consideration as it deliberates about the final ranking of applications. If it is necessary to discuss proprietary information, then the ICOC may meet in closed session before a final vote is taken on which applications will be funded. As a result of its private and public deliberations, the ICOC may move applications from one tier to another before taking a final vote, after which applicants are notified about funding decisions. Examination of ICOC records indicates that the shifting of applications from one tier to another does occur. For example, as of October 22, 2012, 62 extraordinary petitions were heard by the ICOC, of which 20 (32 percent) were successfully funded (CIRM, 2012h). Although most of this shifting is between adjacent tiers, there have been cases in which applications have been moved from tier 3 to tier 1 (CIRM, 2011g; IOM, 2012e); this has occurred with applications for major programs with large budgets. As discussed in greater detail below, the committee is troubled by the extraordinary petition mechanism and suggests that this practice be eliminated. The committee recognizes that CIRM has recently initiated a self-study regarding all aspects of extraordinary petitions.

BIOETHICS

Bioethics is part of the portfolio of issues that range across the entire spectrum of projects moving toward the clinic. As stated above, CIRM describes its mission in the 2012 strategic plan as supporting and advancing stem cell research and regenerative medicine under the highest ethical and medical standards. To achieve this mission, CIRM has proposed as one of its main goals advancing stem cell research to clinical trials to establish evidence of therapeutic benefit to patients. The most important milestone toward this goal is achieving clinical proof of concept for new therapies within the next 5 years (CIRM, 2012a). This is an ambitious goal, and CIRM acknowledges the importance of fostering a new regulatory path for stem cell therapies.

Current NIH standards for informed consent and human subjects research do not address specific challenges related to clinical trials involving complex stem cell–based biologic products. Unlike drugs and many medical devices, transplanted progenitor cells have the potential to integrate and proliferate within their human recipient and as a result may be difficult to remove if necessary. Transplanted cells can last for the lifetime of the recipient and cause deleterious effects that are difficult to ameliorate. In light of the complexity and novelty of new stem cell–based biologics—many of which may not be directly analogous to local, well-characterized donor tissue transplants or drug therapies—all stem cell–based clinical trials research raises crucial ethical concerns. Given the nature of stem cell–based therapies, safety and clinical proof-of-principle studies are likely to involve



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