“As is often the case, I had a lot of great 40,00-foot view conversations with Doug Melton. When we worked next door to one another, he would wander into my lab and say things like, ‘why don’t we make disease specific stem cell lines?’” Eggan and his colleagues created the first patient-specific cell lines, which they generated from adult patients with the genetic muscle-wasting disease amyotrophic lateral sclerosis (ALS). In a collaboration with HSCI’s Clifford Woolf and Brian Wainger, Eggan studied the functional properties of motor neurons affected by ALS and discovered that an FDA-approved anti-epileptic drug, retigabine, lessened the impact of the disease in the cells. A Phase II clinical trial will test the effects of the drug in ALS patients.
