promoters also tried to make a virtue out of its nearly total independence from legislative oversight, another artifact of anti-government sentiment. The stem cell program has indisputably supported excellent science, but its lack of legislative oversight remains a flaw."
The lack of legislative oversight will continue under the proposed 2020, $5.5 billion ballot initiative that is now before California election officials. If approved by voters in 11 months, the initiative will save the California stem cell agency from financial extinction. 

The new initiative is sponsored by Robert Klein, the first chairman of the state stem cell agency. Klein's measure does not address a number of sharp criticisms of the program, including those found in a $700,000 study of the agency by the prestigious Institute of Medicine

Klein's new initiative is sort of a son of Proposition 71. Klein also directed the writing of the 2004 measure, which did not provide any source of stem cell funding beyond the $3 billion in state bonds. 

As for the $6 billion figure cited by Hiltzik, it originated in a 2004, state ballot analysis of Proposition 71. The figure was an estimate of the total cost, including interest, to taxpayers of the stem cell agency, which is financed by $3 billion borrowed by the state. 

The interest estimate did not anticipate the 2008 Great Recession and the related drop in interest rates. The latest estimate is that the total will be about $4 billion. Read more about that estimate here. 

Tuesday, October 29, 2019

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. 

Saturday, January 26, 2019

The Odd Stem Cell Position of the Golden State: Hype, Hope and Dubious Clinics


The California stem cell agency and the Golden State's robust scientific stem cell community received some notice this week in the the Los Angeles Times, the state's largest circulation newspaper. 

It came in the form of an op-ed article that decried the booming business enjoyed by unregulated stem cell clinics, a field where California leads the nation. The Times says it has 1.4 million readers daily and 2.4 million on Sunday.

Usha Lee McFarling, a Pulitzer Prize-winning journalist and currently an artist in residence at the University of Washington, wrote the article discussing the issues surrounding the dubious clinics and their considerable risks, which do not seem to discourage those seeking help. She said,
"So why do patients keep streaming in for treatments that cost thousands of dollars? Part of the reason, I suspect, is that stem cell research — the serious, scientific kind — has gotten so much hype in recent years. We’ve all heard about how some stem cells have the power to become any type of cell in the body and might one day offer cures for all manner of crippling and degenerative diseases. If you can jump the line, and get those treatments now, why not do it? 
"Here’s why: Because the days of miraculous cures, if they come, are far in the future. Today, there is only one federally approved stem cell product: the limited use of blood-forming stem cells to treat certain blood disorders. Scientists are just beginning to learn how to harness the power of stem cells, and the harsh reality is that clinical trials that could turn that knowledge into effective therapies will take years, if not decades."
McFarling continued, 
"California is in an odd position. It is the state with the most stem cell clinics in the country offering these unproven 'cures.' It also happens to be a world center of serious scientific stem cell research, thanks to a $3-billion ballot initiative, Proposition 71, passed by voters in 2004 to fund research."
She noted the fledgling efforts at the state and national level to deal with the dubious clinics. McFarling wrote, 
"Here’s an idea in the meantime. The many scientists who have benefited from taxpayer support of stem cell research in the state should start speaking out. After all, the hype from proponents of Prop. 71 (which created the state stem cell agency) is part of what created such high expectations for quick cures – and eagerness on the part of patients to get them. Scientists should now take every opportunity both to explain to the public the long-term goals of their research and the absurdity of the so-called cures now flooding the market."
Our take: Her advice to California researchers is sound. However, it should be noted that a number of researchers, notably Paul Knoepfler at UC Davis, have been sounding warnings for years. The mainstream media, meanwhile, largely ignored the problem. It took two scientists to do the legwork, which could have been done by journalists as well, that has been the key building block behind the current regulatory efforts, which are still in their infancy.

Thursday, November 15, 2018

California Kicks Off Major, New Foray into Gene Therapy: First by Any State in the Country

California today became the first state in the nation to launch itself into the sizzling field of gene therapy, backed by tens of millions of dollars and with the hope of creating treatments that could permanently cure afflictions ranging from hemophilia to cancer.

The move came today as the board of the $3 billion California stem cell agency opened its doors to funding gene therapy research that has reached the most advanced stage, clinical trials. The agency said that gene therapy -- minus stem cells -- is "valuable and worthy of pursuit."

