MDA Hosts Workshop on Stem Cell Therapy For MD

Place some stem cells in a petri dish, add the right mixture of growth factors, and they'll transform into a panoply of cell types, from liver cells to muscle and nerve cells.

Gather up dozens of stem cell researchers, mix in a few clinicians, plus scientists from the biotechnology industry, and the results are a bit less predictable. The hope is that they'll hash out a plan for using stem cells to treat muscular dystrophy.

This is the goal of MDA's upcoming stem cell workshop, to be held near MDA's national headquarters in Tucson, Ariz., on March 8 and 9.

Stem cells are primitive cells that assemble the tissues of the body during embryonic life and have the potential to regenerate damaged tissues during adult life. Scientists hope to use the cells to repair muscles damaged by muscular dystrophy, perhaps by transplanting them from a healthy donor (an allogeneic transplant) or by removing them from the patient, correcting them via gene therapy, and reimplanting them where they're needed (an autologous transplant).

While both approaches have shown promise in laboratory experiments, there's little consensus as to how to proceed with human trials. What would be more effective: an allogeneic transplant or an autologous transplant? What kinds of stem cells should be used and how many? How should the cells be delivered?

Those questions will be hammered out by some 30 scientists from around the world during the workshop, co-chaired by MDA Director of Science Technology Donald Wood and geneticist Louis Kunkel of Children's Hospital in Boston. Kunkel identified the defective gene underlying Duchenne muscular dystrophy in 1986, and recently showed that stem cells administered by a bone marrow transplant procedure could partially repair muscles in mice with Duchenne.

On Friday, an all-star line-up of stem cell researchers — including Johnny Huard of Children's Hospital in Pittsburgh, featured in the current issue of Quest — will present their latest findings. On Saturday, experts on stem cells, muscular dystrophy and transplantation will hold a series of roundtable discussions on how to move stem cells into clinical trials.

This week and next, be sure to check this site for updates from the workshop.