The California Institute for Regenerative Medicine, the Golden State’s stem-cell agency, has unveiled a revised draft strategy that seeks over the next eight years to increase funding opportunities for biotech companies performing stem-cell research, perform more public and legislative outreach, and hire additional grant reviewers.
CIRM’s 2008 Strategic Plan Update, Stakeholder Draft 12-1-08, an update of its original 2006 plan, also raises the possibility of a second referendum, putting in writing what the agency’s president, Alan Trounson, said in June during a panel discussion devoted to CIRM at the Biotechnology Industry Organization’s 2008 International Convention [BRN, June 30].
Trounson’s arrival as president late last year [BRN, Sept. 24, 2007] helped spark the institute’s effort to update the 2006 plan, as well as its desire to enact “dramatic increases beyond the 2006 Plan in the types of research targeted to elicit therapeutic candidates,” to provide “significantly more investment in ‘disease team’ awards, translational research awards,” and to facilitate “collaboration with industry.”
One “dramatic” funding increase recommended by the revised draft plan includes increasing the $120 million committed by the 2006 Plan over three years ending in 2011 for so-called “disease team” grants, including a 75-percent jump in the current fiscal year alone.
At a regular monthly meeting last week, CIRM’s governing board, the Independent Citizens’ Oversight Committee, approved a plan to award grants to multi-disciplinary disease teams that will be able to begin human clinical trials for stem-cell therapies within four years of receiving funds. In February CIRM will ask research teams interested in the funds to submit a formal request for applications.
“It is anticipated that the scope of disease team awards will be expanded to include funding for clinical trials and that they will be awarded annually (and more frequently if needed)," the draft report added.
The ICOC said it won’t take up the draft strategic plan until its January meeting at the earliest, after placing the report on its agenda for its monthly meeting last week. During that meeting, the CIRM governing board also expanded its awarding of research funding to stem-cell companies, naming six private-sector winners among 18 awardees that will share $19.8 million spread across 23 Tools and Technologies grants.
The ICOC also set a salary for its chairman, Robert Klein, and postponed until January a decision on a new vice chairman to succeed Edward Penhoet. A co-founder and one-time president of Chiron, Penhoet stepped down last month but remains on ICOC’s 29-member board of directors (See SIDEBAR below).
A CIRM spokeswoman told BioRegion News last week that the group is several months away from making a decision on the revised draft strategic plan. The board next month is expected to decide how it will review and discuss the 44-page document, and how it will solicit public comment on it.
“Do we post an e-mail address so the public can e-mail in their comments and we make it very transparent? Do we utilize a special phone number to solicit comment? Do we want to do town hall meetings? How do we want to interact to get this input, both on the public side and on the board side?” the spokeswoman, Amy Adams, said in an interview. “The board wanted more time to look at the draft and think about what they wanted to do. It was posted as a starting point for discussion of what the final strategic plan should be. This is a very draft-y draft.”
The revised draft also calls for adding an unspecified number of researchers with business backgrounds to the Grants Working Group, an influential panel within CIRM that evaluates grant applications and makes recommendations to ICOC.
Saying it will need professionals with “expertise in clinical trials, regulatory requirements, and large-scale good manufacturing practices,” CIRM explained that “the greatest reservoir” of these skills exists in pharmaceutical and biotechnology companies.
“Without the injection of such expertise into CIRM programs, either independently or through formal collaborations between industry and universities and other not-for-profit organizations, CIRM’s ability to accomplish its mission will be jeopardized,” the revised draft concluded.
The revised draft also calls for CIRM to create a “Biotech Advisory Group” comprising representatives from California’s pharmaceutical and biotechnology industries. The group’s principal duties will be to “advise CIRM on industry trends and needs,” help evaluate “CIRM’s interactions” with the sector, and “ensure that businesses benefit from grant and loan programs,” according to the draft.
However, John Simpson, stem cell project director at Santa Monica-based nonprofit Consumer Watchdog and a longtime CIRM observer, cautioned that such a focus on strengthening ties with biopharmas could create conflicts of interest absent clear standards for selecting members and ensuring that their activity is carried out publicly.
“This is a very draft-y draft.”
“If it’s just the president of CIRM saying, ‘I want these five guys to come meet with me weekly,’ I think that’s outrageous, because you essentially would have the people who are lining up and benefiting directly from the money lobbying to get it, going around the well-established procedures, committees, and public forums that now exist,” Simpson told BRN last week.
“If the concern is that business has not bought into the process, it’s not the fault of the organization; it’s that businesses just haven’t bothered to show up at the meetings where things are being discussed,” he added.
CIRM has yet to figure who would serve on a business advisory panel, and how its members would be chosen.
“It seems to me those kinds of things have to be answered. It’s critical that there be some sort of clear criteria for selection, and it’s got to be completely transparent,” Simpson said. "In reading the plan, I don’t see how it’s all supposed to work. If they’re going to get some cures, they’re going to have to involve industry, there’s no doubt about that.”