"This is where the science is going," said Jeff Sheehy, chairman of the CIRM board's Science Subcommittee, at hearing earlier this month,

The state stem cell program has allocated $143 million for research programs next year that could include gene therapy.

A document prepared by the leadership of the agency, formally known as the California Insitute for Regenerative Medicine (CIRM), said, 
"For CIRM and the patients it aims to serve, it is vital to support technologies which prove to be highly complementary and augmenting to stem cells, such as gene therapy."
Gene therapy treatments are expected to be quite expensive, with some forecasts running in the $1 million to $2 million range. Supporters of gene therapy argue that the cost is justified because gene therapy can be a total cure that would eliminate the need for also very expensive lifelong treatments of chronic diseases. 

In its action today, the stem cell agency did not announce any specific research awards. Rather it created a procedure for declaring that a gene therapy project with a regenerative element is a "vital research opportunity."  That would allow CIRM to fund such a project under the terms of the ballot initiative that created the agency in 2004. Today's action is the first time that the agency has acted to use the "vital opportunity" provision. 

CIRM has budgeted $143 million for next year in two areas where the new gene therapy initiative could come into play, clinical trials along with translational research that is intended to advance basic research into clinical stages. 

The agency's new foray comes as its cash is running out. By the end of next year, it expects to have no more funding for new research. The agency is pinning its hopes of survival on a $200 million plus private fundraising effort this year and voter approval of a yet-to-be-written ballot initiative on the November 2020 ballot. 

The gene therapy field is moving swiftly and could generate a result that would resonate with voters and help to win approval of an additional $5 billion for CIRM.

CIRM board members have acknowledged that its new gene therapy effort will mean more competition for the state's research dollars, including possibly less for stem cell research, which is the agency's fundamental reason for being.


The agency, however, has already awarded millions of dollars for gene therapy research that has a stem cell link, including a program at UCLA that has led to a successful treatment for what is known as the "bubble boy syndrome." However, that research has not yet moved into the marketplace.

The target of the UCLA treatment is an immune deficiency that is fatal and for which there is no successful treatment outside of the experimental trials that are still underway. 

Bloomberg News has reported that Scott Gottlieb, head of the federal Food and Drug Administration(FDA), has described the field as "somewhat breathtaking." More than 500 experimental treatments are in the pipeline. 

Gottlieb has said that he expects the FDA to approve 40 gene therapies by 2022 and possibly a cure for sickle cell anemia within 10 years. CIRM is deeply involved in a major national push on sickle cell supported by the National Institutes of Health.


Thursday, November 08, 2018

California's Stem Cell Agency Opening Door to Pumping More Millions into the Sizzling Gene Therapy Market

Gene therapy graphic from FDA
The California stem cell agency today crossed a key threshold into the "somewhat breathtaking" and potentially multibillion-dollar world of gene therapy, a field that it has skirted previously. 

The action came when a key panel of the agency's governing board this morning approved its first-ever procedure for awarding millions of dollars for research not connected to stem cells. 

"This is where the (regenerative) science is going," said Jeff Sheehy, chair of the Science Subcommittee of the board of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known. 

Under Prop. 71, the ballot initiative that created the $3 billion agency in 2004, CIRM is limited to financing research involving stem cells in some fashion. However, a provision of the measure also allows the support of a "vital research oppportunity" under certain conditions. Today's action formalizes the process for making that finding. 

CIRM said in in a document that it is "vital" that the agency expand its horizons.
"CIRM has supported projects that combine stem cell and gene therapy technologies, such as the gene-corrected stem cell transplants at UCLA that essentially cured 5-year old Evangelina Padilla Vaccaro and several CAR-T cell approaches using stem memory T cells that aim to tackle various cancers. 
"The support of stem cell research that contributes to these treatments is and will continue to be the core of CIRM funding. However, treatment opportunities in regenerative medicine that utilize gene therapy technologies but not necessarily stem cells are also valuable and worthy of pursuit."
The agency's statement continued,
"The field of regenerative medicine brings together technologies that include stem cells, gene therapy, and tissue engineering that in many cases combine to produce a therapeutic product. In some cases, one technology leads the way. For CIRM and the patients it aims to serve, it is vital to support technologies which prove to be highly complementary and augmenting to stem cells, such as gene therapy."
Several directors noted during today's meeting that the change could lead to less cash for purely stem cell projects. 

