The Institute has been awarded a $6.6 million grant – the largest single award in the latest competition -- by the California Institute Regenerative Medicine (CIRM) for research aimed at translating basic science into clinical cures. The funds are part of $67.7 million Early Translational Grants CIRM provided to 15 research organizations in April.
Led by senior scientists Inder Verma and Juan Carlos Izpisúa Belmonte, the research will focus on developing treatments for Fanconi anemia and X-Linked Severe Combined Immunodeficiency (X-SCID), more commonly known as the "bubble boy" disease. Both are diseases of the blood and each is caused by the mutation in a single gene.
Using pioneering gene therapy and stem cell reprogramming techniques developed by Verma and Belmonte, they will work with mouse models that mimic each of the diseases. The human hematopoietic (blood precursor) stem cells used in their work are derived from patient hair follicle cells that have been induced into a pluripontent stem cell state (iPS) and corrected of its defective gene. The genetically corrected cells will then be coaxed back into the cells that form the blood and immune systems and used for transplant therapy.
"Working with human patient and disease-specific cells will help us demonstrate the feasibility and evaluate the safety in a pre-clinical setting to advance these techniques, which combine the latest developments in regenerative medicine and gene therapy," said Verma, a professor in the Laboratory of Genetics. "This work will also benefit the successful stem cell-based therapies for many other diseases like Parkinson's and diabetes."
"The opportunity to work with human cells from patients with these diseases will further demonstrate why we believe these cells are perfect candidates for transplantation therapy," said Belmonte, a professor in the Gene Expression Laboratory. "The chances of rejection are drastically reduced when the cells are derived from the patients themselves."
The 15 Early Translational grants approved by the CIRM's board were awarded to 13 not-for-profit and two for-profit organizations. They are intended to either lead to a drug candidate for an unmet medical need or address a bottleneck in the development of new therapies.
"With these Early Translational grants CIRM has taken the first step in funding translational research that will be critical for the development of future therapies," said Alan Trounson, CIRM president. "These grants are an important part of CIRM's strategy to fund the best basic research and then bring the results of that work to patients."