Despite his concerns, Simpson said, he agrees with CIRM that the 2006 plan “understandably needs to be updated and revised. It’s perfectly appropriate they’re coming out with this” revision.
This private-sector proximity was voiced after other critics claimed CIRM showed too much favoritism in its funding policies toward individual researchers and academic institutions.
Adams, the CIRM spokesperson, denied that, saying the revised draft is “a response from our own internal feeling that as this [stem-cell science] moves forward, we’re getting more and more involved in industry, and we need involvement from industry to help guide us. It’s not a response to any one criticism.”
Criticism over private-sector funding surfaced publically at ICOC’s meeting in June by an official from a regenerative-medicine company that had been turned down for CIRM funding.
Kenneth Woolcott, the chief business officer of Cascade Life Sciences, told ICOC board members his company answered an RFA for grants believing that the novelty of its technology would not be a deciding factor, only to be told otherwise
“We believe that the reviewers in this case simply didn’t read our application very carefully, if at all,” Woolcott said, according to minutes of the meeting. “I didn’t see anyone that had commercial affiliation,” referring to the individuals responsible for reviewing the RFA.
He suggested to ICOC that CIRM emulate the National Institutes of Health and other federal agencies that award Small Business Innovation Research and Small Business Technology Transfer programs.
“There’s a process by which you can get … comments” made by officials reviewing grant applications, said Woolcott, whose company is based in La Jolla. “If there’s a misunderstanding or a miscommunication, you can respond back. And so I’m suggesting the committee might consider a little bit of dialogue between the applicants and the reviewers.”
Woolcott did not respond to a request for comment in time for this publication.
The six private-sector winners of CIRM’s Tools and Technologies grants announced last week, in order of descending dollar amount, are:
- VistaGen Therapeutics, which is developing an hES cell-based assay system for hepatocyte differentiation studies and predictive toxicology drug screening, $971,558.
- Gamma-Medica Ideas, which is creating a novel SPECT microscopy system for 3D imaging of single stem cells in vivo, $949,748.
- Vala Sciences, which is developing a technology that optimizes how researchers identify, select, and induce maturation of subtypes of cardiomyocytes derived from human embryonic stem cells, $906,629.
- Invitrogen (now Life Technologies), which is generating disease models for neurodegenerative disorders in hESCs, $869,262.
- Novocell, which is developing an undisclosed cellular-encapsulation system for delivering hESC-derived pancreatic islets and progenitors, $827,072.
- Fluidigm, which is developing a fully automated, microfluidic cell-culture system, $749,520.
CIRM has awarded funds to businesses before, Adams noted. In June, it awarded a $48,950 grant to Novocell to help it develop a cell-replacement therapy product for insulin-dependent diabetes. The Novocell team, led by chief science officer Emmanuel Edward Baetge, was the only winner among nine companies applying for “Disease Team Planning” grants.
Howard High, a spokesperson for South San Francisco-based Fluidigm, told BRN last week that the company’s research will pick up on work performed by Stanford University graduate students studying under company co-founder Steve Quake.
High said he did not know if CIRM’s effort to fund more stem-cell businesses would prompt Fluidigm to seek additional grants from the agency: “Since [stem-cell research] hasn’t been a main thrust of ours in the past, what we’re going to try to do is do an excellent job” on the original CIRM award.
“Really, our main focus as a company is the commercialization of integrated fluidic circuits across a broad range of research and commercial applications,” said High. “I can see stem cells being an increasing part of what we look at, but in terms of our overall business, I’m pretty sure it would remain a relatively small portion of what we do in total.”
Details of the winning grants are available here.
Lobbying Lawmakers, Wooing the Public
The revised draft also recommends that CIRM step up its outreach to lawmakers and the public. Adams told BRN the group would work on issues with the Biotechnology Industry Organization and California’s three regional life-science groups — an effort now in discussion stages.
Eager to gain greater clout in Sacramento through a united voice, the regional groups in June formed the California Life Science Alliance [BRN, June 23], an effort she said CIRM would work with rather than duplicate.
“The first thing we’re doing is just talking to the lawmakers, finding out what they want to hear from us that would be useful,” Adams said. “To some extent, what might be useful to them is getting them more involved in what we’re already doing.”
To maintain the support of lawmakers, the report said, CIRM will conduct advocacy workshops on pending state and federal legislation; organize briefings for legislators and their staff, sign up many of them for its RSS feed, and continue in-person meetings between lawmakers and CIRM representatives, both its government relations representative, and senior administrators and board members.
“CIRM is building a rapid response team to answer legislative requests and to effectively assimilate legislative input,” according to the revised draft.
