The governing board of California’s stem cell agency approved 13 grants totaling $40.6 million for research intended to identify, then advance into clinical trials potential therapies from basic research in prostate cancer, heart disease, liver disease, autism, and HIV/AIDS.
The Independent Citizens Oversight Committee (ICOC) approved grants for investigators based at 10 institutions under the Early Translational IV Research awards program of the California Institute for Regenerative Medicine (CIRM). The program is designed to fund research that will result in the development of new potential drug or cell therapies, or make what CIRM deems “significant” strides toward such therapies.
"The strategies are focused on problems where we think there is a very reasonable chance that they will evolve into clinical studies for treating some of the worst diseases we have in the community,” CIRM president Alan Trounson, Ph.D., said in a statement.
Of the 13 grants, the largest was the nearly $6.4 million awarded to John R. Cashman, Ph.D., president and founder of Human BioMolecular Research Institute. Dr. Cashman’s team told CIRM it will use patient-derived human induced pluripotent stem cell-cardiomyocytes toward developing a safer drug envisioned to treat the human genetic heart disorder Long QT syndrome type 3.
The institution capturing the largest share of the $40 million in total grants was the Eli & Edythe Broad Center of Regenerative Medicine and Stem Cell Research at University of California, Los Angeles, where four researchers won a combined roughly $13 million.
The largest of the four UCLA grants went to Jerome Zack, Ph.D., professor of medicine and microbiology, immunology and molecular genetics. His research team will use an approximately $5.3 million award – second-largest of the 13 grants – to engineer blood-producing stem cells designed to create T cells capable of eliminating HIV-infected cells, to be given to patients through a bone marrow transplant. UCLA’s next largest grant at about $4.1 million was won by Robert E. Reiter, M.D., to develop a monoclonal antibody targeting castration-resistant prostate cancer stem cells, with the goal of long-term remissions through possibly eliminating the cancer stem cells responsible for recurrent disease.
Among other winners of early translational awards:
- Dan Gazit, Ph.D., D.M.D., director of the Skeletal Regeneration and Stem Cell Therapy Laboratory at Cedars-Sinai Medical Center, won $5.3 million – the third largest of the 13 early translational grants – toward using gene-modified stem cells to help speed up healing in segmental bone fractures.
- Magdalene J. Seiler, Ph.D., of UC Irvine won $4.3 million toward creating sheets of retinal cells to repair damage from both age-related macular degeneration and retinitis pigmentosa.
In addition to UC Irvine, UCLA, Cedars-Sinai, and HBRI, the 10 winning institutions included one business, Numerate, as well as Stanford University, UC Davis, UC San Diego, The J. David Gladstone Institutes, Sanford-Burnham Medical Research Institute, and The Salk Institute for Biological Studies.
The early translational grants mark the latest large investment for CIRM, which is funded through $3 billion in bonds approved by California voters when they passed Proposition 71 in 2004.
In July, the agency approved a $70 million plan to create the Alpha Stem Cell Clinics Network, a new statewide network of sites that will act as a hub for stem cell clinical trials. In May, CIRM awarded $36 million in funds to attract six scientists to the state, and more than $6 million to create a partnership with Sangamo BioSciences to develop a therapy for the blood disorder beta-thalassemia. And in March, the organization announced a plan to set up a stem cell bank and approved nine applications to generate the cells to fill it.