Researchers at Harvard Stem Cell Institute (HSCI) found that independent genetic mutations that trigger ALS could be linked by their ability to abnormally high activity in motor neurons. The researchers used neurons derived from stem cells made from ALS patient skin cells and conducted clinical trials test whether the anti-epilepsy drug retigabine was effective and found that it was able to reduce the cells' hyperexcitability.
The researchers have now partnered with Massachusetts General Hospital (MGH) and hope that they will be able to come up with an initial clinical trial to test the safety of the treatment in ALS patients.
"It looked like there's a deficit in potassium channels in the ALS motor neurons, and that led us to then test whether drugs that open the potassium channels may reduce this hyperexcitability. And indeed that's exactly what we found. We found that retigabine, which has recently been approved as an anticonvulsive, normalized this activity. So now we can formally go from the dish to the patient and actually explore whether the drug might have any beneficial effect", HSCI's Clifford Woolf said.