Wednesday, May 22, 2019

Looking Back and Forward at the California Stem Cell Agency: 1,000 Awards, $2.6 Billion, 1,200 Patients in Clinical Trials

A CIRM slide from tomorrow's presentation
on the stem cell agency's work.

The California stem cell agency has whomped up a fulsome presentation on its progress since it was created in 2004 and plans to unveil it at a public meeting tomorrow in Oakland. 

The presentation covers everything from cell therapy to de-risking stem cell investing for biotech companies. Maria Millan, CEO of the $3 billion enterprise, will run through the agency's 1,000 awards and 53 clinical trials during tomorrow's meeting of the governing board of the research effort. 

Her presentation comes at an increasingly critical time for the agency. It expects to run out of cash for new awards later this year. It is attempting to raise $220 million privately to help tide it over until November 2020, when it hopes to persuade California voters to give it another $5 billion. 

The agency, known formally as the California Institute for Regenerative Medicine (CIRM), is making the meeting available to the public through its usual Internet audiocast in addition to teleconference meeting sites in Riverside, La Jolla, South San Francisco, Fresno, San Diego, Palo Alto and two in Los Angeles.  That is in addition to the main meeting site in Oakland. (Full directions are on the meeting agenda.) The public can also ask questions via the Internet or the teleconference locations. 

Also up for tomorrow's meeting is discussion of possibilities of changes in CIRM's operations that might be embodied in a ballot initiative in 2020. Two governing board committees mulled over a wide range of options at a session last week, but no decisions were made. The measure is yet to be written but will need to be officially filed later this year.  Sphere: Related Content

Tuesday, May 21, 2019

California Stem Cell Funding Flap: Stanford Ponies Up $1.8 Million

Stanford University has come through on matching funds for state-financed stem cell research, coughing up $1.8 million after California's stem cell agency applied a little financial pressure.

The matter involves Stanford  researcher Judith Shizuru, who is conducting a clinical trial that is aimed at treating the "bubble boy" genetic affliction without requiring high-risk chemotherapy or radiation. Her work has implications for reducing the need for the dangerous treatments in other diseases. 

In 2012, Shizuru was awarded $19 million by the stem cell agency for her trial. Late last year, she applied for $6 million more. 

Sharp questions arose, however, from the governing board of the $3 billion California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known. It was the first time that the CIRM board had publicly rebuked a grantee and an institution on co-funding.

The board noted that the $19 million grant required $1.8 million in matching funds that had not been forthcoming.  

Directors pointed out that Stanford was well-endowed and should not be hard-pressed to provide cash to help develop a potentially "transformative" product that would eliminate the toxic impact of chemotherapy for a number of diseases. 

That was back in January. The CIRM directors set a May 1 deadline for seeing the cash. 

Last month the agency, which expects to run out of funds for new awards later this year, received a letter from Robert Harrington, the chairman of the Department of Medicine at Stanford. 

The letter said, 
"This letter is to confirm that the Department of Medicine will provide funding in the amount of $1,784,953 to meet the required co-funding for Operational Milestones #3 and #4 disbursements as referenced in your email dated 3/14/19 for the above referenced grant. These funds are available immediately to Dr. Shizuru for the study."
The flap also led to an inside look at how research funding works at the stem cell agency in a case involving a rare affliction, delays in clinical trials and the financial pressures now facing CIRM. 

Over its 14-year life, the agency has awarded $2.6 billion. Stanford is the No. 1 recipient with $383 million. Its total far exceeds the No. 2 recipient, UCLA, which has chalked up $284 million. 

Nine out of the top 10 recipient institutions, including Stanford and UCLA, have members on the CIRM governing board. They are not allowed to vote on awards to their institutions, but they establish the research award programs and their rules. 

Sphere: Related Content

Sunday, May 19, 2019

Legislation to Regulate 'Snake Oil' Stem Cell Clinics in California Hits Fiscal Speed Bump

California legislation to crack down on "snake oil" stem cell clinics has stalled after a cost of $100,000 was assigned to the measure, which is backed by the state's stem cell research program. 

The bill was sent last week to a "suspense" file, which holds fiscal legislation while lawmakers juggle priorities in the Golden State's yet-to-be-approved, $213 billion budget.

If all goes well, the measure could clear the Assembly Appropriations Committee in the next month or so and be sent to the floor of the Assembly. It would then go to the Senate for more committee hearings and a Senate floor vote.

