Phase 3 Pridopidine Trial for ALS Begins Enrollment
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.
📌 Key Facts
- Columbia University’s Eleanor and Lou Gehrig ALS Center is testing an experimental antisense‑style treatment that targets a rare FUS gene mutation with the goal of preventing ALS onset in high‑risk carriers.
- The treatment is delivered via periodic spinal infusions.
- Patient Jeff Vierstra had early EMG abnormalities that were interpreted as possible very early disease; those abnormalities normalized after a year of the infusions and he remains free of ALS symptoms three years into treatment.
- Lead neurologist Dr. Neil Shneider described the work as a "very big deal" and said researchers hope to make ALS a "liveable" rather than inevitably fatal disease.
- Columbia’s Silence ALS initiative is working to develop individualized gene‑based therapies for other rare genetic forms of ALS.
📊 Relevant Data
FUS mutations account for approximately 3.2% of familial ALS cases, representing the second most common gene abnormality after SOD1.
U.S.-based ALS clinical trials significantly under-enroll non-White participants, with trends toward slight underrepresentation of women.
Participation in U.S.‐Based ALS Clinical Trials by Sex and Race — PMC
More than 26% of Black ALS patients in the U.S. are diagnosed before age 50, compared to 12.9% of White patients.
Racial Disparities in the Diagnosis and Prognosis of ALS Patients in the United States — Springer
Lower ALS mortality rates among U.S. non-Hispanic Black, Hispanic, and other non-White groups compared to Whites may be due to genetic differences.
Race/ethnicity, socioeconomic status, and ALS mortality in the United States — Neurology
📰 Source Timeline (2)
Follow how coverage of this story developed over time
- Describes an experimental antisense‑style treatment at Columbia University’s Eleanor and Lou Gehrig ALS Center targeting a rare FUS gene mutation in an effort not just to treat but to prevent ALS onset in a high‑risk carrier.
- Reports that patient Jeff Vierstra had early EMG abnormalities interpreted as possible very early disease, then saw those abnormalities normalize after a year of periodic spinal infusions, and remains free of ALS symptoms three years into treatment.
- Quotes lead neurologist Dr. Neil Shneider calling the work a "very big deal" and expressing hope of making ALS a "liveable" rather than inevitably fatal disease, and notes Columbia’s Silence ALS initiative to develop individualized gene‑based therapies for other rare genetic forms of ALS.