Monday, December 23, 2013

Golden State Milestone: Stem Cell Agency Announces its First Phase Two Clinical Trial

The California stem cell agency today scored a first with the announcement that one of its projects – a heart disease therapy -- has now advanced to a phase two clinical trial after successfully completing a phase one safety trial.

In a press release, Jonathan Thomas, chairman of the $3 billion research effort, hailed the Allstar trial by Capricor Therapeutics, Inc., of Beverly Hills. He declared,
"This is a highly significant announcement for us at CIRM as it's the first time we have funded a therapy into a Phase 2 clinical trial.
"Heart disease claims around 600,000 American lives every year, so clearly there is a huge need for new approaches and more effective therapies. We are hopeful this is the first of many treatments to turn the tide against this disease, and that this will be the first of many projects we are funding to get to a Phase 2 trial."
The adult stem cell therapy “uses unrelated donor-derived stem cells, called cardiosphere-derived cells, that are then infused into a patient’s artery with the aim of reducing scarring caused by heart attacks,” CIRM said.

Capricor Therapeutics, which has its roots in Cedars-Sinai in Los Angeles, describes itself as as “a diversified heart failure biotechnology company.” It came into being last summer as the result of the merger of Capricor and Niles Therapeutics. The company has benefited from about $27 million in support from the stem cell agency, including $6.9 million for early work by one of its founders Eduardo Marbán at Cedars-Sinai.

The company's stock price closed at $2.48 today, down 17 cents. Its 52-week range is from $2.48 to $2.65.

The firm's CEO, Linda Marbán, said the move into phase two was a “giant leap” for the firm and the heart therapy field.

The executive chairman of the firm is Frank Litvack, a heart surgeon who was the only other candidate for chairmanship of the stem cell agency when Thomas won the job in 2011.

The announcement today is a milestone for the stem cell agency, which is aggressively seeking results that will help to generate financial support after 2017, when its current state funding runs out.

The CIRM news release said,
“The next phase will involve an estimated 300 patients who have had heart attacks, and they will be evaluated in a double-blind, randomized, placebo-controlled trial.  This will be further broken down into two groups: one will include patients 30-90 days post attack, the second will be 91 days to one year after the incident.”
The phase two trial is to determine the therapy's effectiveness and further study its safety. If successful, it can move into a phase three trial to confirm its effectiveness, monitor side effects, compare it to common treatments and collect information that will allow the drug or treatment to be used safely.

Here is a CIRM video in which Eduardo Marbán discusses some of the issues involved in the therapy. Here is a link to a 2011 presentation to the CIRM governing board about the early research – a presentation that generated some excitement on the board.

Thursday, December 19, 2013

Cost-saving Stem Cell Cuts Puzzle California Scientists

Jeanne Loring, the head of the Scripps stem cell program, reflected last week on the move by the $3 billion California stem cell agency to scuttle a program that she and other scientists believe is critical to the research effort.

Writing on, she discussed the effort to save the “shared” labs program at 17 institutions around the state. Continuation of the program was axed by directors last week in a cost-saving move. The agency is down to its last $600 million or so, and the governing board was feeling the pressure last week of having to deal with competing interests.

The board has 29 members, but only six in attendance could vote on the program because of the board's longstanding conflict-of-interest problems. Four approved the recommendation by CIRM staff and its Scientific Advisory Board to halt the lab program. That was all it took – the votes of four directors out of 29.

Loring and 11 other scientists attended last week's board meeting in an unsuccessful effort to the save the labs. She later wrote,
“We are back home now, but still puzzled about why CIRM would destroy one of its most successful programs.  The irony is that the board would like to continue to support training.  The training is done in the shared labs.  There are no more courses without the shared labs. 
“We aren’t giving up; we’re just taking some time to think.  Meanwhile, the positive side:  we did an amazing thing:  we are normally very competitive with each other, but on Wednesday we came together from all over California to support a single cause that is dear to our hearts.  That will remain a high point for me, even though I also feel a profound sense of loss.”

Sunday, December 15, 2013

Bad Link Repaired

A bad link in the "$61 Million" item has been fixed, thanks to an anonymous reader who called it to our attention. The link is to the list of all the applications in that round.

Thursday, December 12, 2013

Wish List on Criteria for New President of $3 Billion California Stem Cell Agency

Directors of the $3 billion California stem cell agency today moved closer to possibly selecting a non-scientist as the new president of the nine-year-old research enterprise.

On a unanimous voice vote with no discussion, they approved criteria that did not make it mandatory that a scientist fill the position being vacated by Alan Trounson, a noted IVF researcher who is returning to Australia.

Instead, the governing board decided that candidates should have “experience with and personal commitment to medical and scientific research including familiarity with stem cell research.”

In terms of academic credentials, the criteria state that the new president have either or both an M.D. or Ph.D. degree or “equivalent industry experience or similar body of knowledge developed in professional roles.”