No public opposition has yet surfaced to the measure (AB617), authored by Assemblyman Kevin Mullen, D-San Mateo. It would not take effect any earlier than 2020.

The bill would require California's Medical Board to move on the clinics, which have proliferated across the country in recent years. California has more than 100, according to the latest estimates. 

The clinics charge thousands of dollars for treatments using substances that they describe as stem cells. However, in virtually all cases, the treatments have not been tested scientifically. Some have led to serious injuries. 

The state Medical board would be required to create a stem cell advisory group by February 2020 that would make recommendations by July 2020 for regulation of the dubious clinics.

The new group would have nine members. Three would be appointed by the California stem cell agency, three by state Medical Board, two by the state osteopathic medical board and one by the state nursing board. 

The group could also recommend emergency regulations that could be adopted by the state Medical Board with a 90 day notice. 

Last week, a committee of directors of the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known, endorsed the bill. The full board is expected to ratify that endorsement on Thursday. An analysis prepared by the legislative staff already lists CIRM as a supporter.  Sphere: Related Content

Wednesday, May 15, 2019

Exploring California's Stem Cell Future: A Start Today on the 16th Floor in Oakland

OAKLAND, Ca. -- "Phantom equity," the affordability of $1 million cures, maximizing returns to the state and conflicts of interest -- all that and more was on the table today at the $3 billion California stem cell agency.

The occasion was a discussion of "ideas for enhancement" by some of the directors of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. 

This was more than simply kicking around possibilities. The meeting was called because the agency expects to run out of money for new awards later this year.

Its hopes for financial survival rest largely on a possible $5 billion bond initiative on the November 2020 ballot. Writing an initiative also means an opportunity to improve agency operations. To pass an initiative, California voters would also need to be inspired by the work the agency has done and how it will do even better in the future. 

So the governing board put together today's session to hash over ideas, starting with a 10 page list of initial thoughts. No decisions were reached, however. The discussion will carry on next Thursday at a meeting of the full board, which will also be held in CIRM's 16th floor offices here. But internally the agency plans to begin to explore some of the areas in more depth.

The implications of changes could have a major impact not only on California researchers but biotech businesses looking for possible products that could generate profits, preferably large ones.

One area of concern by CIRM directors was maximizing the return to the state for its $6 billion investment, which includes the interest on the $3 billion borrowed for the CIRM program. One of several suggestions included the use of "phantom equity," a loose term that implies having an investment but not ownership in a company that is tied to the value of its shares.  (CIRM is barred from actually owning stock.)

At the same time, directors also said they did not want to raise financial obstacles that would stop biotech firms from turning CIRM research into cures.

CIRM Director Jeff Sheehy, who has served on the board since its inception in 2004, brought up affordability and access suggestions by the man who led the 2004 campaign, Robert Klein. 

In a recent interview with the California Stem Cell Report, Klein said he was interested in seeing more stringent requirements to assure that state-backed therapies are available to all.  Esimates of costs of some legitimate stem cell treatments have ranged up to $1 million or more. 

Another subject of some discussion was the matter of perceived conflicts of interest involving the current structure of the CIRM board. About 90 percent of the awards made by CIRM have gone to institutions with links to members of the board. While some board members acknowledged the importance of perceptions, they said no conflicts of interest have actually occurred. 

A proposed initiative will have to be filed late this year in order to qualify for the November 2020 ballot. In the interview with Klein, he said he was looking at filing around the middle of September. Sphere: Related Content

California Stem Cell Crossroads: A Researcher Muses About the Golden State's Research Program

OAKLAND, Ca. -- A California stem cell researcher weighed in this morning on the future of the state's $3 billion stem cell agency, a topic that its leaders will delve into here today for two hours.

Paul Knoepfler, writing on his blog, said,

"We are at a crossroads for stem cell research in California as the original form of the California Stem Cell Agency (CIRM)winds down. Will CIRM get a new infusion of billions in state funding via a ballot initiative a la Proposition 71 from more than a decade ago? Will CIRM instead find a different source, probably much smaller, of research funding such as via philanthropy? Could CIRM cease to exist within a few years?"
Knoepfler is a longtime supporter of the agency. He wrote,
"CIRM has such strong clinical momentum right now, I hope it continues robustly for another 10 years."
The agency, however, expects to run out of cash for new awards late this year. It is pinning its hopes on a possible $5 billion ballot measure in November 2020. 

