Friday, December 09, 2016

California's Stem Cell Research Spending Up for Review Next Week

If you are interested in how the state of California is going to spend its final $800 million or so on stem cell research, you should catch a key meeting next Tuesday in Oakland, which also can be heard online.

The session involves the 29-member, governing board of the $3 billion California Institute for Regenerative Medicine (CIRM), as the state stem cell agency is formally known.

The agency was created by California voters in 2004 with voters' expectation that stem cell therapies were all but just around the corner. So far, no therapies have been developed by the agency that are available for widespread use.

Nonetheless, the agency, which runs out of money in 2020, is pushing hard. It has more than 20 clinical trials underway, which is the last step in bringing a therapy to market. However, those trials can take years with no guarantee that a proposed product will emerge successfully.

On tap on Tuesday will be a look at the agency's research plans for the next three years with a review of how it has performed so far in 2016. The agency's proposal for research spending in 2017 is likely to have a significant impact on the hundreds of stem cell researchers in California. The proposal is not yet available online, however, with only two business days left before the meeting.

CIRM is also hinting that there will be some surprises at the meeting, but it is unlikely that a product announcement will be forthcoming.

Additionally on tap are applications for a total of $14.9 million for two early-stage clinical trials. One is for $6.7 million (CLIN2-09439) to test using stem cells and T cells to eliminate the life-long need for immunosuppresive drugs by kidney transplant recipients. The other (CLIN2-09698) is for $8.3 million for a mid-stage trial (2b) for a therapy for retinitis pigmentosa.

The applications were approved for funding by the agency's grant reviewers, who meet behind closed doors and do not publicly disclose their economic or professional interests. Ratification of the reviewer decisions is a formality for the agency. although the names of the recipients are not generally disclosed prior to board action.

The board is expected to re-elect Jonathan Thomas as chairman of the panel. Thomas was elected to the position in 2011. He has sent a  two-page letter to the board detailing his work during the last five years. Also scheduled to be re-elected is Art Torres as vice chairman.

Thomas receives $400,000 annually for his "80 percent effort" in the part-time position. Torres, a former state lawmaker, receives $225,000, also for an "80 percent effort."

Instructions for listening to the meeting online can be found on the agenda. In addition to main meeting site in Oakland, public telephonic locations exist in San Diego and La Jolla. Specific addresses can be found on the agenda. Sphere: Related Content

Voting Underway for Stem Cell Person of 2016

The latest tally of voting for the stem cell person of the year shows that patient advocate Ted Harada, who died in October, is leading with 47 percent of the vote with the nearest contender at 11 percent.

However, only one vote counts since this is not a democratic election. The sole individual choosing the stem cell person of the year is Paul Knoepfler, a stem cell scientist at UC Davis.

Knoepfler originated the award a few years back and personally puts up $2,000 in prize money. He has compiled a list of 20 possible honorees, ranging from patient advocates to researchers. Voting is advisory only. Deadline for the ballot is 11:59 p.m. Dec. 15.

Knoepfler wrote on his blog last month about Harada, who was an ALS patient advocate. Knoepfler said,
"Ted approached all that life threw at him, whether it was ALS or a brain tumor, with a characteristic passion, sense of humor and classiness. I don’t recall anyone dealing with adversity as well as Ted did and I never heard him say anything like, 'why me.' Instead there was always kindness and grace."
Distant second to Harada this morning was Randy Mills, president of the $3 billion California stem cell agency. Sphere: Related Content

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.  Sphere: Related Content

Monday, December 05, 2016

California Approves $15 Million for Stem Cell Research Ranging from Arthritis to Alzheimer's, But Not Without a "Hair-Cut"

Highlights
Snafu in November
Ankasa whacked
Across-the-board cuts rejected

After a hiccup last month, the California stem cell agency today coughed up $15 million for a quartet of researchers looking into Alzheimer's disease, cartilage repair, arthritis and sickle cell disease, but not before lopping off a big chunk of one proposal.

Action by the governing board of the $3 billion agency came after a snafu at its Nov. 17 meeting. The proposals, all previously approved by the agency's reviewers, hit a roadblock when the budgeted cash was not enough to fund all four. The session last month also stalled as a result of quorum problems and research priorities.

Normally applications approved by reviewers during their closed-door sessions slip through the later public meetings of the agency board with no discussion. But this time around, reviewers approved the four applications, but gave one of the researchers a sizable, financial "hair cut."

Despite concerns about fairness and imposing new conditions on applicants, the board stripped $1.6 million from a $3.7 million application to develop a stem cell therapy for osteoarthritis by Ankasa Regenerative Therapeutics of La Jolla.

The action came after the lead researcher, Jill Helms of Stanford, said her company would make up the shortfall.  She made the comment after being told she could re-submit the application, which was the last to come up for a vote. She indicated that she would prefer to pursue that route rather attempting a problematic and "onerous" re-application with a new set of reviewers early next year.

The other three applications were approved with full funding after a motion to cut all four by about 10 percent failed on a 1-9 vote with one board member abstaining. Board members used the term "hair cut" to describe the reductions, which the applicants would have to make up.

