Amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig’s disease, is a devastating neurological disease of the motor nervous system. Within a few short years, its victims fall from good health—often in the prime of life—and ultimately perish due to progressive motor neuron deterioration. ALS is surprisingly common: people have a lifetime risk of about 1 in 400.
Prior investigation led by Brian Wainger, MD, PhD, (Wainger et al., Cell Stem Cell, 2014) has identified abnormalities in the electrical activity of motor neurons derived from ALS patients using stem cell technology. The research culminated in the discovery of the FDA-approved drug retigabine (ezogabine) as a candidate therapeutic, and we are now investigating this drug in a clinical trial of ALS subjects.
CHRONIC PAIN RESEARCH
Chronic pain does not have the same lethal impact as ALS; however, any sufferer of chronic pain can testify to the profound impairments in quality of life, mood and functioning that plague pain patients. Chronic pain affects over one quarter of adult Americans and is one of the most common reasons for physician visits, lost productivity and disability.
Ongoing work by Dr. Wainger has yielded a technique for deriving pain-sensing neurons from patient skin samples (Wainger et al., Nature Neuroscience, 2015). This novel method may offer a way to investigate causes of pain in human patients, thus potentially overcoming the limits of animal models, which have resulted in only limited success in identifying effective treatments for human pain. The goal is to use the human pain-sensing neurons to identify and evaluate novel treatments for pain in patients.
The combination of specialized clinical and research training places the group in a prime position to investigate disease-related research questions and find practical and promising ways to directly advance the application of basic science research to clinical medicine.