Knoepfler speculated about other sources of fund, including some from the legislature and possible private support. He wrote,

"While California has been very successful in getting NIH research funding for stem cell research by individual labs, the feds don’t fund state-wide efforts. The stem cell-related biotech industry in California is robust and should there be a few blockbuster successes from ongoing clinical trials here, which I expect will occur in the coming decade, this will likely draw in much more funding in that area."
Knoepfler also discussed the impact of "snake oil" stem cell clinics on the future of the agency. 
"In my view," Knoepfler said, "a newly funded CIRM is also going to have to deal directly with the unproven stem cell clinic problem in our state. With more than 100 such clinics here, Californians are at risk but so is stem cell research funding from these rogue efforts. The new bill on stem cell clinics could be the ticket, should it become law."
The California Stem Cell Report will be covering live this morning's hearing on the agency's and will file reports as warranted.
Sphere: Related Content

Tuesday, May 14, 2019

California Eyes "Makeover" -- Sort Of -- For Its $3 Billion Stem Cell Research Program

One might say that the $3 billion California stem cell agency is going into the botanical business this week. That's because tomorrow it will be "let-a-thousand-flowers-bloom" time at the agency.

For a start, the agency has posted 10 pages of "ideas for enhancement" of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. And it wants even more suggestions. 

The initial ideas include barring persons from sitting on the CIRM governing board who have connections to institutions that receive CIRM funds. Others involve creation of a mechanism that would permit CIRM to act more like a venture capitalist.

Still others include topics such as the current 6 percent administrative budget cap, prohibitions on CIRM owning stock, restructuring the research application review process and the expense of renting office space, which drove the agency from its previous headquarters in San Francisco. 

The wide-ranging discussion, scheduled for two hours, is driven by the fact that CIRM will run out of money for new awards this year, possibly as early as September. The agency is hoping for passage of a new ballot initiative in the fall of 2020 that will provide it with $5 billion more.

A fresh, citizen-written initiative means an opportunity to recast CIRM from version 2.0 into version 3.0 with improved abilities to develop stem cell therapies, to deal with the changing economics of medicine and to fix things that weren't quite right in Proposition 71, the 2004 measure that created the agency. 
"CIRM has a unique perspective to offer regarding possible enhancements to Proposition 71 that might further CIRM’s mission more efficiently and improve its operations," the agency said in a document prepared for tomorrow's meeting. 
The agency also that said that the ideas offered in advance "are intended to spark discussion and are not intended to limit discussion topics."

Robert Klein, who led the 2004 campaign and was the agency's first chairman, has indicated that he is once again ready to move forward on a new campaign. He offered other ideas involving a new initiative in an interview recently with the California Stem Cell Report. 

As for the "thousand flowers" business, that is a phrase that is often attributed to Chairman Mao who reportedly actually said 100 -- not 1,000. His remark came in 1957 and was ripe with financial and political implications. So it is once again this week in connection with ideas for what could be CIRM 3.0. 

Tomorrow's meeting will be carried live and interactive on the Internet. Members of the public will be able to listen in and comment. Instructions are on the agenda. 

The meeting will be based in Oakland with teleconference locations for CIRM board members, which the public can share, in Riverside, South San Francisco, San Diego, Napa, San Francisco, Irvine, La Jolla and Rancho Cordova. Specific addresses can be found also on the meeting agenda. 
Sphere: Related Content

Monday, May 13, 2019

Stem Cell Treatments on the Installment Plan, Cash for Patient Families, State Grants for Stem Cell Training? Perhaps Part of a New, $5 Billion California Ballot Measure

In November 2017, Robert Klein appeared before directors of the California stem cell agency to discuss a new bond initiative for the agency. He had more to say last month. See story below.
 California Stem Cell Report photo. 

PALO ALTO, Ca. -- The man regarded as the father of the $3 billion California stem cell agency is thinking about changes in the program to help win voter approval of another $5 billion for the research program. 

They include a stronger requirement to make state-backed, stem cell therapies more affordable and accessible and to provide more cash for creating a greater stem cell work force in the Golden State.

