The California Institute for Regenerative Medicine Science, Governance, and the Pursuit of Cures (2013) / Chapter Skim
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4 Nature, Scope, and Accomplishments of the CIRM Scientific Program
4 Nature, Scope, and Accomplishments of the CIRM Scientific Program
Pages 75-104
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From page 75... ...
It then presents an analysis of the Institute's grant management system, as well as the bioethics- and industry-related challenges that lie ahead for clinical applications of stem cell research. As discussed in Chapter 1, it was outside of the scope of the committee's work to rigorously evaluate the details of CIRM's scientific contributions, specific grant awards, or impact on the field of regenerative medicine.
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From page 76... ...
The goals during this initial phase were to develop appropriate laboratory facilities for stem cell research, to fund basic research in stem cell biology, to invest in programs directly focused on research on a broad range of diseases, and to establish a long-term foundation for California leadership in stem cell research and development (CIRM, 2006a)
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From page 77... ...
Hence CIRM was authorized to spend up to 10 percent of its research budget on new facilities that could then operate independently of federal stem cell legislation. CIRM also focused heavily on developing the manpower necessary to sustain a longterm capacity in stem cell research and development in California.
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From page 78... ...
. The committee appreciates this concern, because CIRM would be well served to take the fullest advantage of the special skills and opportunities these collaborators can provide to its joint ventures.3 Given the state of science in regenerative medicine, the 2006 5-year strategic plan, and CIRM's 10-year funding horizon, the committee believes it made good sense to begin with investments in basic research; in physical infrastructure (especially given the matching funds that were required and mobilized by each institution awarded a major facilities grant and the requirement that construction be completed with a rapid timeline)
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From page 79... ...
, with the goal of eventually enabling or at least moving toward Phase 1/2 clinical trials. The Disease Team Research Awards have now emerged as fundamental to CIRM's core ongoing mission.
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From page 80... ...
The 2012 Strategic Plan In 2012, CIRM developed a new strategic plan outlining 10 goals that build on and extend those of the 2006 plan. The 2012 5-year plan increases the priority of projects clearly focused on moving toward clinical trials to produce evidence of therapeutic benefit and articulates the importance of developing partnerships with both industry and other centers for research in regenerative medicine (CIRM, 2012a)
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From page 81... ...
. The goal is to advise CIRM on how to make its programs attractive to industry, identify research areas most appropriate for industry, identify CIRM-funded inventions that should be patented, create opportunities for follow-on funding for CIRM-funded research approaching clinical trials, assist CIRM in fostering industry-academic partnering opportunities, and identify and advance business models for regenerative medicine (CIRM, 2012a)
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From page 82... ...
UCLA: Genetic Re-Programming of Stem Cells to Fight Cancer This latest round of disease team awards brings the total funding for this program to roughly $360 million, and CIRM-supported late-stage research projects now address 37 different disease areas (CIRM, 2009d, 2012f)
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From page 83... ...
The committee also suggests that CIRM continue making its scientific staff available to potential applicants and working with this constituency to maximize the effectiveness of this aspect of the grant submission process. CIRM staff recognized that the number of applications that would potentially be received for a given RFA could overwhelm the Institute's ability to review each rigorously for scientific merit.
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From page 84... ...
. The committee agrees that, despite its limitations, the current pre-application procedure opens up the opportunity for CIRM funding to a broader cohort of investigators and is, in principle, an appropriate process.
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From page 85... ...
Participation of these experts, none of whom, as non-Californians, is eligible for CIRM funding and stand to gain directly from CIRM, is instrumental in providing the rigorous scientific review required for making funding decisions. The success that CIRM has had in commissioning outstanding review committees for each of its RFAs is a testament both to the Institute's stature in the eyes of the stem cell community and the willingness of stem cell scientists outside of California to contribute their time and effort to facilitate the work of their California colleagues Full proposals received by CIRM by the RFA deadline are entered into the CIRM database, and all GWG members assigned to this review cycle declare any conflicts of interest with any of the applications (CIRM, 2009g)
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From page 86... ...
For an application to be moved from one tier to another, a majority vote of the GWG is required; all members of the GWG not in conflict (scientists and patient advocates) participate in this vote.
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From page 87... ...
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.
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From page 88... ...