The criteria that was approved was not available to the public today on the CIRM Web site prior to board action. It had been altered on Tuesday night from an earlier version that was posted on the Web site.

Here is the text of what appear to be the only alterations (all additions) in the new criteria.
  • "Comfort working in the public sector, including an awareness of the need to comply with the laws that govern them, such as transparency, conflict of interest, and public accountability laws." 
  • "Experience dealing with, and commitment to, diversity and gender equality in the workplace."
It is ironic that the addition of the need for awareness of the need for transparency was added but the document was not available to the public prior to board approval.

CIRM Chairman Jonathan Thomas said yesterday that the agency is on verge of signing a contract with a search firm, Korn Ferry, to help in finding the new president. Directors have been emphatic about the need for speed in finding the new president.

Trounson has agreed to stay on for an unspecified period, but he could leave at any point. If he leaves before a new president is chosen, presumably Ellen Feigal, senior vice president at the agency, would pick up his duties temporarily, as she did earlier this year for three months.

She is also likely to be a candidate for the top spot, and if she is not selected after serving again on an interim basis, she might well decide to look for opportunities elsewhere.

California Stem Cell Agency Board Quits for the Day

The governing board of the California stem cell agency adjourned its two-day meeting in Los Angeles at 2:40 p.m. PST today. We will have an item coming up shortly on approval of the criteria for selection of the new president. The board approved a revised criteria that was not available to the public prior to board action. It is unclear whether any of the changes were substantive.

Stem Cell Agency Identifies Grant Recipients

The California stem cell agency has posted its press release on this week's meeting of its governing board. The press release includes the identities of the grant recipients. All of the recipients are affiliated with institutions represented on the CIRM board. Here is a link to the press release.

$61 Million Approved for Stem Cell Clinical Trials

Directors of the California stem cell agency today handed out $61 million in its ambitious disease team program that is designed to push stem cell research into clinical trials.

Action on the six awards followed yesterday's move by the board to set aside $200 million to beef up its efforts for stage one and two clinical trials. The agency is focusing ever more intently on commercializing its research.

The $3 billion program is expected to run out of cash for new grants in 2017. Its efforts to develop a new stream of funding depend a great deal on showing results that resonate with the public and possible funding sources.

This grant round was originally budgeted for $100 million, but directors today were loath to approve additional funding. They noted that the agency is now living an era of financial limits.

Today's awards are aimed at cancer (three grants), sickle cell anemia, macular degeneration and severe airway obstruction. Size of the awards ranges from $20 million to $4.4 million. Terms range up to four years.

The agency did not immediately release the names of the winners, in keeping with its policy of secrecy until after the board votes. However, the names of the applicants could be determined by a knowledgeable person searching articles in the various fields. A CIRM news release is expected shortly with the names of the researchers and institutions. (Here is a link to the press release. All of the recipients are employed at institutions represented on the governing board.)

Thirteen applications, including one from a business, were considered by reviewers.

Here are links to the information provided by CIRM staff on the grant applications: summaries of the grant reviewsstaff justification for funding tier two applicationsPower Point presentations to be made to directors on the applications, including the range of scores on individual applications. However, the range of scores on the rejected applications was not provided.

Stem Cell Board Nixes $13.4 Million to Help Cure Cartilage Disorders

Directors of the California stem cell agency today rejected a $13.4 million proposal that would have been the first disease team research that it has backed that would deal with cartilage disorders.

The project would have built on earlier research funded by CIRM. The agency staff cited work at UC Davis  by Kyriacos Athanasiou and at Scripps by Peter Schultz. Funding of the application was supported by the CIRM staff, which did not identify the applicant.

However,  the governing board, however, went along, on a 10-1 vote, with arguments by Director Jeff Sheehy and others that the agency had finite resources and had to make difficult decisions. He noted that many alternative treatments exist for cartilage problems. Sheehy said,
"We are here to make progress on serious diseases and conditions."  
Former Chairman Robert Klein, in an unusual appearance as a member of the public, urged approval of the grant. He said that it would address conditions "very prevalent in the older population."  Klein told the board that the research would be a "very important milestone that will help you get more money in the future."

While Klein did not mention it, older persons participate in elections to a much greater degree than younger persons. They would be an important group in winning support for another bond issue to fund CIRM, which runs out of cash for new grants in 2017.

$13.9 Million Approved for Sickle Cell Research at UCLA

The California stem cell agency today effectively approved a $13.9 million grant involving UCLA to develop stem cell gene therapy for sickle cell anemia.

CIRM directors acted on a staff recommendation to place the grant in a category that is automatically approved by the board.

(Here is a press release that was issued later by UCLA. The investigator is Donald Kohn.)

CIRM Directors Reject $16 Million Eye Research Application

Directors of the California stem cell agency today rejected a $16 million application from Cedars-Sinai for research involving retinitis pigmentosa.

The action came on a split vote despite a personal appearance by the principal investigator, Kent Small, and a patient advocate.