The field of gene therapy has attracted widespread interest within the regenerative medicine industry and among federal medical regulators. Scott Gottlieb, commissioner of the Food and Drug Administration, earlier this year announced steps to speed development of the therapies. 

"'The pace of progress in gene therapy has been somewhat breathtaking,' he (Gottlieb) said, with more than 500 experimental drugs now in development. 'The promise is becoming very much a reality.'"
In April, Novartis AG ponied up $8.7 billion to buy AveXis Inc., of Illinois to strength its efforts to produce a marketable gene therapy.

Gottlieb has said that he expects the FDA to approve 40 gene therapies by 2022 and possibly a cure for sickle cell anemia within 10 years. 

CIRM's new gene therapy policy will go before the full CIRM board one week from today where it is expected to be approved. Additional information on the policy can be found on the Science Subcommittee agenda. 

Thursday, November 01, 2018

California Stem Cell Agency Moving to Expand its Reach into Big Market for Gene Therapy

The definition of gene therapy under proposed changes for
 research funding by the California stem cell agency. CIRM chart
California's $3 billion stem cell research program is set to add gene therapy -- minus stem cells -- as research that it will be able to legally finance. 

The change comes as the stem cell agency is looking to generate results that are likely to resonate with voters in November 2020 who may be asked to provide an additional $5 billion in funding for the program. The agency expects to run out of cash by the end of next year. 

Gene therapy has received considerable attention in the last few years. Yesterday, Orchard 
Therapeutics, a British gene therapy firm that has links to CIRM (also see here) and research by Donald Kohn of UCLA, raised $200 million in an initial stock offering. The company said in its prospectus that the total market potential "in the diseases areas underlying our five lead programs could be greater than $2 billion annually."

Donald Kohn, UCLA photo
Kohn has received nearly $31 million in backing from the stem cell agency, which is formally known as the California Institute for Regenerative Medicine (CIRM).

The Science Subcommittee of the agency is expected to approve extension of its gene therapy efforts next Wednesday in a teleconference meeting that will be available globally through the Internet. 

Under the provisions of the ballot measure that created the agency in 2004, the agency is limited in scope. But exceptions are possible if a finding is made that a "vital research opportunity" exists. 

Next week's meeting is expected to formalize the process of making that determination in regards to gene therapy that does not involve stem cells. It will require a 2/3 vote of CIRM's grant review group, among other things. 

Members of the public can participate in the hearing remotely via the Internet or at locations in Oakland, San Francisco, La Jolla, Riverside and Napa. Directions can be found on the agenda.

Wednesday, November 08, 2017

'Critical Stage' for $3 Billion California Stem Cell Effort and its Search for More Cash

California's $3 billion stem cell research program later this month is expected to unveil detailed plans for extending its life beyond the middle of 2020 in hopes of avoiding a lingering death.

The latest proposals, which are not yet public, are scheduled to be discussed Nov. 27. Possibilities range from another multi-billion dollar bond measure to private fundraising to possible merger with some sort of private entity.  

The stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), projects its cash for new awards will run out in about 2 1/2 years. At that point, unless more money is forthcoming, CIRM will only be overseeing the dwindling number of awards whose terms extend beyond June of 2020.

The agency's fate was dictated by Proposition 71, which created CIRM in 2004. It also provided $3 billion in state bond funding, which flows directly to CIRM without oversight by the governor or the legislature. No additional, significant resources were contained in the ballot initiative.

CIRM's future has been an occasional topic for its board for some time. But the issue has taken on more urgency this year. At a meeting in September of a newly formed Transition Subcommittee of the governing board, CIRM Chairman Jonathan Thomas said in what may have been an understatement,
"CIRM, as we know, is at a critical stage of its mission here." 
The meeting on Nov. 27 will additionally involve the board's Science Subcommittee. What emerges from the session will go to the full, 29-member board in December for ratification.

Options in September included a multi-billion dollar bond ballot measure in 2020 and a possible merger, which was described as something of a last resort. Whatever path is chosen, it likely would lead to changes in the agency, which has been criticized for conflict of interest issues and its dual executive arrangement, among other matters.

The agency has awarded $2.3 billion in 919 grants during its 13-year history. About 90 percent of the awards has gone to institutions with links to members of the governing board, past and present, according to calculations by the California Stem Cell Report.