Robert Klein, the real estate investment banker who led the formulation of the 10,000-word ballot initiative that created the stem cell agency, recently told the California Stem Cell Report,
"The original initiative stated really a commitment to the idea of access....
"It's my intention to actually put a directive in for funding the infrastructure to work on access, to work with insurance companies, to work with Covered California to work with Medicare, to make sure that there's access, to make certain that public hospitals and public clinics in California get discounted prices."
Klein discussed some of his thinking for another ballot initiative in 2020 during a 70-minute interview last month in the offices of Klein Financial Corp. 

Also housed in his building is the office of Americans for Cures, a nonprofit stem cell advocacy group. Klein, who founded the group and is its chairman, said he is consulting with its scientific advisory group to develop possible changes in CIRM. 

The co-chairs of the group are Irv Weissman, director of Stanford's Institute of Stem Cell Biology and Regenerative Medicine, and Larry Goldstein, director of the UC San Diego stem cell program.

Klein's thoughts on affordability and access were expansive. 
"We need to also address the issue of how do patients get treatment. They need transportation to get there right? They need housing for their family member who's going to take care of them."
As for possible costs of $1 million for a single treatment, Klein called for fresh thinking in terms of insurance coverage and financing. He notes that stem cell therapies can result in a total cure, eliminating the need for recurring, expensive treatments. He said,
"These curative therapies -- we need to work on a new business model for biotech where instead of paying $500,000 upfront or something where companies price it."
As for payments by insurance companies for treatments, Klein said,  
As long as you're in remission from cancer, they pay for it, but they pay for it over a period of five years or 10 years. Right? It’s pay for performance and medicine, which is important to access. Right?"
As for training medical professionals to handle what Klein hopes will be a burgeoning industry, he said,
"I'm looking at a directive section that deals with specifically broadening the doctors who are specifically educated. The only way you can do that is to buy a part of the residency time. Otherwise they don't have time to do anything other than deal with current patients and current methodologies, which are radically insufficient as compared to the new cellular therapies. 
"In terms of having the workforce that can implement these new therapies.... You need individuals that are trained in high speed cell sorters. You need individuals that understand the handling of cellular therapies, keeping frozen transportation, the procedures within the operating rooms on delivery, specific structures that have been created to be able to precisely insert them at the right place."
Klein, who was the first chairman of CIRM, has more ideas that may well be aired at a CIRM meeting Wednesday to discuss a possible $5 billion bond initiative to rescue the agency from financial death.

The bond funds that were authorized in 2004 are expected to run out for research awards as early as September.

Wednesday's meeting involves the science and governance committees of the agency's 29-member board. The session will be aired live over the Internet. The public can participate as well. Instructions are available on the agenda. 

The meeting will be based in Oakland with teleconference locations for CIRM board members, which the public can share, in Riverside, South San Francisco, San Diego, Napa, San Francisco, Irvine, La Jolla and Rancho Cordova. Specific addresses can be found also on the meeting agenda. 
Sphere: Related Content

Sunday, May 12, 2019

$15.6 Million Operating Budget Slated for California's Stem Cell Agency

California's 14-year-old stem cell research program is set this week to approve a $15.6 million operating budget as its funding is drawing to a close.

Cash for new research awards is expected to run out later this year. But the agency, known formally as the California Institute for Regenerative Medicine (CIRM). still needs to administer multi-year awards for the next few years. It has set aside money to wind down those existing programs.

The proposed operating budget for the 12 months beginning July 1 totals $15.6 million, up from an expected $14.9 million in spending in the 2018-19 fiscal year. The budget is slated to taper down to $1.8 million in 2023-24.

For the new fiscal year, employee salary and benefits consume most of the budget, accounting for $11.4 million, up slightly from $11.3 million this year.  The most recent body count at CIRM shows about 45 employees. 

The second largest spending category for next year is "external services," largely outside contracting. That area totals $1.5 million, up from $1.1 million for the current year.

The 2019-20 budget provides $838,000 for grant application reviews, meetings and workshops. That is up from $794,000 this year.

The $838,000 appears to assume the success of a private, $220 million fundraising drive that is now underway. It would allow the agency to continue its awards in a significant fashion. However, no public announcement of any major fund donations has yet been forthcoming.

The budget comes before the Finance Committee of the agency's governing board on Wednesday. The committee routinely approves the annual operating budget with few, if any, major major changes. The spending plan will then go to the full board next month for ratification.

More details of the budget can be found on the agenda for the meeting. The session will be aired over the Internet. The public can listen in and ask questions online as well. Instructions for participating can be found on the agenda. Sphere: Related Content