CIRM staff had recommended rejecting the grant because the agency is funding a number of other eye research projects. The vote was 7-4 to approve the staff recommendation.

Small and Clive Svendsen, also of Cedars-Sinai, noted that other diseases have many grants from CIRM and have not been subjected to the same sort of objection. They noted that the eye is a particularly good target for stem cell therapy compared to other areas of the body.

$4.4 Million Grant Approved for Severe Airway Obstruction

The governing board of the California stem cell agency today effectively approved a $4.4 million grant for research involving severe airway obstruction.

The board approved a staff recommendation to place the grant in a category that the board automatically votes to fund. Names of the applicant and employer have not been released by the agency.

Disease Team Applications Under Discussion

The $100 million disease team round applications are under discussion currently at today's meeting of the governing board of the California stem cell agency.

CIRM Chairman Jonathan Thomas outlined the appeal process prior to the discussion and cautioned rejected applicants in the audience that the board is not the first avenue of appeals.

The board has revised its appeal process in the last year to discourage appeals directly to the board.  Over the last couple of years, the appeals have often involved emotional presentations by patient advocates seeking to support applications.

Nonetheless, under state law, any person has a right to address the board on any subject.

CIRM Board Meeting Begins in Los Angeles

Directors of the California stem cell agency opened their meeting at 9:08 a.m. PST today with a presentation on the annual audit.

Live Coverage Upcoming of CIRM Board Meeting: Presidential Selection, $100 Million in Grants on Table

The California Stem Cell Report will provide live coverage of today's meeting of the governing board of the $3 billion California stem cell agency beginning at 9 a.m. PST.

Directors are scheduled to approve criteria for selection of a new president and award roughly $100 million in their ambitious disease team program, which is aimed at pushing stem cell research closer to the marketplace.

Here is a link to the agenda, which has background material on most of the items.

Wednesday, December 11, 2013

Sharp Exchanges as Stem Cell Board Tables Future Funding Proposal

A plan to finance the California stem cell agency after its $3 billion runs out in 2017 was shunted aside today after it ran into sharp opposition and questions from several of the agency's governing board members.

Vice chairman Art Torres attacked the preparation of the report as "offensive" because none of the patient advocates on the board, of which he is one, were consulted in its preparation. He said the CIRM staff should have prepared the $150,000 report -- not a consultant. Torres said the issue of sustained funding for the agency is the responsibility of the Chairman Jonathan Thomas, not the outgoing President Alan Trounson.

Torres' heated remarks drew a sharp retort from Trounson, who summarized the report for the board and said it was part of his management goals. Trounson said that he considered Torres' remarks an "attack on me," an assertion that Torres denied.

Other board members, including Thomas, said the report requires further thought and testing. They said the new president, who will replace Trounson, should be involved. CIRM Director Jeff Sheehy, another patient advocate, moved to table the report and put it in the hands of Thomas with the proviso that no further funds be spent on it pending further action. Sheehy's motion was approved on a voice vote.

The report was aimed at dealing with the loss of state bond funding for the agency.  The report called for private-public partnerships that would mean closer ties to industries. The funding would undoubtedly be considerably less than the $300 million or so that the agency currently hands out annually.

The proposal would also mean a considerable change in the nature of CIRM's program, likely focusing even more on research that is close to the marketplace.

Another bond issue for funding the agency has not been ruled out by the agency. But taking it to the voters is difficult politically and financially. The initiative campaign in 2004 that created the stem cell agency cost more than $30 million.  Stem cell scuttlebutt has it that former CIRM Chairman Robert Klein is talking up another bond election and perhaps even raising campaign funds.

Here is a link to Trounson's Power Point presentation today.

New Funding Plan for Stem Cell Agency Coming Up Shortly

Next up before the California stem cell agency board of governors is the proposal for a public-private funding plan to carry the research forward after 2017, when its current funding runs out. The board is on a short break and will resume in about five minutes.

Stem Cell Lab Program Nixed in California

Directors of the California stem cell agency today eliminated future funding for a $72 million "shared" labs program that supporters said was a key to successful stem cell research in the Golden State.

The action came on a 4-0 vote with two abstentions following a presentation by 20 stem cell scientists that the labs were vital to research and needed for the training of scientists. Jeanne Loring, head of the Scripps stem cell program, told the board today that the labs have evolved to become the place "where all of the research for CIRM takes place." She later said in an email that each lab is currently receiving only $200,000 a year.

The agency's Scientific Advisory Board recommended no further funding of the labs, saying their original justification was no longer "compelling." CIRM staff agreed, saying alternatives to shared labs were available.

CIRM President Alan Trounson said the agency had a finite number of dollars and "sometimes we have to cut off some these things." He said that the labs are "probably not as important as some other things" and that the labs are a "luxury."

The labs have another six to 12 months of remaining funding. Steve Juelsgaard, a CIRM board member, said the labs should have been planning for a loss of funding from the agency.

