Silence ALS is a new effort that will create tailored experimental medicines for patients with uncommon hereditary variants of ALS (commonly known as Lou Gehrig's disease). Patients with the most uncommon gene mutations are the focus of the effort. Each mutation is thought to affect anywhere from one to thirty persons around the world.
The Silence ALS programme, which focuses on individuals identified through Columbia University's ALS Families Project—a study of pre-symptomatic carriers of ALS-associated gene mutations—aims to treat patients with antisense oligonucleotides early in the course of their disease, ideally before onset of symptoms.
The project, which was founded by Columbia University and the n-Lorem Foundation with a $400,000 funding from Target ALS, would provide patients with the cutting-edge medicines for free for the rest of their lives.
There are very few therapy choices for ALS patients, and none of them significantly modify the disease's natural course."
Neil Shneider, MD, PhD, co-founder of Silence ALS and head of the Eleanor and Lou Gehrig ALS Center at Columbia University Vagelos College of Physicians and Surgeons
The majority of ALS cases are caused by unknown reasons, however genetic mutations that trigger the generation of motor neuron-toxic proteins account for 10% to 15% of cases.
Antisense oligonucleotides (ASOs), which are small pieces of DNA or RNA that attach to RNA and inhibit it from generating harmful proteins, are one intriguing treatment option for people with hereditary types of ALS.
Some ASOs are now being tested in persons with more common genetic types of ALS, but they are not yet available for people with uncommon or unique mutations.
Related Articles
A healthy diet is linked to a lower risk of diabetes at all levels of hereditary risk.
Women with the highest hereditary risk of diabetes benefit from more intensive lifestyle treatments.
The genetic diversity of C. difficile is studied using a systems biology approach.
"Silence ALS focuses on patients with the rarest of gene mutations, which impact between one and thirty persons worldwide and for whom industry-sponsored studies are not an option," Shneider explains. "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, with the goal of developing ASOs that are specific to each patient and beginning treatment before symptoms appear."
Columbia University will use Silence ALS to identify pre-symptomatic people with unusual or unique ALS mutations and collaborate with n-Lorem to create individualised experimental ASO medications to treat them.
Stanley Crooke, MD, PhD, founder and CEO of n-Lorem and co-creator of Silence ALS, states, "Silence ALS will provide the most efficient mechanism to connect the diagnosis and treatment of people with some of the exceedingly uncommon genes that cause ALS." "I'm especially excited to collaborate with Dr. Shneider at Columbia, who has extensive expertise finding and treating patients with extremely uncommon ALS mutations, so that we can provide hope and treatment to individuals with these rare types of ALS earlier in the disease's course."
"We hope to have the first of these novel ASOs available to our patients soon," Shneider says.
Posts
No comments:
Post a Comment