In the past year, CIRM has begun funding early phase clinical trials for stem cell-based therapies. These early phase clinical trials are done to make sure that the prospective therapy is safe, and can also show preliminary evidence that it may be effective. What we don't fund are the large, later phase clinical trials that the Food and Drug Administration requires to confirm whether or not the therapy is safe and effective before approving a new therapy for patients. (This resource has information about the stages of therapy development.)
These large clinical trials cost a lot of money, and conducting these trials can benefit from the kinds of expertise that industry partners can provide.
Ideally, what would happen is that CIRM would fund the research leading up to FDA approval to begin a clinical trial, and we'll fund some early phase trials (or provide matching funds through a loan, such as the Geron trial we're supporting now). For trials that start with investigators in universities, or in small companies, larger industry partners would then have the opportunity to help fund the early trials and would pick up any project that succeeded in early trials and carry out the expensive later-stage confirmatory trials.
The new $30 million Strategic Partnership Funding Program will promote the partnerships between academic groups or small businesses and the larger industry partners whose participation will be critical for carrying out the late-stage clinical trials. Forming those relationships also provides our grantees with a source of advice that can help them design their early trials and overcome regulatory hurdles.
Elona Baum, CIRM General Counsel and Vice President of Business Development, led the effort to develop this fund. She said:
“With this fund we want to make sure that CIRM grantees get early access to the expertise of biopharmaceutical companies who will then, at Phase III, bring the project through the goal line. We want to increase the likelihood that CIRM research has access to funding in the later stage of development which is critical to ensuring it gets into the market place and helps patients in need. We're being proactive by engaging industry early in the process of developing new therapies.”This Partnership fund is one portion of an overall Opportunity Fund. The two other parts of that fund will be presented to the board for approval in December or January. Together, the funds are designed to keep CIRM nimble and looking ahead to the resources and expertise our grantees will need in the future if our funding is going to be a success.