Only seven of the 29 members of the agency governing board could participate in the discussion or vote because of conflicts of interest. One of the seven was absent from the meeting. The other members of the board are tied to institutions that operate shared labs.

The program was begun in 2007 and has been funded to the tune of $72 million since then. Loring later said in an email to the California Stem Cell Report,
"You should mention that each of the shared labs was getting $200,000 a year…that's it. That's what they decided to cut.  We have leveraged that money 10-fold from NIH, but NIH will not support cores directly."
(Loring's comments in her email were added following publication of the original version of this story.)

California Stem Cell Agency: $200 Million Approved to Speed Up Stem Cell Cures

The California stem cell agency today set aside $200 million to accelerate research into early stage clinical trials over the next several years.

The plan was unanimously approved as directors considered ways to generate therapeutic results as they strive to fulfill promises made to California voters in 2004 when they approved creation of the $3 billion research effort.

Ellen Feigal, senior vice president for research and development for the agency, told directors that about 14 existing projects would be examined for acceleration with possibly up to six to eight ultimately selected. The grants review group would consider the projects, and its recommendations would be brought to the governing board for final approval. One key criteria would be the likelihood that the trial would demonstrate proof of concept by 2017. (See here for her presentation and full criteria for selection.)

The $200 million set aside would go for possible phase two clinical trials. Some directors said more money was needed because costs generally rise. Additionally stem cell research is a new field and is likely to be more expensive than conventional research. The agency would also beef up support for the projects with more advice from external advisers and CIRM staff.

Funding for the agency is set to expire in 2017, and the agency is seeking results that will support a drive to raise additional cash.

California Stem Cell Meeting: CIRM Directors Decide to Press Harder on Therapy Development

Directors of the California stem cell agency today voted to intensify their focus on a handful of projects most likely to advance through clinical trials despite concerns that other efforts by the agency could suffer as a result.

On a unanimous roll call vote, the board approved a motion to create a priority system as recommended by its new Scientific Advisory Board.

"It is time for us to be brutal," said CIRM Director Sherry Lansing, a former Hollywood studio chief and former chair of the University of California board of regents. She said,
"Unless we have a home run in will be extremely difficult to get more funding."
The agency is expected to run out of cash for new grants in 2017. It is in the process of trying to devise a plan for some sort of private-public financial partnership. Discussion at the meeting also made it clear that another bond ballot measure has not been ruled out by some directors, who emphasized the need to show results to voters.

Several directors expressed concern that by making a decision to establish priorities the agency would be forgoing efforts to create "the strongest possible foundation" for stem cell science. They questioned whether the agency should behave like a business "trying to hit a home run." However, no votes were registered in opposition to setting priorities.

During this morning's meeting in Los Angeles, CIRM staff briefed directors on the status of CIRM research. The governing board was told that six to eight projects could be selected out of 23 with the goal of accelerating them into phase one and phase two clinical trials.

A figure of $200 million was mentioned as a possible set aside to help the projects move ahead, but the directors this morning did not approve a specific amount. That may come later today.

Several directors commented that today's meeting on priorities and future funding plans may well be the most significant in years.

The board is now at lunch.

California Stem Cell Meeting Resumes

Directors of the California stem cell agency have returned from their break following repairs to their audiocast on the Internet. Discussion of the priorities of the agency is continuing.

California Stem Cell Agency: The Search for More Cash

Directors of the California stem cell agency have mentioned the possibility of cancelling some RFAs for grant rounds that have not yet moved forward as they consider ways to raise cash for additional endeavors.

The governing board is in the midst of a discussion of the agency's priorities and how to raise money for its operations beyond 2017, when cash for for new grants runs out.

The board has taken a short recess while equipment is installed to improve the quality of the Internet audiocast, most of which has been inaudible so far.

California Stem Cell Meeting: Technical Difficulties with Web Audiocast

Alan Trounson, president of the California stem cell agency, is discussing proposals for new directions at the agency at its governing board meeting this morning in Los Angeles. However, his comments are all but inaudible on the Internet audiocast of the meeting.

We have queried CIRM concerning the problem.

Stem Cell Board Meeting: Future Priorities Discussion Underway

"We're down to our last $600 million," said the chairman of the California stem cell agency this morning as he introduced a discussion of future priorities for the research effort.

Jonathan Thomas, head of the governing board, introduced the subject and outgoing President Alan Trounson to kick off what promises to be a very lengthy briefing by CIRM staff, coupled with discussion by board members.

Prieto on the Future of Stem Cell Therapies

One of the longtime directors of the California stem cell agency has commented on “The Elusive Stem Cell and the Power of Hope” item on Monday, which noted that fully tested stem cell therapies are still far from the marketplace.

Francisco Prieto, a Sacramento physician who has served on the CIRM board since 2004, said in an email yesterday,

“The advantage of being an old(er) doctor: I can remember the early 1980’s, when monoclonal antibodies had been identified and there was a great deal of excitement about the potential they held for treating previously untreatable diseases. It took a couple of decades, but these 'biologicals' have indeed become routine parts of medical therapy.  It may take a while yet, but I fully expect that we will see the same thing happen with stem cell based therapies.”