Helms' application was the only one in this round from a business. The others came from non-profit institutions.

Randy Mills, president of the California Institute of Regenerative Medicine or CIRM as the stem cell agency is formally known, opposed the 10 percent cuts on all four awards. He said he and his staff did not want to see programs cut "on the fly." He said applicants were not told that they would have to come up with matching funds when they originally applied.

The other three researchers winning awards are Yadong Huang of the Gladstone Institutes, $6 million for Alzheimer's;  Mark Walters of Children's Hospital, Oakland, $4.5 million for sickle cell, and Denis Evseenko of USC, $2.5 million for cartilage repair.

All the awards went to researchers or institutions that have links to persons who sit on the 29-member board. Overall, about 90 percent of the money awarded by CIRM since 2004 has gone to institutions with ties to past or present board members. While board members set rules for funding and the scope of awards, they are not allowed, however, to vote on specific applications from institutions with which they are connected.  

Summaries of reviewers comments, scores and more can be found on this document from CIRM, which is based in Oakland.  Here is a link to the CIRM press release. Sphere: Related Content

Sunday, November 27, 2016

Dubious Stem Cell Therapies Attract Coverage in California's Capital

The leading newspaper in California's state capital this weekend published a lengthy piece exploring the world of unproven stem cell therapies, including one being offered in its own backyard. 

Reporter Claudia Buck of The Sacramento Bee wrote,
"For long-suffering patients...stem cells offer tantalizing hope. In the last few years, more than 570 stem cell clinics have popped up nationwide, advertising treatment for a range of maladies, from autism and Alzheimer’s to neuropathy and Parkinson’s disease, according to a recent UC Davis study. About 113 of those are operating in California. 
"But do they really work? According to most stem cell experts and the federal government, there’s no way to know yet."
Buck quoted Kevin McCormack, spokesman for the California stem cell agency, as saying,
“It’s quite clear that these people are offering treatments that haven’t been tested in clinical trials. It’s a little concerning,”
Buck wrote,
"'My view is that it’s a giant human experiment that doesn’t have FDA approval,' said Paul Knoepfler, a UC Davis stem cell expert, who co-authored the study identifying the 570 clinics. 'I don’t know how much patients are aware of how uncertain the benefits and risks are. As a scientist, it’s worrisome.'"
Knoepfler, who publishes a blog on stem cell matters, has written in the past about advertising in The Bee by a stem cell business called Nervana. The firm was mentioned in Buck's article, which, however, did not note that Nervana has taken out full page advertisements in the past in The Bee.

Knoepfler's most recent piece on Oct. 21 also noted that the firm had taken out a full page ad in the San Diego Union Tribune as well.  The UC Davis researcher said at the time,
"I don’t believe there is a solid, medical or scientific basis for what they are selling."
Sphere: Related Content

Stem Cell Suspense to End Dec. 5 for Four California Researchers

Four California stem cell researchers could well receive a total of $16.6 million in awards next week from the Golden State's 12-year-old stem cell program.

The researchers were caught in a bit of a snafu earlier in November when the $3 billion agency appeared to come up $1.6 million short. The story about the snag also involved quorums, priorities and fiscal discipline, more than is necessary to discuss right here in this item. But you can read all about it here. 

The two researchers identified so far are Yadong Huang of the Gladstone Institutes, Jill Helms of Stanford and Ankasa Regenerative Therapeutics of La Jolla.  The identities of the others are being withheld by the stem cell agency.

Whether all four will receive funding will be determined on Monday Dec. 5 when the stem cell agency's board will hold a telephonic meeting out of its base in Oakland. The agenda gave no indication of how the agency plans to overcome the difficulties that stymied it on Nov. 17.

The public can attend the meeting at the agency's headquarters in Oakland or from telephonic locations in Napa, Beverly Hills, Elk Grove, Los Gatos, Irvine, Sacramento and two each in South San Francisco and San Diego.

The session is also expected to be audiocast on the Internet. More information and addresses can be found on the agenda. Sphere: Related Content

Monday, November 21, 2016

Quartet of Researchers Snagged in Budgeting, Parliamentary Web at $3 Billion California Stem Cell Agency

Highlights
Fiscal discipline at CIRM
The 10 percent solution
A quorum shortage pops up

Four California scientists who are ready to kick off highly rated projects to treat everything from Alzheimer's to rotting jaw bones became tangled last week in a financial and procedural briar patch involving the directors of the $3 billion California stem cell agency.

The basic problem, however, was simple. Money.

The agency had budgeted only $15 million for this latest round of awards last Thursday. But the four applications -- already approved by the agency's reviewers -- totalled $16.6 million. Typically, the agency's full board rubber stamps in public the decisions of its reviewers, who act behind closed doors without disclosing their economic or professional interests. The board has reversed approvals by reviewers on only four occasions out of hundreds of awards over the past 12 years, according to the agency.

Last week, the chairman of the agency, Jonathan Thomas, began the public discussion by declaring that the board should go through the applications one by one and vote on them. When the money ran out, that would finish action on funding for November.

Fiscal discipline was cited as the main reason for such a course.

