State stem cell grant will fund muscular dystrophy work

Michele Calos

Michele Calos, PhD, professor of genetics at the School of Medicine, has been awarded $1.9 million from the California Institute for Regenerative Medicine to develop a stem cell-based therapy for a muscle disease called limb-girdle muscular dystrophy type 2B.

The award was part of $40 million distributed today to researchers at 10 institutions to enable the rapid movement of promising, basic stem cell science out of the laboratory and into clinical applications.

"These awards are moving discoveries into the clinical pipeline for patients," CIRM president Alan Trounson, PhD, said in a prepared statement. "The strategies are focused on problems where we think there is a very reasonable chance that they will evolve into clinical studies for treating some of the worst diseases we have in the community."

The grants represent the fourth 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. (In 2010, Calos received $2.3 million in the second round of the awards to develop a stem cell therapy for Duchenne muscular dystrophy.)

With the new grant, Calos will use induced pluripotent stem cell technology to create muscle stem cells carrying a healthy copy of the gene that is mutated in human patients with limb-girdle muscular dystrophy. She will then assess the ability of the stem cells to engraft and generate new muscle in mice with a form of the disorder.

In her proposal, Calos said that if the therapy proves successful, "similar methods could be used to treat other degenerative disorders, and perhaps even some of the degeneration that occurs during muscle injury and normal aging."

With this award, Stanford has received about $277 million from the stem cell agency.

CIRM was established in November 2004 with the passage of a statewide ballot measure that provided $3 billion in funding for stem cell research at California universities and research institutions.