Cash to Cures: California's Stem Directors Begin Today's Meeting

Directors of the $3 billion California stem cell agency this morning began a key meeting in Los Angeles as they pursue new directions and more cash to continue their efforts beyond 2017.

The two-day meeting also will see action on about $100 million in awards in the agency's ambitious disease team round, which is aimed at pushing research into clinical trials. Additionally on tap is discussion of the search for a new president.

Bloomberg: California Stem Cell Cash Shrinking, Pressure for Results

The headline this morning on Bloomberg News read “California’s Stem-Cell Quest Races Time as Money Dwindles.”

 The status report on the $3 billion California stem cell agency came as its 29 directors meet in Los Angeles today to consider new directions for the agency – not to mention finding a source to replace the funding which runs out in 2017. (Live coverage of the meeting will begin at 9 a.m. PST on the California Stem Cell Report.)

 The article by Mark Melnicoe is a rare national/international look at the Golden State's unprecedented, nine-year-old effort to turn stem cells into cures, as the agency's motto goes. The California Institute of Regenerative Medicine (CIRM), the formal name for the agency, is largely ignored by the national media. California news outlets also devote few resources to covering agency matters.

Melnicoe's piece covers familiar ground for readers of California Stem Cell Report and others familiar with the agency. But it is a valuable tool for understanding how “outsiders” may view the effort.

Melnicoe wrote,
“California’s government-run stem-cell research agency, on course to spend $3 billion in taxpayer money to find treatments for some of the world’s most intractable diseases, is pushing to accelerate human testing before its financing runs out.”
He continued,
“The largest U.S. funding source for stem-cell research outside the federal government, it’s under pressure to show results to attract new money from pharmaceutical companies, venture capitalists or even more municipal bonds. 
“'We need to figure out how to keep them going,' said Jonathan Thomas, a founding partner of Saybrook Capital LLC in Los Angeles, and chairman of the institute’s board, which meets today. 'We could do public-private partnerships, venture philanthropy, a ballot box.'” 
The Bloomberg article also sounded a cautionary note. It said,
“Brock Reeve, executive director of the Harvard Stem Cell Institute, said a rush could waste money by going too far down paths with false promise. 'There have been a lot of clinical trials in the stem cell field broadly that haven’t panned out,' Reeve said.” 
Specifically mentioned in the Bloomberg piece was the clinical trial by Geron, the first in the nation of an hESC therapy, which was partially financed with a $25 million CIRM loan. The trial was abandoned by Geron, which then sold its stem cell assets to Biotime, an Alameda, Ca., firm, whose top executives come from Geron. Biotime has not resumed the trial.

Tuesday, December 10, 2013

California Stem Cell Researchers Fight for Shared Labs

Twenty California stem cell scientists are seeking to save the state's heralded, $72 million “shared” stem cell lab program, arguing that it is vital to research and necessary for the continued training of scientists.

The researchers made their appeal in a letter (full text below) expected to be presented tomorrow to the governing board of the $3 billion stem cell agency, which funds the effort. The agency's new Scientific Advisory Board (SAB) has recommended the program be discontinued and more emphasis placed on a handful of clinically oriented projects.

In a very brief recommendation that has been accepted by CIRM staff,  the advisory board said that “the importance of these resources to the mission of CIRM and achieving sustainability of earlier investments is not as compelling (as when the program began).

The SAB continued,
“These resources should operate on a revenue-neutral basis through recharge mechanisms and gain other needed support from the host institutions.” 
The lab program began as a $50.5 million effort in 2007 and was given an additional $21.5 million in 2011 after CIRM staff cited its "exceptional" performance. Former CIRM Chairman Robert Klein praised the effort, citing its ability to leverage funding. He said,
“The $4 million awarded, for example, to Stanford for its shared lab have enabled its scientists to successfully compete for $35 million of grants from numerous sources, with another $15 million in process.”
Responding to the SAB recommendation, supporters of the labs said that without them, “development of protocols for generation of clinically needed cell types will not be possible.” Their letter said the 17 labs have been “vital to current and future CIRM-funded efforts.” There is “no alternative place' for the type of research underway at the labs, said the scientists.

The researchers signing the letter were from institutions throughout the state, including UCSF, Stanford, UC Berkeley, USC, UCLA, Salk, Scripps, Sanford Burnham, UC Irvine and UC Santa Barbara.

