A New York scientist living with a rare inherited mutation is getting regular spinal gene silencing infusions in an attempt to stop ALS before it ever shows up. The treatments, given every few months over the past three years, have, by his account, reversed earlier mild muscle signal abnormalities and kept him free of evident symptoms so far. Clinicians say the case is an early test of whether targeted antisense therapy might turn a fatal genetic prognosis into a more manageable condition.
As reported by CBS News, the patient, 41-year-old scientist Jeff Vierstra, lost his mother and two sisters to ALS and later tested positive for a mutated FUS gene that runs in his family. After examining Vierstra and seeing mild abnormalities on an electromyography (EMG) test, Dr. Neil Shneider of Columbia's Eleanor and Lou Gehrig ALS Center offered experimental intrathecal infusions. According to CBS News, Vierstra's EMG readings returned to normal within a year and he has not developed ALS symptoms while staying on the regimen.
Silence ALS and personalized gene therapy
Columbia has launched an initiative known as Silence ALS to build individualized antisense oligonucleotide (ASO) medicines that target ultra rare ALS mutations, with the aim of treating people as early as possible, ideally even before symptoms appear, and at no cost to qualifying patients. The program, co-founded by Dr. Shneider and the n-Lorem Foundation and backed by grants from research funders, sets up a faster path to design an ASO tailored to a single patient's mutation and move it toward first-in-human testing. This custom approach is intended to reach extremely small patient groups that commercial drug developers typically do not serve, according to Columbia University Irving Medical Center.
Early signals, wide caveats
Researchers and patient advocates stress that the evidence so far is very early, even as some presymptomatic patients have shown improved muscle signal readings after treatment. The ALS Association has praised Dr. Shneider's work and the Silence ALS model as a promising way to reach patients with ultra rare mutations, according to The ALS Association. Clinicians say much more safety data and time are needed before anyone can say whether these individualized ASOs can reliably prevent disease onset.
How antisense medicines work and what's next
Antisense oligonucleotides are short strands of nucleic acid that attach to a gene's RNA messages and dial down production of proteins that can be toxic to motor neurons. Columbia recently received a $15 million URGenT grant to speed up individualized ASO development and notes that Silence ALS's first custom ASO led to an FDA cleared program for a different ultra rare mutation, setting a regulatory precedent for future bespoke drugs. Even so, researchers caution that turning one promising case into a prevention strategy that many people can access will require more patients, extensive toxicology work, and formal trials, per Columbia University Irving Medical Center.
What this means for families
Gene targeted, presymptomatic treatment offers guarded hope to families facing hereditary ALS, but it is still experimental and is unlikely to be widely available any time soon. The Centers for Disease Control and Prevention's National ALS Registry projects about 34,720 people living with ALS in the U.S. in 2026, a reminder of the disease's overall scale even as the genetic subtypes targeted by ASOs remain rare. Families who are considering genetic testing or looking for trial options are advised to talk with their neurologist, and specialty clinics and research networks continue working to identify patients who may qualify for individualized programs.
