$2.3 million in stem cell research money given to Stanford labs to study Duchenne muscular dystrophy

From The Stanford Daily at Stanford University:

A $2.3 million grant this fall from the California Institute of Regenerative Medicine (CIRM) is the most recent installment of stem cell research money poured into Stanford labs.

Since 2004, Stanford has received about $175 million from CIRM, according to the School of Medicine. In late October, the CIRM awarded genetics professor Michele Calos $2.3 million for her work on Duchenne muscular dystrophy (DMD). The grant was part of a $67 million effort to mobilize stem cell research in California.

One of the most common and serious forms of muscular dystrophy, DMD affects one in every 3,500 newborn boys. This disease causes irreversible, widespread muscle degeneration and is, in most cases, fatal. Mutation of a vital muscle protein, dystrophin, is one of the key causes of DMD. Absence of this muscle-maintaining protein causes weakening of the muscles in the hips, pelvic area, thighs and shoulders.

“To begin to approach a therapy for this condition, we must provide a new supply of stem cells that carry the missing protein that is lacking in DMD,” Calos said. “These cells must be delivered to the body in such a way that they will engraft in the muscles and produce new, healthy muscle tissue on an ongoing basis.”

Like most retrogressive diseases, DMD eventually leads to destruction of most of the muscles in the body. DMD is caused by an x-linked recessive inheritance, and patients with DMD typically die before the age of 30.

Calos hopes a cure for DMD will stem from a therapy that could correct this muscle mutation by reprogramming skin and fat cells into new, healthy stem cells.

“We start with mice that have a mutation in the same gene that is affected in DMD,” Calos said. “We reprogram some of their adult cells, add the correct gene and grow the cells in incubators in a manner that will produce muscle stem cells.”

Calos and her team then evaluate the effect of transplanting these corrected muscle stem cells into DMD mice and the mice’s ability to generate new muscle tissue.

Medical School spokeswoman Krista Conger praised the efforts of Calos’s team and their mouse model development.

“If successful,” Conger said, “the researchers will develop standard procedures to test and ensure the safety of all cells for eventual use in humans in DMD and possibly other degenerative disorders.”

And the funding provided by CIRM will certainly aid Calos’s efforts. Established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act, CIRM has provided $3 billion in funding to California universities and research institutions to date.

With the opening of the nation’s largest stem cell center, The Lorry I. Lokey Stem Cell Research Building, last month, the Stanford School of Medicine is continuing to make strides in its stem cell research efforts. The 200,000-square-foot building will house more than 600 scientists hoping to collaborate with one another.

Irving Weissman, director of the Stanford Stem Cell Biology and Regenerative Medicine Institute, said of the building, “What is important is that it will give people the opportunity to apply stem cell thinking to different problems, including regeneration, aging and cancer.”

While Calos’ research is not yet at a stage to involve clinical trials, she hopes her mouse research will provide the insight necessary to begin work in humans.

“In order to make this process into something that could be used in the clinic, we will develop standard procedures for making and testing the cells to ensure that they are effective and safe,” Calos said. “In this way, this project could lead to a new stem cell therapy that could improve the clinical condition of DMD patients.”