Experimental Drug May Benefit Some Patients with Rare Form of ALS

Neil Shneider, MD, PhD, director of the Eleanor and Lou Gehrig ALS Center at Columbia University, talks about the surprising and promising results from 12 volunteer Amyotrophic Lateral Sclerosis (ALS) patients treated with experimental drug, ulefnersen (previously known as jacifusen). “These responses show that if we intervene early enough and go after the right target at the right time in the course of disease, it's possible to not only slow disease progression, but actually reverse some of the functional losses,” Dr. Shneider says. “It’s also a wonderful example of precision medicine and therapeutic development based on science and an understanding of the biology of disease.” The 12 participating patients have a rare genetic mutation in the FUS gene that causes an aggressive form of ALS. Two of the patients undergoing treatment have shown remarkable results. In one case, the person regained the ability to walk independently and breathe without a ventilator, and another person who was asymptomatic but whose test indicated that the symptoms would appear soon has remained symptom free. Several other patients have also shown the benefits of undergoing this treatment. [read more]

Source: CUIMC Newsroom