CIRM awards researcher $2.3 million to develop stem cell therapy for muscular dystrophy

Michele Calos

Michele Calos, PhD, professor of genetics at the Stanford University School of Medicine, has received $2.3 million from the California Institute for Regenerative Medicine to develop a stem cell-based therapy for Duchenne muscular dystrophy. The award was part of $67 million distributed today by the institute to enable the rapid movement of promising basic stem cell science out of the laboratory and into clinical applications.

The grants represent the second round of the institute’s Early Translational Awards, which are expected to either result in or make significant strides toward a candidate drug or cell therapy for human disease. The ultimate goal of the awards is to develop therapies for submission to the Food and Drug Administration for approval for use in clinical trials. The institute expects to award such grants on a 12- to 18-month cycle.

“This second round of Early Translational Awards will strengthen CIRM’s portfolio of future therapies,” said CIRM president Alan Trounson, PhD, in a statement distributed by the institute. “We are looking for ways to complement our leading edge of stem cell-based treatments for patients and these projects will load our frontline portfolio with promising studies on autism, muscular dystrophy, Canavan disease and liver disease. These projects will enhance the potential medical options available for patients and hopefully in the near future produce cures for such debilitating handicaps and diseases.”

Calos received the award to investigate how to reprogram adult cells from skin or fat to become muscle-generating stem cells expressing the protein missing in people with Duchenne muscular dystrophy, the most common childhood form of the disease. Calos and her lab members have developed a mouse model of the disease in which to conduct their experiments; they will then pursue a similar strategy in cells from patients with DMD. If successful, the researchers will develop standard procedures to test and ensure the safety of the cells for eventual use in humans with DMD and possibly other degenerative disorders.

With these grants, Stanford has now received a total of about $175 million from CIRM.

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure provided $3 billion in funding for stem cell research at California universities and research institutions and required setting up the agency, CIRM, to oversee allocation of the money.