Researchers get $51.7 million in state grants to develop stem-cell-based therapies

- By Krista Conger

Irving Weissman

Irving Weissman

Three teams headed by researchers at the Stanford University School of Medicine today received $51.7 million from the California Institute for Regenerative Medicine to develop FDA-approved therapies within the next four years for acute myeloid leukemia, the lethal skin-blistering disease epidermolysis bullosa and ischemic stroke. It is the largest amount Stanford has received in a single round of funding from CIRM.

The teams are headed by Irving Weissman, MD; Alfred Lane, MD; and Gary Steinberg, MD, PhD, respectively. The Stanford grants were part of a total of $250 million awarded today by CIRM's 29-member governing board to 14 multidisciplinary teams in California, Canada and the United Kingdom. This is the agency's largest funding round since its inception.

The Disease Team research awards are designed to quickly bring promising therapies to patients through teams that include basic scientists, clinicians and industry. CIRM President Alan Trounson, PhD, said in a statement that the pace of the Disease Team projects stands in contrast to the decade or more that's usually required to reach clinical trials. "Scientists have talked for years about the need to find ways to speed the pace of discovery," he said. "By encouraging applicants to form teams composed of the best researchers from around the world, we think CIRM will set a new standard for how translational research should be funded."

"We are gratified that CIRM has funded our application to develop an antibody therapy for human acute myeloid leukemia," said Weissman, whose team will receive $20 million over the next four years. He is the director of Stanford's Institute for Stem Cell Biology and Regenerative Medicine. "The funding will help us advance our preclinical finding that the AML cancer stem cells express a 'don't eat me' signal called CD47 that protects them from the immune system. Blocking this signal with an antibody results in the elimination of these cells and is a promising avenue to human therapy."

Together with Weissman, co-principal investigators Ravindra Majeti, MD, PhD, assistant professor of hematology, and Beverly Mitchell, MD, director of Stanford's Cancer Center, will collaborate with partners in the United Kingdom to conduct coordinated basic research, clinical studies and the development of pre-clinical therapeutics leading to a phase-1 clinical trial of a stem cell-targeted antibody therapy for the disease. Weissman and Majeti are also members of Stanford's Cancer Center.

Lane, professor and chair of dermatology, heads a team that will receive $11.7 million to use stem cell therapy to treat a devastating genetic skin condition called epidermolysis bullosa, or EB. People with a version of the condition called dominant dystrophic EB suffer severe blistering and sloughing of the skin that is usually lethal by young adulthood. The team will use patient-specific induced pluripotent stem, or iPS, cells to correct the genetic defect that causes the disease.

"The Stanford group has been working on EB for the past 15 or 16 years, and this is a very exciting next iteration of our work" said co-principal investigator Anthony Oro, MD, an associate professor of dermatology specializing in skin differentiation and development. "We feel that we're ready to take the treatment of this disease to the next step. We have an amazing group of people working on this disease." The international group includes Stanford stem cell expert and co-principal investigator Marius Wernig, MD, as well as Paul Khavari, MD, PhD; Peter Marinkovich, MD; Howard Chang, MD, PhD; and Seung Kim, MD. Khavari, Marinkovich, Chang and Kim are also members of Stanford’s Cancer Center.

Steinberg, professor and chair of neurosurgery, heads a team focused on ischemic stroke. His team will collaborate with co-principal investigator and neurologist Stanley Carmichael, MD, PhD, from the University of California-Los Angeles. They will receive $20 million to investigate ways to use neural stem cells derived from human embryonic stem cells to ameliorate motor deficits that may arise when parts of the brain are deprived of oxygen when a blood vessel becomes blocked by a clot or an artery narrows-perhaps extending the window for effective treatment of the condition from hours to weeks or months after the incident.

In addition to the three teams headed by Stanford faculty, Stanford microbiologist and immunologist Garry Nolan, PhD, is a co-principal investigator on a $20 million grant awarded to oncologist Dennis Slamon, MD, PhD, at UCLA. The team, which includes co-principal investigator Michael Press, MD, PhD, an oncologist at the University of Southern California, and researchers in Canada, will work to develop new ways to destroy cancer stem cells in solid tumors. Nolan is also a member of Stanford's Cancer Center.

To date, Stanford has received $163 million in funds from the state institute, more than any other institution.

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