"If you want a bike, don't ask for a toy"
Scientists welcome to comment
Directors of the $3 billion California stem cell agency this month will
take a crack at building a new, basic research “railroad” with the hope of creating
a “predictable path” to development of therapies and cures.
The move will involve funding for basic and translational research, the
initial steps that lead to producing therapies that can be widely used by the
public.
The effort is scheduled for approval at a meeting July 15 of the agency
directors’ Science Subcommittee. The agenda for the meeting was posted last
week minus details of the “concept plan” for the move. However, the changes are
part of the CIRM 2.0 effort initiated by Randy Mills, president of the
California Institute of Regenerative Medicine (CIRM), as the agency is formally
known.
Mills kicked off CIRM 2.0 in January. His initial foray involved the
more advanced research efforts that are engaged in testing treatments on human
beings. Mills described the CIRM 2.0 program as “radical.”
In May, Mills sketched out the thinking behind extension of the CIRM 2.0
approach to basic research.
He told directors at their meeting that month,
“The first thing is we need is a continuous predictable path from discovery to clinical. So if we have a discovery program (basic and translational research), the purpose of that discovery program ought to be to deliver something downstream to where it ultimately impacts patients. That kind of makes sense.”
Mills quoted CIRM senior science officer Lila Collins as saying about
the approach,
“If you want a bike, don’t ask for a toy.”
Under past procedures, Mills said basic research findings financed by CIRM only advanced 5
percent of the time. He acknowledged that it is “really difficult” to
foresee where basic research is headed. But he said an analysis of the CIRM
grants showed that when the agency specified that it wanted the research to
progress, it advanced 30 percent of the time instead of 5 percent.
Using primarily a railroad analogy, Mills said,
“We need to put intent into the RFA that says we want you to be creating something, and we want you to be thinking forward ….Maybe you don't take it there, but how is it going to move ultimately downstream? We want you to be thinking about that at the time you apply because if you do, we know it has a much better chance of going to thenext station.”
Mills said,
“We need to have track that's continuous….We need to make sure thetrains run on time. We need to have predictable progression. We call this baton passing. We want the cargo on the train to proceed down the track. And we need to be really creative on how we're going to do that.
“We need to be responsive to new developments and downstream bottlenecks. And then, lastly, we need to have multiple development pathways so we basically serve all of the destinations we want to serve.”
Even if scientists are not entirely pleased with the new approach, they
are likely to climb aboard. Research funds nationally are scarce. CIRM itself
has not offered a basic biology grant round since March 2013.
However, Mills’ latest proposal is still subject to modification. The
Science Subcommittee may ask for changes before sending it to the full board
for approval on July 23 at a session in Berkeley. Researchers and the public are welcome to comment and make suggestions.
Details of the concept plan are likely to be posted at least a few days
before the Science Subcommittee meeting July 15. The meeting is open to the public with sites
available in San Francisco, Cedars-Sinai in Los Angeles, UC Irvine, Pasadena
and San Diego. See the agenda for addresses.
Written comments can be emailed to CIRM directors via
mbonneville@cirm.ca.gov.