New hope for treating ALS
Patient stem cells help identify common problem, leading to clinical trials
Harvard stem cell scientists have discovered that a recently approved medication for epilepsy might be a meaningful treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, a uniformly fatal neurodegenerative disorder. The researchers are now collaborating with Massachusetts General Hospital (MGH) to design an initial clinical trial testing the safety of the treatment in ALS patients.
The investigators all caution that a great deal of work needs to be done to assure the safety and efficacy of the treatment in ALS patients before physicians should start offering it.
The work, laid out in two related advance online publications in April by Cell Stem Cell and Cell Reports, is the long-term fruit of studies by Harvard Stem Cell Institute (HSCI) principal faculty member Kevin Eggan, who in a 2008 Science paper first raised the possibility of using ALS patient-derived stem cells to better understand the disease and identify therapeutic targets for new drugs.