The letter, which was made available to the California Stem Cell Report,  said,
“In the worst case scenario, the loss of indirect cost support to the institutions will mean that the core lab space will be reallocated (a certainty for many independent research institutes), effectively dismantling the Shared Laboratories and putting CIRM equipment into storage. Shared Laboratory personnel provide support not only for the millions of dollars of CIRM purchased equipment, but often also the additional equipment that has been funded by other sources as a result of the CIRM infrastructure investment but often also the additional equipment that has been funded by other sources.... 
“Even in the best cases, there will be no means to support the staff and maintain the equipment. Some institutions may be able to provide space without indirect costs, but will not fund personnel and equipment maintenance. Thus, the original CIRM investment in infrastructure will be lost, and CIRM’s highly successful training programs for new workers (CIRM Bridges and Training Grants), will be left without homes.”
Here is the full text of the letter and a summary of survey of activity at the shared labs.

Live Coverage of Tomorrow's Hefty Meeting of the California Stem Cell Agency

The California Stem Cell Report will provide gavel-to-gavel coverage of the meeting tomorrow and Thursday of the governing board of the California stem cell agency, which is scheduled to give away roughly $100 million and at least initially lay out its future direction.

The Los Angeles meeting begins at 9 a.m. PST tomorrow and is scheduled to end at 4:30 p.m. Stories will be filed as warranted.

For those wanting to participate in the meeting, the location in Los Angeles can be found on the agenda. Remote teleconference locations for participation are located in Sacramento (tomorrow only), La Jolla, Pleasanton, Costa Mesa and Davis. Addresses are on the agenda.

Also on the agenda are instructions for listening via an audiocast (listen only) and a Webinar Web site that provides access to Power Point presentations used by staff.

The agenda is robust, to use one of the agency's favorite words, and is loaded with data and information on virtually all of the items. However, much of the information is raw with little analysis concerning its significance.

The California Stem Cell Report will base its coverage on the Internet audiocast from its post aboard a sailboat in Huatulco, Mexico.

Monday, December 09, 2013

The Elusive Stem Cell and the Power of Hope

It was, you might say, the best of times and the worst of times last week for the stem cell community, particularly in California. At least based on a spate of headlines and news stories.

Largely ebullient rhetoric rolled out of San Diego, only to be tempered or contradicted elsewhere. The World Stem Cell Summit was underway in Southern California, generating a small wave of coverage.

On Tuesday, Bradley Fikes of the San Diego UT reported,
“Stem cell research has already yielded historic breakthroughs against incurable diseases, a panel of top stem cell researchers said at a public forum Tuesday evening. And that's just the start....”
On Wednesday, the $3 billion California stem cell agency carried on with the theme and blogged about “breakthroughs.”

On Thursday, GEN News quoted Bernard Siegel, the major domo of the summit, as saying,
 "The field is a true scientific revolution and reflects the transformative power of hope...."
But then there was this midweek story, also by Fikes,
“Stem cell research faces a budget crunch -- The cash-strapped federal government’s ability to fund stem cell research has become severely limited....”
On Saturday, Bloomberg headlined a discouraging story about research based in Boston,
“HIV Returns in Two Men Thought Cured After Stem Cell Transplant."
And earlier in the week, a consultant to the $3 billion California stem cell agency made it clear that after 2017, it will no longer be handing out $300 million a year in research grants. His report also noted that it would take 10 years or so for stem cell research to emerge from early clinical trials to become real, commercial therapies.

The diverging stories and comments reflect the ebb and flow of a young technology. They also reflect the inveterate optimism of many researchers and patient advocates, who publicly see few barriers to stem cell therapies. Catriona Jamieson, a scientist at UC San Diego who appears to be enjoying remarkable success in her work, last week commented,
"The biggest barrier is nihilism."
Cash is short, however, not only in the federal government and at the California agency, but also in the private sector, which has basically stiff-armed stem cell research for the last several years or longer.

Despite some recent, promising signs of interest from some companies, Siegel's comment may be the most on the money about the current state of affairs: The stem cell field continues to reflect the “transformative power of hope.” And widely available, safe, effective and reasonably priced stem cell therapies remain elusive.

Sunday, December 08, 2013

$98 Million for Stem Cell Therapies for Cancer, Arthritis and More

Directors of the California stem cell agency this week are expected to approve nearly $98 million in awards for early stage clinical trials for treatments of diseases ranging from sickle cell anemia to cancer.

The awards will involve the $3 billion agency's signature disease team program, which is aimed at pushing stem cell research into the marketplace.

In addition to sickle cell anemia and cancer(see here, here and here), the applications involve therapies for macular degeneration, arthritis and severe airway obstruction. Names of the applicants were withheld by the agency. The identities of successful applicants will be released following board action. Names of the rejected applicants, however, are not released by the agency. The number of business applications was also not immediately released.

The goal of the latest round in the disease team program is completion of an early phase clinical trial during the award period, which can be up to four years. Amounts of the proposed awards range from $20 million to $4.4 million. CIRM originally budgeted $100 million for this round.

Four applications won outright approval from the agency's grant reviewers. CIRM staff recommended that three out of four “tier two” grants be approved. The scientific scores of all seven of those applications range from 79 to 67 on a scale of 100.