A rapid-response team may have helped CIRM this year in its effort to beat back a state Senate bill intended to address drug access and governance issues affecting the agency. The state Assembly and state Senate quickly passed the California Stem Cell Research and Cures Act, but CIRM prevailed when Gov. Arnold Schwarzenegger vetoed the bill, citing arguments made by the agency and patient advocates.
To win over the public and press, CIRM will have to hold more seminars for journalists on stem-cell science and produce more video clips to go along with its press releases, the revised draft said. “These clips will feature certain faculty describing science at a lay level and employ compelling visual images suitable for TV to convey the excitement that stem cell science holds for the future of medicine,” according to the document.
Adams said CIRM will attempt to improve its public outreach by launching a new website either later this month or in early January that will contain better-organized information on grants the agency is funding.
The group also plans to hold town hall meetings in San Diego, Los Angeles, and San Francisco, at which agency-funded researchers would discuss their work, said Adams.
Adams said CIRM will also use an RFA to seek a professional to help it develop curricula for local high schools, and will start trying to form collaborations with local groups that promote science. The agency has begun talking to the San Francisco science museum Exploratorium to see how they can work together, she said.
The revised draft also calls for CIRM to hire more staff scientists to review grant applications, as well as administrative employees.
“Over the next several years, CIRM will need to hire additional employees to carry out its scientific and administrative functions. The anticipated staffing need at steady state is 30-32 science officers and support staff,” the report stated. “Since CIRM’s aggressive science programs will result in approximately 400-500 grants and loans being managed and monitored at any one time, sufficient staffing of the Science Office will be essential to ensure quality control and appropriate investing of CIRM funds in the best science.”
Simpson agreed that CIRM would benefit from additional science staffers. “It’s important that they get the appropriate scientific staff — the people who do much of the work — hired.”
The referendum that created CIRM also limits its staffing to 50 people. The agency is not looking to lift the cap, Adams said, and has yet to consider how many employees it would need, and how quickly it would hire them.
CIRM Board OKs Chairman’s Salary; Vice Chair Appointment, Salary Will Have to Wait
Directors of the California Institute for Regenerative Medicine last week approved a salary for its chairman, Robert Klein, for the first time — $150,000, or just over half of the low end of the position’s salary scale — but held off on two thornier issues: whom to appoint vice chairman, and at what salary.
Klein’s salary, which went into effect Dec. 8, is meant to reconcile his view that the chairmanship is a full-time job, and ICOC’s opinion that the position is part-time work.
Klein has worked without pay since taking the job, but recently told ICOC he could no longer afford to do so.
The salary also wraps up ICOC’s effort to find a compromise after Gov. Arnold Schwarzenegger expressed “deep concern” over the annual salary originally requested by Klein: between $275,000 and $508,750.
“CIRM has a responsibility to spend taxpayer money with great care, so I urge you to ensure that compensation for these positions is offered only if and to the extent absolutely necessary to implement its mission,” Schwarzenegger wrote in a Dec. 4 letter to the ICOC board.
John Simpson, stem-cell project director at the Santa Monica, Calif., nonprofit group Consumer Watchdog, which tracks CIRM activity, said the board made the correct decision. Simpson suggested Klein be paid no more than the director of the National Institutes of Health; NIH’s most recent permanent director, Elias Zerhouni, was paid $191,300.
“If he in fact behaves like a true state employee, and it’s a full-time job, and he’s really making a contribution, then he does deserve a salary,” Simpson said. “He doesn’t deserve a salary of $500,000.”
ICOC governs the California Institute for Regenerative Medicine, which noted that the agency is funded from the $1 billion in bonds approved by state voters through Proposition 71 in 2004, and not the state budget, which has a combined $41.8 billion shortfall projected for this year and next, according to a revised forecast released Dec. 12 by Schwarzenegger.
Klein’s salary was decided during a closed-door executive session held by ICOC as part of its Dec. 10 meeting. During that meeting, ICOC members postponed to January a decision on whom to select from two candidates seeking to succeed Chiron co-founder Edward Penhoet as the board’s vice chairman.
The dispute has political overtones since Schwarzenegger, a Republican, and two Democratic top state officials have nominated different people to the position. Schwarzenegger has nominated Duane Roth, a longtime ICOC board member who is CEO of Connect, an organization that promotes the growth of life science and other technology businesses in the San Diego region.
But Lt. Gov. John Garamendi, Controller John Chiang, and state Treasurer Bill Lockyer, all Democrats, have nominated Art Torres, the chair of California’s Democratic Committee since 1996, and a former state Assemblyman. Torres is seeking a salary for the post, which according to CIRM can range from $180,000 to $332,000.
Roth has said he would not accept a salary if he is selected.
Lockyer last week told a joint session of the state Legislature that the lending source of state agencies borrowing money for bonds, the Pooled Money Investment Account, will be forced to stop loaning money unless the state resolves its budget shortfall.
CIRM is funded through late 2009, a Lockyer spokesman told the San Francisco Business Times.