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Family's ALS Curse Broken by Gene Therapy Trial
5 Apr
Summary
- Experimental gene therapy targets mutated FUS gene linked to ALS.
- Treatment shows promise in preventing ALS onset in high-risk individuals.
- Research offers hope for making ALS a manageable, non-fatal disease.
Jeff Vierstra, whose mother and two sisters succumbed to Amyotrophic Lateral Sclerosis (ALS), is participating in a groundbreaking experimental treatment aimed at preventing the disease. His family carries a mutation in the FUS gene, which is crucial for nerve cell function and increases the likelihood of developing ALS.
This experimental therapy, administered via spinal infusions over the past three years, targets and disables the mutated FUS gene. While Vierstra's sisters, who also received the treatment, eventually developed ALS complications and passed away, Vierstra believes the therapy extended their lives. He himself has not developed the disease, and his muscle test abnormalities have normalized.
Neurologists leading the clinical trial at Columbia University's Eleanor and Lou Gehrig ALS Center are optimistic. They believe this research represents a significant step towards making ALS a liveable, rather than fatal, condition. The insights gained from studying familial forms of ALS, like Vierstra's, could pave the way for treatments for other rare genetic forms of the disease.