Some of the five rejected applicants and sympathetic patient advocates may well appear at the board meeting in Los Angeles on Wednesday and Thursday to lobby for their proposals. The CIRM board can fund any of the applications, regardless of their scientific scores.

Here are links to the ample information provided by CIRM staff on the grant applications: summaries of the grant reviews, staff justification for funding tier two applications, Power Point presentations to be made to directors on the applications, including the range of scores on individual applications. However, the range of scores on the rejected applications was not provided.

Thursday, December 05, 2013

The Californa Stem Cell Agency: A Blueprint for Living Without $300 Million a Year

The likely future of the $3 billion California stem cell agency was unveiled this week, and it envisions an enterprise no longer tied to state funding and much more closely linked with industry as a collaborator and “bundler” of resources.

Gone would be the $300 million a year in cash that the state borrows so that agency can award grants to academics and occasionally to business. Likely to be missing are faculty recruitment awards, non- business training programs and perhaps most of the agency's basic research effort.

Instead, the California Institute for Regenerative Medicine(CIRM), as the agency is formally known, would build a relatively small number public-private partnerships to back projects close to turning out commercial therapies. It would ally itself with the Alliance for Regenerative Medicine, a national lobbying and industry group in Washington, D.C. And the agency's funds would come possibly from foundations, philanthropists, investors, biopharma, the health insurance industry and federal agencies such as the NIH and Medicare.

That is part of the scenario painted in a $150,000, 69-page report by consultant James Gollub of Tiburon, Ca. CIRM earlier this year commissioned the report because the agency will run out of state funds for new grants in 2017.

The governing board of the stem cell agency will hear a report on the Gollub's recommendations at its Dec. 11 and Dec. 12 meeting in Los Angeles. The report will come as part of consideration of proposals by the agency's new Scientific Advisory Board that the agency should sharpen its focus on six to eight projects to push them closer to bearing commercial fruit. The proposed “strategic roadmap” also comes as the agency is looking for a new president to replace Alan Trounson, who is leaving to rejoin his family in Australia.

Trounson has prepared an outline of a plan on how to start implementing the proposals.

Gollub made three major recommendations, one of which would require a $50 million investment from CIRM with another $50 million to $100 million coming from other sources, including wealthy individuals such as businessman Denny Sanford who recently gave UC San Diego $100 million for stem cell research.

The report said additional costs, including those for outside consultants, could be absorbed by CIRM or funded through awards on a charge-back basis. In others words, a grant would include funds that a researcher would have to pay to the agency for its “internal program management services.” CIRM is limited by law to an operating budget that can total only 6 percent of its $3 billion, which keeps its overhead quite lean.

Gollub noted that that CIRM has already moved partially into the areas of his three proposals, all of which would begin in the next year or so. They are:

  • Create public-private partnerships to move projects into early clinical trials, focusing on specific disease areas. Co-funders providing at least $50 million would screen and select the projects from those presented by CIRM. This could be scaled up from a pilot project next year and possibly involve creation of a nonprofit group by CIRM.
  • Create a “regenerative medicine accelerator” to provide “commercial readiness services” to each(Gollub's italics) grant recipient whether academic or business. The accelerator effort would be linked to agency's proposed $70 million Alpha Clinic plan. The accelerator also would assure that the “clinical trials structure meets pharmaceutical industry expectations.”
  • Create a pre-competitive regenerative medicine R&D program that would organize collaborative efforts to break through barriers to development of therapies. This would involve production but also what the biotech industry calls “reimbursement,” which is a catch word for making a profit from development of a therapy. One of the issues in the industry is the expense of some medical treatments, and stem cell therapies are expected to be very high. The idea is to work with the insurance industry and the federal government to be sure the appropriate cost is supported by Medicare and insurance programs. Partnering with the Alliance for Regenerative Medicine would enter into this effort, which would be ultimately funded by both CIRM, the new non-profit and other partners.
The general direction of the recommendations is to “de-risk” stem cell therapy development and provide a focal point for overcoming many of the regulatory and manufacturing obstacles facing a new medical technology.

The report said CIRM has as a “strong innovation feedstock” of more than 90 projects that are close to moving into clinical trials. Some of those projects are likely to appeal to Big Pharma, which has been increasingly looking outside of its own companies for R&D.

For donors willing to pay for the privilege, Gollub's “strategic roadmap” recommended that CIRM create a group that would have “'a seat at the table' to see early stage  research, discoveries and clinical performance.”

The report said the CIRM's new world would require a “robust, dedicated fundraising group” within the agency, which now has minimal capacity in that area. However, the study envisions members of the governing board, many of whom are top notch fundraisers, as making a major effort to raise cash.

Scaling up the initial public-private partnerships, accelerators, etc., would also require additional staff. Gollub's report did not present costs beyond the initial pilot project stage.

Monday, December 02, 2013

California Stem Cell Report Taking Some Time Off

The California Stem Cell Report is going dark for a number of days while yours truly makes an ocean passage from Chiapas to Zihuatanejo.

