New Initiative to Develop Personalized Therapies for People with Rare Genetic Forms of ALS

May 10, 2022

Focusing on individuals identified through Columbia University’s ALS Families Project—a study of pre-symptomatic carriers of ALS-associated gene mutations—the Silence ALS program aims to treat patients early in the course of their disease, ideally before onset of symptoms, with drugs called antisense oligonucleotides (ASOs).

The initiative, established by Columbia University and the n-Lorem Foundation with a founding grant of $400,000 from Target ALS, will offer the unique therapies to patients for free, for life.

“There are very few therapeutic options for patients with ALS and none that alter the natural course of the disease in a meaningful way,” says Neil Shneider, MD, PhD, director of the Eleanor and Lou Gehrig ALS Center at Columbia University Vagelos College of Physicians and Surgeons and co-founder of Silence ALS. “Silence ALS focuses on patients with the rarest of gene mutations, which each affect between 1 and 30 people worldwide—and for whom industry-sponsored programs are not an option.

“With the goal of developing ASOs that are specific to each patient and beginning treatment before symptoms appear, Silence ALS will provide the infrastructure to rapidly identify patients with very rare ALS mutations and design therapies that may delay or prevent disease onset or slow its progression once it has begun.” [read more]

Source: CUIMC Newsroom