Tuesday, December 09, 2014

CIRM 2.0 -- California's New Urgency for Stem Cell Therapies

This is a big week for Randy Mills, the man who took control of the $3 billion California stem cell agency just seven months ago.

His proposals for radical changes at the agency are expected to be approved by its governing board on Thursday along with a restructuring aimed at improving speed, efficiency and innovation.

It all comes under the rubric of CIRM 2.0,” a phrase coined by UC Davis stem cell researcher Paul Knoepfler and adopted by Mills after he became president of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.  

 How important are the changes? Important enough for the agency to tout their mere outline in a press release out of last October’s board meeting instead of focusing on the hard news of the approval of its much-ballyhooed Alpha stem cell clinic program.

Randy Mills
CIRM photo -- Todd Dubnicoff
Mills has made his career in business, serving as 10 years as the CEO of Osiris Therapeutics of Maryland. He managed to make Prochymal the world's first government-approved stem cell drug approved for use on an off-the-shelf basis. Mills likes to move fast, which is largely the point of CIRM 2.0, and says that he wants to make the agency “radically more effective and efficient.”

The agency’s news release in October quoted Mills as saying,
“Right now it can take almost two years for a promising idea to go from the application to the final funding stage. That’s just unacceptable. We are going to shorten that to just 120 days.”
He also told directors at their October meeting,
 “We are in the business of trying to save people’s lives….We have to behave with the appropriate sense of urgency.” 
Mills knows that stem cell companies can’t sit around waiting two years for cash. Most operate on a short financial leash and are perpetually having to raise money. Mills also knows that researchers with non-profit institutions cannot wait for whenever it is convenient for the agency to bless them with greenbacks. Those researchers want to be first with their findings. And they need cash to meet payrolls and to satisfy their sponsoring institutions.

So Mills says,
“We’re not just making it faster, we’re also making it easier for companies or institutions with a therapy that is ready to go into clinical trials to be able to get funding for their project when they need it. Under this new system they will be able to apply anytime, and not have to try and shoehorn their needs into our application process.” 
At the end of January, the agency will begin accepting grant applications related to clinical trials on a monthly basis. The promise is for quick decisions and quick cash. Rejected researchers will – if their thinking has CIRM potential – be coached and guided into preparing a fundable project. Accepted researchers will find themselves working more closely than ever with CIRM staffers to develop something that will emerge as a marketable product. 

Mills is convinced that CIRM 2.0 will also improve applications, generating more proposals that will be scored at 95 out of 100 by scientific reviewers instead of the many applications scored at 75 that have been regularly approved.  

All of the details of the plan are still not clear. But $50 million will be set aside for the first six months of next year. Applicants can ask for whatever amount they want, but budgets will be scrutinized before they even get to the scientific reviewers. Appeals will not be allowed.  Milestones must be met or the cash dries up.  

The whole process sounds a bit like a venture capital operation minus face-to-face pitch meetings.
New rules dealing with conflicts-of-interest on the part of reviewers will be in place. They will allow applicants to seek to disqualify up to three reviewers for almost any reason. However, applicants will not be told who is reviewing their applications behind closed doors. They will have to guess by ferreting out reviewer names from CIRM’s list of more than 100.

Little public criticism of the plan has been heard at the two initial briefings that Mills has given board members. It is clear that there will be hiccups or worse, like any new process. One question involves transparency. Will the public or other scientists know which applications have been rejected along with the subject of their research, as they do currently. Another question involves board involvement. Currently seven members of governing board sit on the review committee. How will they participate on a monthly basis?

Coaching rejected applicants can lead to better or more targeted proposals. But is that something that researchers will readily accept? Does the whole process move the board farther away from the grant-making process. Already the directors have turned over what once were public appeals by rejected applicants to agency staff to be handled behind closed doors. However, applicants can still speak directly to the board on their own on any subject under a state law that CIRM cannot change.  But none has been successful recently in winning grant approval through that route.

Mills says his reorganization plan for CIRM staff – he prefers the word “team” – will create “organizational clarity and operational efficiency.”  It will certainly help to break up ossified structures that may have grown up during the tenure of former President Alan Trounson, who was not known for his managerial or organizational skills.

The plan also would seem to have some impact on the controversial dual executive arrangement involving the president and the chairman of the agency, Jonathan Thomas, who is also salaried. On the surface, the reorganization would seem to remove some responsibilities from the chairman and cost him some of his staff. However, the agency has not responded to questions concerning that area.

Kevin McCormack, senior director of communications for CIRM and who is also one of the top executives at the agency, will no longer be reporting, for example, to both the chairman and the president. That shared reporting was insisted on by former Chairman Robert Klein when the position that McCormack came to occupy was created. 

The dual executive arrangement, which is enshrined in state law, has been criticized for minimizing accountability at the agency. It has also led to tussles between Klein and other executives, although no serious disputes have surfaced in public for several years.

CIRM 2.0 holds great promise. It also depends mightily on Mills' leadership and managerial skills in a considerably different environment than he has previously experienced. Will CIRM 2.0 make a favorable impression in the scientific community and with the public? That depends, of course, on the outcome of the research it generates. So far, CIRM 2.0 is barely visible in the stem cell community -- at least according to an item on the blog of UC Davis researcher Knoepfler. His readers were recently asked to vote on the top stem cell story of 2014. CIRM 2.0 came in last with 0.44 percent of the vote.

That sort of response does not discourage Mills.  He said, in an item on Knoepfer’s blog prior to the 0.44 percent showing:
 “We think the more the word gets out about this and all of the other great features of CIRM 2.0, the more high quality interest we will see from industry and academia alike.”
The details of the plan were approved yesterday by the Governance Subcommittee of the CIRM governing board. The plan will come before the full board at its meeting Thursday in Berkeley, with teleconference locations in Los Angeles, San Francisco and Sacramento and is virtually certain to be approved.

 (Here are links to the CIRM blog item on the 2.0 plan and to Mills’ slides that he presented to CIRM directors nearly two months ago. The transcript of that meeting has not yet been posted by the agency. Perhaps Mills can add posting of transcripts to his fast-track efforts.)
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