As some of you know, this report is published from a sailboat called Hopalong on which this writer lives fulltime south of the border with his spouse. And Hopalong does move about from time to time.

We may have Internet access along the way to monitor the affairs of the California stem cell agency but cannot count on it. We should be back to the grindstone in about two weeks.

Searching for CIRM's Financial Future, Plus a $100 Million California Stem Cell Round

The California stem cell agency next week is expected to give away as much as $100 million as it pushes aggressively to generate therapies that will benefit the Golden State as well as the world.

At the same time, the agency will decide whether it will focus more intensely on a handful of projects as it tries to come up with a plan to finance its future. The $3 billion agency is currently funded by money that the state borrows, but cash for new grants will run out in 2017.

The agency is looking for a way to continue operations through some sort of public-private partnership. A consultant's report was due on that subject on Nov. 30. At its meeting Dec. 11 and Dec. 12 in Los Angeles, the board is scheduled to spend one hour hearing a presentation on the report at the end of a lengthy examination of recommendations from the agency's new Scientific Advisory Board(SAB), the panel that proposed that CIRM sharpen its focus. The California Stem Cell Report has asked for a copy of the document, which is a public record.

The SAB report plays into the agency's need to come up with results that will stimulate both private and public interest in supporting the agency's future operations. So far, CIRM has lacked the research results that resonate widely with the public.
The agency has prepared a tightly organized schedule for examining the SAB proposals, which could mean that the agency's shared labs, bridges and training grants programs would be phased out. However, CIRM staff is only recommending an end to the shared labs program.

The staff has proposed creation of an “accelerated development pathway” for six to eight projects, as recommended by the SAB. The staff document did not describe what that would entail or how much it would cost.

Also on the staff list is a proposal to advance the most promising early translational research programs and a $10 million proposal by CIRM President Alan Trounson to participate in a global effort “to create (a) Haplotype Bank and GMP library.” No further details on that plan were available at the time of this writing.

For the stem cell community, the immediate big news next week will be board action on applications in its signature disease team program. This is the third round, and awards could run as high as $20 million each for a total of $100 million. The round also involves possible collaborators in England, Spain and China, a country that has been pushing hard on stem cell research. No state funds will be used overseas.

According to the RFA, the purpose of the disease team effort is “to advance early clinical development of novel therapies derived from or targeting stem cells, potentially offering unique benefit with well-considered risk to persons with disease or serious injury.”

The summaries of the grant review findings have not yet been posted on the CIRM Web site, but are likely to come later this week. The CIRM governing board almost never overturns a positive recommendation from its reviewers. Sometimes it will approve an application rejected by reviewers. However, directors are feeling squeezed financially and may not be as open to elevating rejected applications as they have been in the past. Look for rejected applicants to personally petition the board for approval along with affected patient advocates.

Also on the agenda is the topic of the search for a new president for the agency to replace Trounson, who is resigning to return to Australia and his family. An item was posted on that yesterday.

Next week's meetings are open to the public in Los Angeles and at teleconference locations in La Jolla and Pleasanton. Addresses can be found on the agenda. The board also usually provides an Internet audiocast and a Webinar link to PowerPoint presentations used at the meeting. Directions for using those tools are likely to be posted on the CIRM Web site later this week.

Sunday, December 01, 2013

California Stem Cell CEO Search: Non-Scientist Candidates Quite Welcome

The $3 billion California stem cell agency could well have a non-scientist as its new president as it pushes to develop commercial products and develop future funding sources for the nine-year-old research enterprise.

Directors of the agency meet next week to settle on the criteria for the new CEO, who directors would like to see in place as soon as possible. Specifically missing from the board's latest wish list is a requirement that the individual be a scientist or researcher. Instead, the criteria say candidates should have “experience with and personal commitment to medical and scientific research including familiarity with stem cell research.”

In terms of academic credentials, the proposed criteria specify either or both an M.D. or Ph.D. degree or “equivalent industry experience or similar body of knowledge developed in professional roles.”

That sort of broad description could mean several things. One is that the board already has a candidate in mind and directors don't want to hampered by perceptions that only a scientist can run CIRM, as the agency is known. The description also significantly widens the field of possible candidates, virtually inviting non-scientists to apply for the position, which carries a salary of up to $548,788 annually.

The criteria, however, make it clear that candidates should be heavyweights in development of scientific products. The agency wants tested leaders of scientific organizations, experience with R&D, including the regulatory process for development of new treatments, and persons who have no problem shutting down projects that aren't going anywhere.

The latest job description for the person to replace CIRM President Alan Trounson, an internationally known scientist, came together after an earlier meeting of the board's Presidential Search Subcommittee, which will review and perhaps modify it at a public meeting Dec. 10 in Los Angeles. Then the full board will act on it during its two-day meeting Dec. 11-12, also in Los Angeles.

Teleconference locations for both public meetings can be found on their agendas, which are here and here.

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