Tofersen reverses symptoms for some patients with genetic ALS
Updated
Updated · The New York Times · May 6
Tofersen reverses symptoms for some patients with genetic ALS
8 articles · Updated · The New York Times · May 6
Amanda Sifford, 58, in Florida, said the drug improved her breathing after her lung function had fallen to 48% from 86% in five months.
The FDA approved tofersen in May 2023 as the first treatment for a genetic form of ALS, despite clinical trials not yet proving it worked.
The milestone offers hope for families affected by inherited ALS, though the benefits described appear limited to some patients rather than all people with the disease.
This breakthrough A.L.S. therapy targets a single gene. Can this approach unlock cures for other incurable brain diseases?
The first gene therapy for A.L.S. helps only 2% of patients. What hope does it offer the other 98%?
Long-Term VALOR Trial Shows Tofersen Stabilizes or Improves Function in 25% of SOD1-ALS Patients Over Nearly 3 Years
Overview
Tofersen, an antisense oligonucleotide therapy, significantly reduces toxic SOD1 protein in patients with SOD1-associated ALS, leading to less motor neuron damage. This reduction slows disease progression, reflected by lower neurofilament light chain levels, and results in functional stabilization or improvement in about 25% of patients over nearly five years. Early treatment enhances benefits, including slower decline in muscle strength and respiratory function, and notably extends survival in faster-progressing cases. While tofersen offers a transformative shift in ALS care, its administration carries risks of serious neurological side effects and procedural discomfort. Access remains limited by genetic requirements, cost, and regional disparities, highlighting ongoing challenges despite its groundbreaking impact.