So far the agency has not fulfilled expectations of voters that it would generate a widely available therapy. Something may emerge in the next couple of years from the 38 clinical trials currently backed by the agency. (Forty-three have been funded but five were terminated.) The trials, which can take years, are the last stage before a therapy is approved by the Food and Drug Administration for widespread use. CIRM plans to add more trials in the next couple of years.

This month's meeting will be based at CIRM's Oakland headquarters with teleconference locations elsewhere in the state where the public can take part. It is scheduled to begin at 1 p.m. PST and run until 4 p.m.

The California Stem Cell Report will provide full coverage of the meeting that day with advance information as it is posted on the CIRM web site.

(Here is a link to the transcript of the September meeting. Here is a link to Thomas' slide presentation.)

Sunday, October 08, 2017

Preview of Bond Campaign: California Stem Cell Agency Described as 'Dismal,' 'Disappointing' and a 'Waste'

A couple of die-hard opponents of the $3 billion California stem cell agency minced no words this weekend. In an op-ed piece in the Orange County Register, they described the agency as nothing more than an "advanced high school science project."

The article was written by Jon Coupal, president of the Howard Jarvis Taxpayers Association in Sacramento, and state Sen. John Moorlach, R-Costa Mesa. 

They described the agency's efforts as "dismal" and declared that "this disappointing abuse of taxpayer dollars" should be terminated.

The print version of the article appeared in Sunday's paper, which has a circulation of about 312,000. Both the print and online version carried photographs involving stem cell research. But neither was from UC Irvine, which has received $108 million from the agency and has a member on the board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.  UC Irvine is located only 17 minutes away from the offices of the newspaper. 

The piece is a relatively tame preview of the rhetoric that will confront CIRM if it goes to the ballot in 2020 for more billions from California taxpayers. The article cited arguments from the 2004 Prop. 71 ballot measure campaign that created the agency. 

Coupal and Moorlach said, 
"According to the ballot pamphlet mailed to voters, proponents promised the bond proceeds would advance the 'cure and treatment' of  'cancer, diabetes, heart disease, Alzheimer’s, Parkinson’s, spinal cord injuries, blindness, Lou Gehrig’s disease, HIV/AIDS, mental health disorders, multiple sclerosis, Huntington’s disease, and more than 70 other diseases and injuries.' 
"But actual outcomes for these promised advances are speculative at best and nonexistent at worst. Similar benefits were promised to the California economy to 'generate millions of new tax dollars.' 
"With such a dismal record, this would be a good time to shut the spigot on issuing the remaining $345 million — meaning some $690 million would be saved by state taxpayers. That money could be better spent on pensions, schools, roads, housing or better basic medical care for our residents."
As for the high school project business, the article said CIRM "continues to operate as a kind of advanced high-school science project, instead of moving toward the cures promised to voters in Prop. 71."

Wednesday, March 29, 2017

State Lawmaker Aims to End California Stem Cell Program, Calls It a 'Boondoggle'


State Sen. John Moorlach in video from his office

A California legislator has launched an effort to terminate the $3 billion California stem cell agency, which is already set to go out of business in about three years.

Republican state Sen. John Moorlach of Costa Mesa said in a video,
"It's time to shut this down....We as taxpayers need some protection. We need to stop the boondoggle."
Moorlach has authored a proposed constitutional amendment that has been referred to the Senate Health Committee. No hearing date has been set. The measure would strip from the state constitution the language that created the agency in 2004.

The proposal, SCA7, requires a two-thirds vote of both houses of the legislature and approval by a vote of the people. Given the Democratic dominance of the legislature, that makes the chances of enactment of SCA7 unlikely.

Nonetheless, Moorlach's effort reflects the sentiments of a certain segment of the public. It also provides ammunition for those seeking to fund the agency with another $5 billion, which would additionally be placed before voters, probably in November 2018.  It is useful for campaigns for such measures to be able to point to what they consider threats to science and medical progress.

Backers of a $5 billion bond measure are proposing it because the agency is slated to run out of cash for new research awards by June 2020.

Moorlach's office produced a short statement in support of elimination of the California Institute for Regenerative Medicine or CIRM as the agency is formally known. It said,
"California voters approved a ten year stem cell program that they thought would produce widespread cures and save thousands of lives. They were also promised revenue-producing intellectual property that would help the state financially. These remain empty promises.
"More than thirteen years after its passage, around $2 billion in funds have been dispersed and $1.2 billion has been spent on servicing the principal and interest of the debt . With a $1.6 billion dollar budget deficit and crumbling infrastructure, we need to stop the issuance of bonds on an ineffective and unaccountable agency. Scarce taxpayer funds could be of better use elsewhere."
Asked for a comment on the legislation, an agency spokesman, Kevin McCormack, said,
"We are aware of the bill and are monitoring it." 

