The California Institute for Regenerative Medicine today granted cardiothoracic surgeon Robert Robbins, MD, $20 million to lead a team of researchers in an investigation of the use of human embryonic stem cell-derived heart muscle cells in patients with end-stage heart failure, and $20 million to oncologist Judith Shizuru, MD, PhD, to develop an antibody-based method to deplete diseased or dysfunctional blood and immune stem cells in patients with severe combined immunodeficiency. In the video above, cardiologist and co-investigator Joseph Wu, MD, PhD, describes the research proposed in the first grant.
From our release:
“This will be first time anyone has implanted cells derived from human embryonic stem cells into a human heart,” said Robbins, professor and chair of cardiothoracic surgery and director of Stanford's Cardiovascular Institute. “We’re excited to assess this potential treatment for patients who currently have very few options other than heart transplant.”
Robbins and his colleagues hope to move into human clinical trials within four years, after first testing the transplantation approach extensively in animal models. They plan to enroll about 10 patients in the proposed phase-1 trial.
The two four-year awards were part of CIRM's second round of disease-team grants. All told, the agency awarded $150 million to eight teams. Stanford investigators received $57.1 million during the first disease-team award round in October 2009.
More from our release about the Shizuru award, which builds on previous work from Stanford's Irving Weissman, MD:
The Shizuru grant focuses on the use of a monoclonal antibody previously tested in mice by Weissman’s group to remove, or deplete, diseased or dysfunctional blood and immune system stem cells in patients with severe combined immunodeficiency. Currently these cells must be killed by high-dose chemotherapy or radiation, a process that itself can be life-threatening, prior to transplantation with healthy donor cells. Shizuru and her collaborators plan instead to use an antibody that will specifically recognize and eliminate the faulty cells without the use of toxic treatments, enabling patients to more readily accept cells from a healthy donor.
If shown to be effective, the technique could potentially been used to treat many other types of diseases, including sickle-cell anemia and cancers of the blood cells such as leukemias and lymphomas.
Previously: Stem cell-based heart-attack therapy approved for clinical trials, Lab-made heart cells mimic common cardiac disease in Stanford study and Nature News examines CIRM's public funding uncertainties