At last week's meeting in Sacramento, our governing board heard an update on the progress being made by the 14 CIRM disease teams that they had approved in October 2009. A list of those teams is available on our website.
Funding teams of researchers with scientific and regulatory expertise to work together was an innovative step—one that CIRM anticipates will help those groups get their therapies to clinical trials faster than they would without this team approach.
Before issuing the awards, CIRM’s science staff worked with the teams to come up with milestones and success criteria. Periodically, CIRM meets with the teams along with a group of experts who have experience taking therapies into clinical trials and through to approved therapies.
Taking the advice of these experts into consideration, CIRM decides whether the projects are achieving the agreed upon milestones. These assessments help protect the state’s financial investment by making sure the teams are focused and have the resources and expertise they need to achieve the goals of their research. It also identifies projects that need additional support, or ones in which the original idea is not ready to continue.
Ellen Feigal, CIRM’s senior VP of R&D, summarized the progress being made by each of the teams. You can read the background material she gave to the board members, which has detailed information about the achievements by each group.
The first CIRM and expert assessments of the disease teams met in 2011, at the 12 to 18-month milestones. The group found that thirteen of the projects are on a trajectory toward meeting their milestones. Of those, one is undergoing some revisions to focus the work. One of the projects is not continuing. CIRM will save roughly $13 million on this award through early termination.
The team that is not continuing, led by researchers at the University of California, San Francisco, produced data that did not meet the agreed upon success criteria. That criteria had to do with selecting the best stem cell type to use in their proposed therapy for a type of brain tumor called recurrent glioma.
During the course of their work, the team had generated some new methods that may be useful for other researchers and published two papers. These lessons learned are part of the iterative process of trying to create new therapies.
As with all potential therapies, none of the therapies developed by the CIRM-funded teams can enter clinical trials until they provide evidence to the U.S. Food and Drug Administration (FDA) that the therapy is safe to test in humans. Carrying out these experiments is costly and brings with it the possibility that the original idea won’t work. The researchers then needs to go back a step and take what they learned from the first attempt and use those lessons to guide other approaches.
CIRM created the disease teams as a way of funding this critical stage of research. We instituted the milestones and the input of product development experts to better position the teams for success and protect the state’s financial investment. This way, we are trying to ensure the teams have the resources and expertise they need to bring these much needed therapies into clinical trials in humans.
CIRM and our panel of experts will be meeting with the thirteen teams at their 24- to 30-month milestones in 2012 to assess progress. Regardless of the outcome of those assessments, each of the teams is producing data and gaining experiences that will help guide the next generation of stem cell-based therapies.
A.A.
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