Wednesday, December 21, 2016

'A Good Deal?' -- California Stem Cell Research, Rosy Expectations and Billions of Dollars

Highlights
$6 billion from the Golden State
$1.1 billion in royalties?
40 new clinical trials projected

"Are taxpayers getting a good deal?" That's the question that was raised nationally this week by the New York Times in connection with federally funded research dealing with life-saving treatments.

The same question can be raised concerning the roughly $6 billion, including interest, that California is spending -- unsuccessfully so far -- to generate a stem cell therapy that can be widely used.

In the case of the Times, the article by Matt Richtel and Andrew Pollack dealt with cancer immunotherapies and the soaring business and medical enthusiasm for the treatments. The two writers started their lengthy piece with the example of Kite Pharma, which they said "has struck gold." The company's stock has soared from $17 a share two years ago to about $50.

The Times wrote that excitement over the treatment speaks "volumes about the value of Kite's main scientific partner: the United States government."

California too is partnering with researchers and companies to develop blockbuster treatments, including immunotherapies. It has awarded more than $2 billion for research into possible treatments ranging from arthritis to extremely rare diseases that affect only a few thousand people.

The state is financing its research via the California Institute for Regenerative Medicine (CIRM), which is more informally known as the state stem cell agency. The agency has only $692 million left before its money for new awards runs out in 2020.

CIRM is funded by $3 billion that state borrows (bonds). The interest on the bonds roughly doubles the cost of the research compared to pay-as-you-go financing, which the federal government basically relies on. Like the federal government, CIRM depends on the private sector to actually bring potential therapies into widespread use.

The stem cell agency was created by voters in 2004 when they approved a ballot initiative. The campaign raised rosy expectations that cures were right around corner for afflictions that reached into nearly 50 percent of California families. And supporters also said that the state could expect as much as $1.1 billion in royalties. But royalties and commercial therapies have yet to appear.

The Times piece laid out arguments concerning federal research that apply equally to California's research effort, which is unprecedented in state history and which operates outside of the control of the governor and the legislature.  The Times piece said,
"Defenders say that the (public/private) partnership will likely bring a lifesaving treatment to patients, something the government cannot really do by itself, and that that is what matters most.
"Critics say that taxpayers will end up paying twice for the same drug — once to support its development and a second time to buy it — while the company reaps the financial benefit."
The Times article continued,
“If this was not a government-funded cancer treatment — if it was for a new solar technology, for example — it would be scandalous to think that some private investors are reaping massive profits off a taxpayer-funded invention,” said James Love, director of Knowledge Ecology International, an advocacy group concerned with access to medicines."
California's stem cell agency has yet to find a financial source to continue its work beyond 2020. Some talk has surfaced about another bond measure in 2018, but political observers give such an effort slight chance of success unless the agency can produce a therapy that will resonate with voters. If the Trump Administration, however, imposes restrictions on stem cell research, similar to those of the Bush Administration in 2004, that could create an impetus for passage of another measure.

Bob Klein, the first chairman of the stem cell agency, used to like to trot out the hundreds of scientific journal articles that were published by CIRM-funded researchers as a sign of success. "So what?" was the private comment to the California Stem Cell Report by one former CIRM staffer, alluding to the lack of impact of the journal articles. 

The agency's record, however, is picking up. Last week it cited 70 new projects, 10 new clinical trials plus a $30 million stem cell "pitching machine."  At at an emotional meeting, the agency's governing board also heard a mother thank the agency for saving the life of one of her children. Randy Mills, the president of the agency, is pushing hard and is looking for 40 additional clinical trials in the next several years. 

But the fundamental question remains: Will California taxpayers get their money's worth -- "a good deal" -- for the billions they are spending on stem cell research.   

Wednesday, December 07, 2016

Multi-Billion Dollar Ballot Measure for California Stem Cell Agency in 2018?

California's $3 billion stem cell research effort is scheduled to run out of cash in three short years, but the likelihood seems to be increasing that voters will be asked again to come up with additional billions for the state's stem cell agency.

