New initiative focuses on patients with the rarest of gene mutations to develop personalized ALS therapies

A new initiative, Silence ALS, will develop experimental personalized therapies to treat patients with rare genetic forms of ALS (also known as Lou Gehrig's disease). The initiative focuses on patients with the rarest of gene mutations. Each mutation is thought to affect between 1 and 30 people worldwide.

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.

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 history of the disease in a meaningful way."

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

Most ALS cases arise from unknown causes, but at least 10% to 15% of cases are caused by genetic mutations that lead to the production of proteins that are toxic to motor neurons.

One promising approach to treating patients with genetic forms of ALS uses antisense oligonucleotides (ASOs)-;short pieces of DNA or RNA-;that bind to RNA and prevent it from making the toxic proteins.

Some ASOs are currently undergoing testing in people with more common genetic forms of ALS, but these therapies are not available for people with unique or extremely rare mutations.

"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," Shneider says. "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."

Through Silence ALS, Columbia University will identify pre-symptomatic individuals with unique or nano-rare ALS mutations and work in partnership with n-Lorem to develop personalized experimental ASO medicines to treat them.

"Silence ALS will create the most efficient process to connect the diagnosis and treatment of patients with some of the extremely rare mutations that cause ALS," says Stanley Crooke, MD, PhD, founder and CEO of n-Lorem and co-founder of Silence ALS. "I am particularly pleased to work closely with Dr. Shneider at Columbia, who has significant experience in identifying and treating patients with very rare ALS mutations, so that we can bring hope and treatment to patients with these rare forms of ALS earlier in the course of the disease."

"We are hoping to offer the first of these new ASOs to our patients rapidly," Shneider says.

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