Although the committee applauds CIRM and the Standards Working Group for taking the initiative to address these important emerging issues in the ethical conduct of human stem cell research, the drafting of sample consent forms for iPS cell research and banking could have been aided greatly by empirical studies examining the most effective ways to bolster patient-informed consent during the consent interview process -- studies the Standards Working Group did not sponsor or utilize. Such efforts at policy development should continue in other areas of stem cell science supported through CIRM funding.
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From page 89... ...
governing 6 California Stem Cell Research and Cures Initiative, Proposition 71 (2004) (codified at California Health and Safety Codes § 125291.10-125291.85)
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From page 90... ...
. Approximately 15 for-profit entities have received CIRM grants and loans, totaling around $80 million, covering a broad spectrum of research and development topics: basic research, translation, tools and technologies, disease teams, and clinical trials.
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From page 91... ...
The 2012 strategic plan calls for proof-of-principle animal models for more than 10 diseases (Goal IV) and for 10 therapies in Phase 1 or 2 clinical trials in at least five different therapeutic areas based on stem cell research (Goal VIII)
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From page 92... ...
The committee and solicited experts note that the approaches of several of the disease teams do not fit neatly into what is generally considered stem cell research; rather, they are extensions of more conventional therapeutic strategies not tied to CIRM's basic stem cell research portfolio. This observation is not meant as a criticism of the validity of these efforts or the quality of these disease teams or as denial of the importance of developing these technologies to counter these illnesses.
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From page 93... ...
The goal of the creation of alpha stem cell clinics is to establish a stem cell therapy clinical infrastructure with the requisite scientific, technical, and medical expertise, combined with operational efficiencies, to foster clinical trials, to evaluate and establish safe and effective therapies, and to develop and maintain the delivery of therapies approved by the FDA or other regulatory agencies (CIRM, 2012a; Trounson et al., 2012)
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From page 94... ...
However, the committee believes this step requires more careful planning. These facilities, providing a site for clinical trials of stem cell therapies, would house multidisciplinary activities, cell production capabilities, and trained personnel in a setting attractive to industry involvement.
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From page 95... ...
Going forward, however, all aspects of application review, funding recommendations, and grant administration should be the sole responsibility of the CIRM scientific staff, reporting to the president. While the ICOC should remain responsible for ultimate approval of grants, it should not be empowered to act on individual applications.
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From page 96... ...
The group should continue to include scientists outside of California with expertise in stem cell science and regenerative medicine, with ad hoc scientific reviewers continuing to participate as needed, still as nonvoting members. The committee also believes it is important for patient advocates to continue to be involved in the application review process, in addition to their participation in the ICOC and its decisions about which RFAs to announce and the level of funding for each.
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From page 97... ...
The ICOC should remain responsible for oversight and articula tion of an overall strategic plan. However, grant management, funding recommendations, and grant administration should be the responsibil ity of the CIRM scientific staff, reporting to the president.
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From page 98... ...
Bioethics Given the speedy timeline and the scientific and regulatory complexities entailed in the goal of bringing stem cell research to clinical trials within the next 5 years, the committee recommends that CIRM sponsor projects and offer new grant opportunities aimed specifically at identifying and addressing ethical and regulatory issues surrounding stem cell–based clinical trials research. CIRM should use the information resulting from these initiatives to strengthen its ethical standards for human subjects research.
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From page 99... ...
While the Standards Working Group created and operationalized many rigorous ethical standards for basic stem cell research during CIRM's early years, it has not been equally productive of late in formulating CIRM policies for the ethical conduct of human clinical trials research. The Standards Working Group should be encouraged and empowered to focus on this unmet need.
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From page 100... ...
Other ethical aspects of stem cell research need attention besides ethical issues in stem cell–based clinical trials. These areas include (but are not limited to)
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From page 101... ...
to stimulate investment in emerging and existing regenerative medicine companies by engaging the investment community in California, one of the most sophisticated venture capital communities in the world. CIRM has created an exemplary training program and seeded a pipeline of intellectual property and translational projects that are primed for industry involvement, outside funding, and unique therapy delivery mechanisms.
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From page 102... ...
2008b. RFA 08-04: CIRM bridges to stem cell research awards.
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From page 103... ...
2011c. CIRM approves $27 million for initiatives to accelerate promising stem cell research projects.
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From page 104... ...
IOM Committee on a R eview of the California Institute for Regenerative Medicine Subcommittee Meeting with Boston Stem Cell Scientists on June 26, 2012, Boston, MA. Trounson, A., N
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