In fact, the chairman of the stem cell agency, Jonathan Thomas, is saying flatly this week that his predecessor, real investment investment banker Bob Klein, intends to place a funding measure on the November 2018 ballot.

Klein led the ballot initiative campaign in 2004 that created the unusual -- for a state -- stem cell research program, officially called the California Institute for Regenerative Medicine (CIRM). The agency is financed with cash that the state is borrowing. increasing total costs to roughly $6 billion because of the interest expense.
Jonathan Thomas, left, with Don Reed,
vice president of public policy, Americans
for Cures, Klein's advocacy group --
CIRM photo

Thomas' statement was contained in a letter to the governing board that recapped his work since he was elected by the board in 2011 to replace Klein. One section of the letter dealt with funding of the agency.

Thomas wrote,
"Bob has already announced that he intends to put a measure on the November 2018 ballot. We keep him updated on CIRM's progress so that he is fully informed."
Thomas added that he and two key CIRM staffers have "initiated discussions with a number of philanthropists and foundations interested in medical research who could be potential sources of funds to keep CIRM going in the event Bob's measure is not successful."

The California Stem Cell Report has queried Klein about his plans. The full text of his response will be carried when it is received.

Robert Klein, Americans for Cures
photo
Klein has publicly mentioned a possible bond measure in the past, most recently in 2014, including a figure as high as $5 billion. It is unclear whether he has spoken more specifically on the matter since then.

Klein maintains a stem cell advocacy group, Americans for Cures, which has an active web site and an impressive list of scientific advisors, including Irv Weissman of Stanford, Rusty Gage of the Salk Institute and Owen Witte of UCLA.

Klein's 2004 stem cell campaign cost $34 million to convince California voters that the state needed to begin its own human embryonic stem cell (hESC) research effort in the wake of then President Bush's restrictions on federal funding in that area. The campaign created the impression that cures were close at hand, according to opponents and media observers. The agency is yet to back a therapy that is available for widespread use.

The election of Donald Trump as president is widely expected to trigger new, Bush-like restrictions on human embryonic stem cell research that could create the same sort of climate that helped lead to the success of the 2004 ballot initiative in California. 

Monday, August 15, 2016

Odds for a Stem Cell Cure vs. Odds on Trump Winning the Election

The article explored one particular search for a cure for diabetes, but the headline was bleak.
"Will Embryonic Stem Cells Ever Cure Anything?"
The question came at the top of a piece by Aleszu Bajak in the Aug. 12, 2016, edition of the MIT Technology Review. 

Bajak quoted Doug Melton of Harvard as saying,
“The public definitely doesn’t appreciate that much of science is failure.”
Bajak, a veteran science writer and journalism instructor, continued with his own conclusion, 
"In fact, no field of biotechnology has promised more and delivered less in the way of treatments than embryonic stem cells." 
Hard, hard language that probably disturbs many in the field, including some at the California stem cell agency, which was founded on the promise of therapies from human embryonic stem cells(hESC). The agency has committed more than $2 billion to stem cell research since 2005 and is still looking for its first big score.

One can argue the fairness of the conclusion by Bajak, but it certainly seems tied to the excessive rhetoric that has surrounded the hESC field, particularly the $34 million ballot campaign that created the $3 billion state stem cell agency in 2004.

The rhetoric of the campaign came with a price. The agency is scheduled to run out of cash for new grants in 2020. Whether it can deliver on the campaign promises of 2004 will in large part determine whether it can conjure up additional funding.

The likeliest prospects for success today can be found in the list of clinical trials the agency is helping to fund and the scores of additional trials that it plans to assist in the next several years. They include efforts to deal with afflictions ranging from heart disease to diabetes.

By one widely cited measure, only one out of 10 conventional therapies entering clinical trials reaches the market place. Those are slim odds, ones that do not involve novel stem cell efforts, which are presumably more difficult. To put it another way, Donald Trump has better chances -- as of this writing -- of being elected president than of a clinical trial producing a stem cell cure.

Trump, however, is only going to have one chance. The California stem cell agency is expecting to take more than 50 shots at a therapy via its involvement in clinical trials over the next several years.

Nonetheless, it is a risky business, as venture capitalist Gregory Bonfiglio, pointed out to the California Stem Cell Report last December.  He said,
“There are risks inherent in the development of new, disruptive technology. The bigger risk is failing to deliver on their underlying promise to bring new regenerative therapies to patients…. The bigger risk is not doing anything.”

