n-Lorem Foundation and Columbia University Establish ALS Initiative

The nonprofit foundation n-Lorem said it has joined with Columbia University to form Silence ALS, an initiative to support the characterization of nano-rare amyotrophic lateral sclerosis (ALS) patients and the discovery and development of personalized experimental antisense oligonucleotide (ASO) medicines for these patients for free, for life.

The initiative, backed with a founding $400,000 donation from the medical research foundation Target ALS, creates an infrastructure that employs n-Lorem’s nonprofit model to discover and develop personalized investigational ASO medicines for nano-rare ALS patients. Additionally, the initiative utilizes Columbia University’s resources to conduct a comprehensive clinical and scientific evaluation of pre-symptomatic ALS patients with the ultimate goal of identifying and treating nano-rare ALS patients prior to symptom onset.

ALS is a fatal neurological disorder caused by the degeneration of motor neurons in the brain and spinal cord. People with ALS rapidly lose muscle strength and eventually their ability to move, swallow, and breathe. In at least 10 to 15 percent of ALS cases, the disease is caused by a mutation in one of a large and growing number of ALS-associated genes. Silence ALS targets those nano-rare ALS patients who have a genetic mutation found in only 1 to 30 affected individuals worldwide.

“Despite great progress in our understanding of the genetics and biology of ALS, no therapy is available today that alters the natural history of the disease in a meaningful way,” said Neil Shneider, director of the Eleanor and Lou Gehrig ALS Center and the Claire Tow Associate Professor of Motor Neuron Disorders in the Department of Neurology at Columbia University, Vagelos College of Physicians and Surgeons. “Through our partnership with n-Lorem, Silence ALS aims to have a significant impact on the lives of nano-rare ALS patients, and to generate findings relevant to all patients and families afflicted with this devastating disease.”

Manish Raisinghani, CEO of Target ALS, said the partnership is an extension of its collaborative approach and part of its vision to find treatments for all forms of ALS. The organization will initially support the initiative by funding a portion of the costs associated with preclinical toxicology for two ALS programs. Over time, Raisinghani said Target ALS hopes to expand its participation to support clinical trial work and to analyze biosamples collected during the course of treatment.

“The Silence ALS initiative aligns our strengths with a world-renowned healthcare institute and a novel nonprofit to bring hope and potentially lead to the development of new therapeutics for these patients,” he said. “In short, this effort has the potential to make an immediate positive impact for patients with nano-rare forms of ALS while advancing understanding of the disease biology overall.”

Author: Rare Daily Staff