Phase 3 study shows positive results for ulefnersen in genetic form of ALS
There is encouraging news for people with FUS-ALS, a rare genetic form of ALS. The Phase 3 FUSION study has shown positive results for ulefnersen, an investigational treatment that specifically targets the underlying genetic cause of FUS-ALS.
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FUS-ALS is caused by changes in the FUS gene and accounts for approximately 0.6 per cent of all ALS cases. It often affects people at a younger age and can progress rapidly. Currently, there is no approved treatment specifically for FUS-ALS.
Ulefnersen is designed to reduce the production of the harmful FUS protein, which can build up in motor neurons and damage these nerve cells. The treatment is given through a lumbar puncture, allowing it to reach the central nervous system directly.
In the international FUSION study, people receiving ulefnersen had significantly better outcomes compared with those receiving placebo. The results showed a positive effect on daily functioning and survival: people receiving ulefnersen lived longer or were able to go longer without needing permanent ventilation.
“This is very exciting news. Ulefnersen builds on the development of genetically targeted treatments such as tofersen for SOD1-ALS and provides further hope that similar approaches could be developed for other genetic forms of ALS. These findings are an important step towards more personalized medicine for people living with ALS,” says Caroline Ingre, research group leader of ALS research at the Department of Clinical Neuroscience, and founder of the clinical treatment center of ALS, specialising in ALS clinical trials.
Ulefnersen is not yet an approved treatment. Ionis Pharmaceuticals and Otsuka Pharmaceutical are planning to discuss the results with regulatory authorities and work towards making the treatment available to patients. It is not yet known when this may happen.