FUS-associated ALS

Interferon-gamma therapy may help delay familial ALS: Cell study

People with amyotrophic lateral sclerosis (ALS) due to mutations in the FUS gene may benefit from interferon-gamma treatment to delay disease onset and/or progression, an early preclinical study suggests. Researchers found that an immune signaling protein called interferon (INF)-gamma protected patient-derived motor neurons from oxidative stress. INF-gamma also restored…

Early stress pathway activation combats cell death in FUS-ALS

Early activation of certain cellular stress response pathways may help prevent the death of motor neurons in amyotrophic lateral sclerosis (ALS) patients with FUS mutations, according to a recent study. In particular, heat shock response (HSR) pathways and the integrated stress response (ISR) were increased in patient-derived motor neurons…

Ionis Opening Phase 3 Trial of ION363, Antisense Therapy for FUS-ALS

Ionis Pharmaceuticals is opening a Phase 3 safety and efficacy trial of ION363 (jacifusen) in amyotrophic lateral sclerosis (ALS) patients with confirmed mutations in the FUS gene, a known cause of juvenile-onset disease. The trial (NCT04768972) will enroll up to 64 people, ranging in age from 12 to 65,…

Collaboration Funds Experimental Therapy for Rare FUS-ALS

Eight patients will receive jacifusen, an experimental therapy for amyotrophic lateral sclerosis (ALS) caused by FUS gene mutations, under a joint effort from the ALS Association, Project ALS, and Columbia University’s Eleanor and Lou Gehrig ALS Center. The new clinical research program, which will be supported…