Steve Juelsgaard 
Wait a minute, said Steve Juelsgaard, a member of the board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. He asked for the amount of
funding already being provided for the afflictions targeted by the proposals. If research in a particular area was already heavily supported, perhaps approving another award in that area was not necessary, Juelsgaard reasoned. However, the agency staff could not provide those figures at the time of the meeting.

A lengthy discussion followed involving several scenarios. One would have cut each award by 10 percent but approve all four.  But that could mean that the proposals would be altered from the versions that were approved by the reviewers. (However, the board is not legally required to accept what the reviewers decide.  Under the terms of the ballot initiative that created the agency, the board has the final say, which is part of the justification for not publicly disclosing the economic interests of reviewers.)

Another proposal would have simply increased the funding for the round. That could not be acted on because it required 10 days advance public notice.

The board ultimately approved a Juelsgaard motion to slice roughly 10 percent from each award with the condition that applicants come up with matching funds to bring the total to the level approved by reviewers.

Yadong Huang, Gladstone photo
One applicant, Yadong Huang from the Gladstone Institutes, said, however, that non-profit research organizations were already hard-pressed and could not necessarily come up additional cash. His $5.9 million application (TRAN1-09394) was top-ranked by reviewers and targeted Alzheimer's.

Jill Helms, Linked In photo
Another applicant, Jill Helms, chief scientific officer of Ankasa Regenerative Therapeutics, Inc., of La Jolla, spoke on behalf of the company's application (TRAN1-09270) to target osteonecrosis, an affliction that "causes jaw bones to rot and thigh bones to snap." She urged the board to give priority to applications that already had co-funding. Her $3.7 million application contained a 20 percent co-funding component.

Helms, who is also a Stanford University medical school professor, unsuccessfully asked the CIRM board in 2015 to overturn a negative recommendation by reviewers.

During the two-hour telephonic meeting, the board did approve conditionally two awards under the matching-fund requirement. They were for the Alzheimers proposal and one dealing with sickle cell disease (TRAN1-09292).  The identity of the chief scientist on the sickle cell proposal was not disclosed by the agency under its longstanding policy and tradition within the research field.

Jeff Sheehy, Science photo
The board failed to complete action on the two others because it  lost the quorum that is required to do business legally. That came after a motion by board member Jeff Sheehy to reject one of the four applications failed on a 3-8 vote. Sheehy said another related proposal was already being funded by the agency and that the time to translate the research into a therapy  "would be enormous." The $2.5 million application  (TRAN1-09288) up for consideration last week involved cartilage repair.

Thursday's meeting was being conducted telephonically. After Sheehy lost his motion, he did not respond to telephonic queries from the board. The meeting was nearing its scheduled end at noon. Other board members also failed to respond, and the meeting was adjourned minutes later.

Juelsgaard and some other members said it was important for board members to stick around for the full meetings. Juelsgaard said,
"For gosh sakes, this is something that you signed up to do."
Termination of CIRM board business because of quorum problems regularly occurred some years ago. (See herehere and here.) But since Thomas has been chairman the issue has rarely popped up.

Thomas indicated the board would try to schedule a special telephonic meeting to deal with the four applications. It also has a face-to-face meeting scheduled for Dec. 13 in Oakland. Both meetings legally require 10 days advance notice.

The review summaries on the applications are consolidated in this CIRM document along with their scores and more information.
Sphere: Related Content

Friday, November 18, 2016

$3 Billion California Stem Cell Agency Now Involved in 23 Clinical Trials

The California stem cell agency yesterday pumped $38 million into three clinical trials with the hope that it will lead to therapies for colorectal cancer, "bubble boy" syndrome and a form of high blood pressure.

The action brought to 23 the number of current clinical trials in which the $3 billion agency is participating via funding in whole or in part, according to information on its Web site. It is hoping that one of those trials will produce its first widely available stem cell therapy.

The trials deal with afflictions ranging from HIV and Huntington's Disease to blindness and skin cancer.

The agency's cash for new awards will run out in about three years. The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, has been in business since 2004 when voters created the agency through a ballot initiative.

Yesterday's funding was expected and largely reported earlier this week by the California Stem Cell Report (see here and here). Yesterday, the agency's board simply ratified -- in a matter of minutes -- decisions in October by its grant review group, which makes the de facto decisions on awards behind closed doors and without publicly disclosing the economic and professional interests of reviewers.

Here is a link to the press release from the agency and a link to its blog item. Here are links to the review summaries of each proposal: colorectal, bubble boy and blood pressure.

The summaries were the only information that the board had in approving the three grants.

During its telephonic meeting yesterday, the board also became entangled in a budgeting and priorities debate involving four translational research proposals, all of which the agency now says are hanging fire. An effort is being made to schedule a special board meeting soon to deal with the issue, but it requires 10-days advance public notice. The California Stem Cell Report will have a full report later on the situation. Sphere: Related Content

Wednesday, November 16, 2016

Correction

The $20 million "bubble boy" item on Nov. 16, 2016, incorrectly reported that co-funding on the award totalled $8.9 million. The correct figure is $18.2 million. Sphere: Related Content