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Topic: ALS Research and Treatment

ALS Research and Treatment

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Mainstream coverage this week focused on two developments: a Phase 3 trial of pridopidine has begun enrolling people with ALS to test whether it slows progression, and Columbia University clinicians reported promising early results from an experimental antisense‑style, intrathecal therapy targeting a rare FUS mutation — including one pre‑symptomatic carrier whose EMG abnormalities normalized and who remains symptom‑free three years into treatment — and the expansion of Columbia’s Silence ALS program to develop individualized gene‑based therapies.

Missing from much of the reporting were important context and equity concerns: FUS mutations are relatively rare (about 3.2% of familial ALS and second only to SOD1 among known genes), so the Columbia case may not generalize to most ALS patients; long‑term safety, durability and access to intrathecal gene therapies remain unclear. Independent sources also highlight chronic under‑representation of non‑White patients in U.S. ALS trials (historically ~95% Caucasian participants), earlier age at diagnosis among Black patients (≈26% diagnosed before 50 vs 12.9% of White patients), differences in prevalence by race (U.S. prevalence ~5.0 per 100,000 in Whites vs 3.4 in Black individuals), and a high estimated heritability for sporadic ALS (~61%), all of which bear on who benefits from new treatments and how trial results should be interpreted. No strong contrarian viewpoints were identified in the materials reviewed.

Summary generated: April 10, 2026 at 11:02 PM

Phase 3 Pridopidine Trial for ALS Begins Enrollment

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A Phase 3 clinical trial of pridopidine has begun enrolling people with ALS to test whether the drug can slow disease progression. Separately, Columbia University researchers report promising early results from an experimental antisense‑style therapy targeting a rare FUS mutation — including one patient whose EMG abnormalities normalized and who remains symptom‑free three years later — prompting optimism from Dr. Neil Shneider and supporting Columbia’s Silence ALS initiative to develop individualized gene‑based treatments.

Columbia Neurologists Test Preventive Gene Therapy in Familial ALS Carrier

A CBS News report profiles 41‑year‑old scientist Jeff Vierstra, a U.S. man from a family ravaged by a rare FUS gene mutation that causes early‑onset ALS, who has spent the past three years receiving experimental spinal infusions at Columbia University in what his neurologist calls the first known attempt to prevent ALS in a high‑risk carrier. Led by Dr. Neil Shneider at Columbia’s Eleanor and Lou Gehrig ALS Center, the treatment uses an agent that targets and disables the mutated FUS gene; after early EMG tests showed mild abnormalities suggestive of incipient disease, Vierstra’s muscle‑signal readings normalized within a year and he remains free of ALS symptoms, even as his two sisters, who also received the therapy after symptom onset, ultimately died of the disease. The piece notes that only 10–15% of ALS cases are genetic but about two‑thirds of those are familial, and that an estimated 35,000 Americans live with ALS, underscoring why researchers see pre‑symptomatic gene‑based interventions as a potential pivot from fatal prognosis to chronic management. Shneider cautions that the work is still experimental but calls it "a very big deal" and says his team is expanding efforts through Silence ALS, a program seeking patients with other rare genetic forms of ALS for individualized gene‑targeted therapies, a development already stirring cautious hope and questions about access among U.S. ALS families and advocacy groups online.