Thursday, July 07, 2016

Monty Python, $3 billion and Stem Cells in the Golden State

Inquisition scene from Monty Python, photo CIRM/Daily Mail
The headline today from California was nearly irresistible:
"The Spanish Inquisition and a tale of two stem cell agencies"
Not only that, but the headline appeared over an article involving Monty Python, the $3 billion California stem cell agency and the lesser,  $38 million United Kingdom's Regenerative Medicine Platform(UKRMP)

The "Inquisition" article ran on the California agency's blog. The author was Kevin McCormack, senior director of communications for the California Institute for Regenerative Medicine(CIRM), as the agency is formally known. 

His topic? How the UK and California efforts have performed, according to an article by Stanford's Irv Weissman and Fiona Watt of King's College London, who is a member of the CIRM scientific advisory board, currently "on hold," according to McCormack.  The Watt-Weissman piece was recently published and buried behind a paywall in Cell Stem Cell.

McCormack noted that the article cited location and politics as important in both operations. He wrote, 
"CIRM was created by the voters of California in 2004, largely in response to President George W. Bush’s restrictions on the use of federal funds for embryonic stem cell research. UKRMP, in contrast was created by the UK government in 2013 and designed to help strengthen the UK’s translational research sector....
"Inevitably the two agencies took very different approaches to funding, shaped in part by the circumstances of their birth – one as a largely independent state agency, the other created as a tool of national government.
"CIRM, by virtue of its much larger funding was able to create world-class research facilities, attract top scientists to California and train a whole new generation of scientists. It has also been able to help some of the most promising projects get into clinical trials. UKRMP has used its more limited funding to create research hubs, focusing on areas such as cell behavior, differentiation and manufacturing, and safety and effectiveness. Those hubs are encouraged to work collaboratively, sharing their expertise and best practices."

Irv Weissman, photo wapiti-waters
Weissman and Watt said it was "not unexpected" that CIRM still has not produced a therapy for widespread use. They cited the sometimes decades-long time frame for transforming basic research into a therapy.

Which is where Monty Python comes in, said McCormack. The two scientists picked up a famous line from the British comedy series:
“Nobody expects the Spanish Inquisition – because our chief weapon is surprise.”
Fiona Watt, photo Watt Lab
McCormack wrote,
"They use that to highlight the surprises and uncertainty that stem cell research has gone through in the more than ten years since CIRM was created. They point out that a whole category of cells, induced pluripotent stem (iPS) cells, didn’t exist until 2006; and that few would have predicted the use of gene/stem cell therapy combinations. The recent development of the CRISPR/Cas9 gene-editing technology shows the field is progressing at a rate and in directions that are hard to predict; a reminder that that researchers and funding agencies should continue to expect the unexpected."

Thursday, June 23, 2016

Stem Cells, Long Odds and the 'Invisible Hand of Hype'

The headline on the California story pretty much told it all: "Stem Cells: Where Science, Hope and Hype Meet."

It could have added that it is also a meeting place for Big Pharma, Big Academia and Big Politics.

All of those yeasty ingredients are embodied in the $3 billion California stem cell agency, which is plugging away at developing a therapy promised to voters 11 years ago during a $36 million ballot campaign..

The headline appeared on the KQED Web site, a public television and radio outlet in San Francisco. The city, coincidentally, is where the world's largest aggregation of stem cell researchers, the International Society for Stem Cell Research (ISSCR), is meeting today. The conference is also just across the San Francisco Bay from Oakland, where the stem cell agency is headquartered.

Danielle Venton wrote the article for KQED. She covered a bit of the history of the California Institute for Regenerative Medicine or CIRM, as the agency is formally known. She noted that the agency is now participating in 16 clinical trials, although it has yet to chalk up production of a commercial therapy.

Venton wrote,
"(T)he frustration many voters feel about CIRM may have more to do with the problematic way researchers, institutional communicators and the media talk about scientific progress in general, and stem cells in particular, than it does with the agency’s performance.
Timothy Caulfield
"'There has always been this high-stakes, extreme rhetoric around stem cells,' says Timothy Caulfield, who teaches science and health policy at the University of Alberta. Caulfield says because stem cell research was so embattled, many spoke of its promise in hyperbolic terms.

