Institute for Regenerative Medicine
The California Institute for Regenerative Medicine (CIRM) is the organization responsible for providing state government funds and grants for advancing stem cell research and regenerative medicine. It was established as the major product of California Proposition 71, otherwise known as the California Stem Cell Research and Cures Initiative.
Stem cell research in the United States has been a controversial issue since the 1990s, after the development of methods to culture human embryonic stem cells from the inner cell mass of early embryos and gonadal ridges of aborted fetuses. In response to these developments, Congress amended laws for regulating the National Institutes of Health with a provision known as the Dickey Amendment. The amendment prohibited use of federal funds to be used for the “creation of human embryos for research purposes, or research in which human embryos are destroyed, discarded, or knowingly subjected to risk of injury or death. . . . ” In 2001, the George W. Bush administration issued a rule limiting federal funding for research involving human embryonic stem cells, including funding for the National Institutes of Health (NIH).
The creation of California Proposition 71 was a response to this federal policy, to promote funding for stem cell research at the state level when federal funds were nonexistent. It was passed in 2004 with the approval of 59 percent of California voters. Robert N. Klein Jr. is a well-known advocate and proponent of Proposition 71. Proposition 71 established the CIRM and secured the right to conduct stem cell research within the state of California. The act ultimately allocated $3 billion to the CIRM to spend over a period of 10 years. Proposition 71 also created the governing body of the Independent Citizens’ Oversight Committee (ICOC) to oversee CIRM administration and financing. The ICOC also coordinates the three policy-setting working groups created by the initiative: Scientific and Medical Research Funding, Scientific and Medical Research Facilities, and Medical Accountability Standards. Proposition 71 also detailed a specific organizational model requiring that the ICOC be comprised of 27 members from specific organizations, such as California’s medical schools, research institutes, and pharmaceutical and biotechnology companies.
CIRM Initial Funding
The first wave of grants established by CIRM focused on development of stem cell research infrastructure, including the development of laboratory facilities, research spaces, and training of stem cell research scientists and faculty. The beginning research grants focused on the basic sciences to build knowledge and understanding surrounding stem cells, with most of the research fueled toward embryonic stem cells but also toward induced pluripotent stem cells and adult stem cells.
In 2008 and 2009, CIRM expanded its research focus from basic sciences to the exploration of therapeutic stem cell applications. Since then, CIRM has focused on stem cell derived treatments for a variety of human diseases, with most of the funds going to neurological disorders, cardiovascular disease, blood and immune disorders, and cancer. Since its founding, CIRM has provided approximately $600 million in funding for 87 therapy-focused studies for the treatment of various diseases, which has resulted in 11 therapeutic clinical trials, with more to come in the future.
In 2012 and in 2013, CIRM committed up to a combined $340 million dollars in order to advance early clinical development of stem cell–based therapies. These awards were funded to support up to 17 promising groups through the preclinical and clinical development of their therapies. The main objective was to complete a Phase I or Phase II clinical trial in four years or less under an Investigational New Drug (IND) filed with the Food and Drug Administration.
In the fall of 2013, CIRM launched the first ever bank for induced pluripotent stem cells (iPSCs) for disease modeling and drug discovery for genetically complex diseases. Several other notable CIRM-funded projects include the Geron trial for spinal cord repair, the first clinical trial to test the use of human embryonic stem cells for treatment in 2009, clinical trials for the treatment of macular degeneration and vision loss, a collaborative study with ViaCyte biotechnology company for the treatment of type 1 diabetes using embryonic stem cell-derived glucose-responsive insulin producing cells, and much more.
CIRM Present Initiatives
As of 2014, CIRM is currently funding over 50 projects developing novel therapies for various diseases. Eleven of these projects are currently in Phase I/II clinical trials, treating diseases including but not limited to Beta-thalassemia, osteoporosis, malignant melanoma, diabetes, and amyotrophic lateral sclerosis (ALS). More than 1,800 research papers have been published as a result of CIRM funding, 24 percent of which are in high-impact scientific journals. CIRM has helped to build 12 state-of-the-art research facilities, attracted 130 new stem cell researchers and scientists to California, and 26 projects will continue on into clinical trials to create new therapies for the future.
In February 2014, CIRM approved a $40 million proposal led by a seven-member consortium from Stanford University to create a new Center of Excellence in Stem Cell Genomics. The new center will focus on the creation of medical treatments for diseases based on the genetic makeup of the patient. In March 2014, a Center of Excellence was started for stem cell genomics to advance application of genomics and bioinformatics to support this project.
CIRM also committed up to $200 million in funding through an Accelerated Development Pathway award for projects already funded by CIRM and conducting clinical research using a stem cell–based therapy. The award aims to provide additional financial support to groups making rapid progress in order to accelerate their cell-based therapies and complete a Phase I and/or Phase II clinical trial by 2017. As it is evident by its awards, CIRM is putting great emphasis on accelerating promising therapies through the early stages of clinical trials by the end of 2017.
In March 2014, CIRM also declared a new $40 million Preclinical Development Award initiative, supporting projects already receiving CIRM funding to accelerate readiness for FDA approval. This includes plans to support stem cell manufacturing needed for therapy trials and testing to ensure quality of stem cell production.
Future of CIRM
The initial $3 billion allocated to CIRM through Proposition 71 is expected to last until 2017. As of April 2014, CIRM has made no official statement on how it plans to continue current and future stem cell research projects, but Robert Klein presented in February 2014 a proposal at the University of California, San Diego Moores Cancer Center to extend CIRM funding. The proposal aims to introduce an initiative for an additional $5 billion in funding to CIRM on the 2016 California ballot.
By the end of 2014, the CIRM plans to launch the Alpha Stem Cell Clinics Network, a localized network for all clinical trials utilizing delivery of regenerative therapies.
Doniel Drazin
Pablo Avalos
Leslie Suen
Cedars-Sinai Medical Center
See Also: California; California Stem Cell, Inc.
Further Readings
Adelson, J. W. “The California Stem Cell Initiative: Persuasion, Politics, and Public Science.” American Journal of Public Health, v.100/3 ( 2010).
Feigal, E. G. and N. D. Dewitt. “The Proceedings of the California Stem Cell Agency.” Stem Cells Translational Medicine (April 25, 2014).
Fikes, B. “$5B Initiative Proposed for Stem Cell Research.” UTSanDiego.com (February 2014.) http://www.utsandiego.com/news/2014/Feb/20/robert-klein-cirm-stem-cell-billion-2016 (Accessed June 2014).
McCormack, K. “Stem Cell Agency Funds More Than $66 Million in Research Including New Genomics Initiative.” CIRM News Room (January 2014). http://cirm.ca.gov/about-cirm/newsroom/press-releases/01292014/stem-cell-agency-funds-more-66-million-research