San Francisco, CA – California’s stem cell agency, the California Institute for Regenerative Medicine (CIRM) today approved $150 million in new funding to help move promising stem cell-based therapies from the laboratory research phase to clinical trials in people.
For Huntington’s disease the funding is enormously important in helping researchers at UC Davis do the most advanced clinical study to date for this untreatable and uniformly fatal disease.
“People are hopeful, truly hopeful for the first time,” says Judy Roberson, a Huntington’s disease patient advocate. “This is a nightmarish, cruel disease in every way but now, thanks to CIRM, we are turning the dream of a stem cell therapy trial into a reality. Research means hope for people with this disease, but research costs money. CIRM has given us all hope.”
The grants, up to $20 million per applicant, go to teams of researchers in both academia and industry who have been working on projects that represent the best possible chances of producing therapies for deadly and disabling diseases and disorders. Those diseases include Huntington’s disease, metastatic melanoma, osteoporosis, critical limb ischemia, spinal cord injury, ALS (Lou Gehrig’s disease) and cardiovascular disease.
“Everything we do in this innovative Disease Team Program is focused on getting good science converted to productive treatments for patients,” says Alan Trounson, PhD, President of CIRM. “These awards reflect and highlight our commitment to identifying the most promising stem cell research and supporting it for the time needed to show both the safety and effectiveness of therapy, with an ultimate goal of producing a new treatment that is approved by the FDA for clinical application.”
The funding is part of the stem cell agency’s Disease Team II awards which are designed to encourage multidisciplinary teams of researchers from academic institutions, medical centers and industry to work together and to develop new treatments for a broad range of therapies. The recipients were selected from 21 applications, all of which were reviewed by an independent group of internationally renowned scientists
“This round of funding highlights how we try to support promising research all the way through from basic science to clinical trials,” says Jonathan Thomas, PhD, JD, Chair of the Governing Board of CIRM. “For example, Antoni Ribas at UCLA was previously awarded a New Faculty award by CIRM (http://www.cirm.ca.gov/content/stem-cells-immune-system-regeneration-fight-cancer), and now he has been given a Disease Team award to further advance his work in metastatic melanoma.
In the case of five awards—DR2A-05735 addressing heart disease, DR2A-05426 addressing muscular dystrophy, DR2-05416 addressing Alzheimer’s disease, DR2-05352 addressing breast cancer and DR2-05739 addressing retinitis pigmentosa—the Board determined that there was enough new information to merit sending the applications back for further expert analysis. The results of that analysis will be brought back before the Board for its consideration at a later date.
The teams that are being given the funding are expected to file a request to begin clinical trials or to complete phase I or II clinical trials within four years. Five of the teams propose to finish a clinical trial within the period of the award.
Two of CIRM’s 20 collaborative funding partners around the world will also be contributing to this round of projects. An investigator at the National Institutes of Health will be a partner Principal Investigator with the University of California, Los Angeles team developing a therapy for metastatic melanoma; and the Andalusian Initiative for Advanced Therapies in Spain will be providing $1.6 million to researchers there to collaborate with the team at UC Davis working on a therapy for limb ischemia.
The board also approved the fourth Research Leadership Award to foster the recruitment of Andrew McMahan from the Harvard Stem Cell Institute to the University of Southern California’s Broad Center for Regenerative Medicine and Stem Cell Research. He plans to use the $5.7 million award to study ways to repair and regenerate kidney tissue.
CIRM funds late-stage research projects moving toward potential therapies in 37 diseases. The full list and descriptions of the projects can be found on the CIRM website.
Disease Team Therapy Development Awards
|DR2A-05415||Vicki Wheelock||MSC engineered to produce BDNF for the treatment of Huntington’s disease||University of California, Davis||$18,950,061|
|DR2A-05309||Antoni Ribas||Genetic Re-programming of Stem Cells to Fight Cancer||University of California, Los Angeles||$19,999,563|
|DR2A-05302||Nancy Lane||Treatment of osteoporosis with endogenous Mesenchymal stem cells||University of California, Davis||$19,999,867|
|DR2A-05423||John Laird||Phase I study of IM Injection of VEGF-Producing MSC for the Treatment of Critical Limb Ischemia||University of California, Davis||$14,184,595|
|DR2A-05736||Nobuko Uchida||Neural stem cell transplantation for chronic cervical spinal cord injury||StemCells, Inc.||$20,000,000|
|DR2A-05394||Robert Robbins||Human Embryonic Stem Cell-Derived Cardiomyocytes for Patients with End Stage Heart Failure||Stanford University||$19,999,899|
|DR2A-05320||Clive Svendsen||Progenitor Cells Secreting GDNF for the Treatment of ALS||Cedars-Sinai Medical Center||$17,842,617|
|DR2A-05365||Judith Shizuru||A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants||Stanford University||$20,000,000|
Research Leadership Awards
|LA1-06536||Andrew McMahon||Repair and regeneration of the nephron||University of Southern California||$6,718,471|
Disease Team Project Descriptions
Huntington’s Disease: Vicki Wheelock, University of California, Davis
This research team plans to use bone marrow derived mesenchymal stem cells to deliver a growth factor to patients’ damaged and endangered nerves. The factor they have chosen, called BDNF, has been shown to be effective in laboratory studies in reducing nerve cell death and improving the function of nerves. During this project they will finalize laboratory tests and begin a phase 1 clinical trial in patients to test the safety of the approach. More information about Huntington’s disease.