"'People had to make bold statements about the future of stem cells in order to counteract those that wanted to have strict laws to stop it. So you have to say, ‘This is going to save lives. This is going to cure a variety of diseases.’ Right from the beginning, the late ’90s, you have that language appearing in the popular press.”  
Venton reported that the international scientists' group, the ISSCR, is "trying to tone things down" with new guidelines about how to talk about stem cell research and its impact. She said the effort "may be facing long odds." She quoted Caulfield as saying,
“It’s really the invisible hand of hype. In most cases these pressures are largely unconscious — whether you’re talking about the media, the researchers, the institutions or the funding agencies.”

Monday, June 06, 2016

$18.9 Million Spending Plan for California's Stem Cell Agency, Up Nearly 10 Percent

The California stem cell agency is set to approve tomorrow an $18.9 million operational budget for its coming fiscal year to pay for such things as employee salaries and pencils, an increase of nearly 10 percent over its current spending levels.

The budget will come before the Finance Subcommittee of the agency's governing board, which is expected to approve the proposal with few changes. The full board is expected to ratify the budget at its meeting June 15 in Berkeley.

The $3 billion agency operates under 6 percent spending cap imposed by Proposition 71, the ballot measure that created the research effort in 2004. The agency has $180 million (6 percent of the $3 billion) to spend on operational matters over its life, not including research grants. It has run through roughly $105 million.

When the $180 million is gone, that is the end of the money unless the agency can wangle extra cash from the legislature or private sources. Funding for both expenses and research grants is expected to run out in 2020.

CIRM documents show that the 10 percent budget increase over estimated spending for this fiscal year is tied largely to the fact that the agency did not hire as many persons as expected during the current fiscal year.  In the budget proposal, presented by Finance Director Chila Silva-Martin, the agency said it plans to fill out its staff about 55 in the coming year, although retention issues are likely to surface in the next few years because of the lack of funding beyond 2020.

Also playing a role in the bigger budget is a step up in the number of grant review sessions.

The largest expenditure for the fiscal year beginning July 1 -- $13.3 million --  is for salaries and benefits. The figure is up from $11.5 million for the current year. The second largest expense categories come from outside contracting and application reviews and meetings. Both categories have nearly identical totals -- about $1.8 million each. Outside contracting is about the same as last year but reviews and meetings are up from $1.4 million.

The public can listen in on tomorrow's session via a toll-free number. A teleconference site where public comment is possible is located in Irvine. That site is in addition to the main location in Oakland. Full details can be found on the agenda.

Sunday, May 29, 2016

The Unique Six Percent Cinch on California's $3 Billion Stem Cell Research Effort

A look at CIRM's calculations on the life left in its operational budget.
Highlights
Six percent budget cap
Restrictions courtesy of Prop. 71
Hearing on June 7

California's $3 billion stem cell agency may be the only state department that has a rigid legal cap on the total it can spend on operational expenses -- such things as employee salaries, office space, computers, meetings and much more.

When that money -- $180 million -- runs out, that's it, as far as the state goes. That is, unless the agency can wheedle more from lawmakers and the governor, which is an unlikely prospect at this time, given competing priorities for state spending.

The agency -- formally known as the California Institute for Regenerative Medicine or CIRM, -- has run through $105 million for administrative/operational expenses over its 11-year life. CIRM has about $75 million left and currently is burning up $15 million to $16 million annually.

The cap -- 6 percent of its $3 billion in bond funding -- was imposed by Proposition 71, the ballot initiative that created the agency in November 2004. From the very beginning, some members of the governing board questioned the cap, saying it was too chintzy for a research effort with such high aspirations.

Nonetheless, the cap is politically impossible to change at this point. It would require a super, super majority vote by the legislature or another vote of the people of California. Such an effort might result in efforts to bring the agency under legislative control. Currently, the governor and legislature have no legal say on how CIRM spends its money or how its awards are made, another feature of Prop. 71.

So parsimony has been a watch word at the agency, which, on June 7, will take up its budget for the fiscal year that begins in July. The CIRM board's Finance Subcommittee has scheduled a 90-minute session at its Oakland headquarters to consider the proposal. Following approval or any modifications, it is expected to be routinely ratified at a full board meeting in June.

The proposed budget is yet to be posted on the CIRM Web site and probably will not be available until later this week. The California Stem Cell Report will carry details on the plan when it becomes available.

The meeting has a teleconference location in Irvine where the public can participate in the discussion. Listen-only access is available via the Internet or through an 800 number. Details can or will be found on the agenda.

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