Stem Cells Could Provide Promising Treatment for Lou Gehrig's Disease (ALS)

Amyotrophic lateral sclerosis (ALS), more commonly known as Lou Gehrig's disease, has puzzled medical experts since 1939 when it became a famous case in the United States after Lou Gehrig was diagnosed that year. Its cause and cure are unknown.

ALS is a progressive neurodegenerative disease that affects nerve cells, particularly motor neurons, in the brain and spinal cord. When these motor neurons are affected, they die and the brain loses its ability to command muscle movement. Those in the later stages of ALS may become completely paralyzed.

Since its first report in 1874 issued by French doctor Jean-Martin Charcot, there is only one drug that helps slow the progression of ALS. There are no treatments that halt or cure the disease. The FDA-approved Riluzole helps slow the disease's aggressive progression, but now Harvard stem cell scientists are collaborating with Massachusetts General Hospital to test the safety of a new clinical trial.

The Harvard researchers discovered a recently approved medication for epilepsy that could be an effective treatment for Lou Gehrig's disease. Kevin Eggan and researchers at the Harvard Stem Cell Institute found that the seizure drug, Potiga (retigabine), "tempers the hyperexcitability of neurons derived from induced pluripotent stem (iPS) cells made from patients who have ALS," as stated in this PLOS.org blog.

Mr. Eggan first raised the possibility of using ALS patient-derived stem cells to conduct further research into the mysterious disease back in 2008. Prior treatments were promising in mice, but they were disappointing in clinical trials, which led Mr. Eggan to seek ways to identify therapeutic targets for new drugs.

"The big problem in ALS is that there are more than 100 mutations in dozens of genes that all cause the disease, but almost all of the therapeutics that have gone forward in the clinic have done so for just one of those mutations, SOD1, which almost everyone studies in mice," he said in this Harvard Gazette news article.

Eggan and his coworkers' findings can be found in this Cell Stem Cell publication with results from their clinical trials conducted on neurons in laboratory dishes. They hope to conduct clinical trials to test for side effects in ALS patients very soon.