Gene Therapy Shows Promise in Slowing ALS Progression and Restoring Function
Translated & summarized from Behadrei Haredim by baba
A new gene therapy, Qalsody, is showing positive results for a small group of ALS patients with a specific genetic mutation. At Ichilov Medical Center, about half of six treated patients experienced improved muscle strength and regained lost functions. International studies also indicate that earlier treatment leads to slower physical decline. Experts suggest wider genetic testing could help identify patients for this promising therapy.
The story in 5 lines · by baba
- Gene therapy Qalsody shows promise in slowing ALS progression and restoring lost functions.
- About 50% of treated ALS patients at Ichilov Medical Center showed improved strength and function.
- International studies suggest earlier treatment with Qalsody leads to slower physical decline.
- The therapy targets a specific SOD1 gene mutation found in about 2% of ALS cases.
- Experts recommend expanded genetic testing for early identification of eligible patients.
A targeted gene therapy has demonstrated success in slowing the progression of Amyotrophic Lateral Sclerosis (ALS) and, in some cases, restoring lost functions, according to recent reports. The treatment, known as Qalsody, is designed for a small subset of patients carrying a specific genetic mutation.
ALS is a neurodegenerative disease that affects nerve cells controlling muscles, leading to progressive weakness in limbs and impairing speech, swallowing, and breathing. While most ALS cases are sporadic, approximately 2% are linked to a mutation in the SOD1 gene, for which this therapy was developed. Qalsody works by reducing the production of a faulty protein that damages nerve cells and is administered via spinal fluid injection.
At Ichilov Medical Center, six patients are currently receiving the treatment. Data from the center indicates that about half of these patients have shown improvement in muscle strength and a return of lost functional abilities. Dr. Tehila Shich-Segev, head of the ALS clinic at the hospital, described these outcomes as a significant development in a field where functional improvement was previously considered rare.
"In the last two years, we have evidence, both from our center and from around the world, that the drug can lead to improved strength and the return of motor functions in some patients," she stated. "This is something we were not accustomed to seeing in this disease, and it is very significant." She recalled one patient who joyfully reported being able to load dishes into the dishwasher for the first time in a year.
International research involving 108 patients also supports these findings. A long-term study showed that participants who began treatment earlier experienced a slower decline in physical function, muscle strength, and respiratory capacity compared to those who started later. However, the article notes that not all differences were statistically significant, and further data is needed to fully establish the treatment's impact. Experts emphasize that expanding access to genetic testing could enable earlier identification of suitable patients, offering them a better chance to preserve their abilities.