Jeff Vierstra Gets Experimental ALS Treatment for Rare Genetic Mutation
Jeff Vierstra has been getting experimental spine infusions every few months for three years to target a genetic mutation that makes him likely to develop ALS. The treatment at Columbia University aims to disable the mutated gene before the disease starts.
Jeff Vierstra receives experimental infusions directly into his spine every few months, targeting a genetic mutation that puts him at high risk for developing ALS. The treatment has continued for three years at Columbia University.
The experimental drug, called ulefnersen, works by disabling specific genetic mutations before they can cause ALS symptoms. Dr. Neil Shneider at Columbia is leading the research, testing the treatment on 12 patients with rare genetic forms of ALS.
About 10% of ALS cases are inherited through family genes. People like Vierstra know they carry these mutations but have had no way to prevent the disease until now. The treatment uses antisense oligonucleotides, which are designed molecules that can turn off specific genes.
Shneider launched the Silence ALS program in 2022 with the n-Lorem Foundation to create these targeted treatments for patients with genetic mutations. The research represents a shift from treating ALS after symptoms appear to preventing it entirely.
This could be the first treatment to prevent ALS before symptoms appear in people with genetic mutations. About 10% of ALS cases are inherited, affecting thousands of families who know they carry deadly genes.
Researchers will track whether the spine infusions successfully prevent ALS symptoms in Vierstra and other patients with genetic mutations.
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