New ALS Treatment Offers Hope to Israeli Man with Genetic Mutation
Translated & summarized from Ynet by baba
Tarek Murad, an Israeli man diagnosed with ALS, is now receiving a new genetic treatment called Qalsody (tofersen) that has shown promise in slowing the disease's progression and, in some cases, restoring lost function. The treatment targets a specific SOD1 gene mutation, offering hope to patients with this form of the disease.
The story in 5 lines · by baba
- New ALS drug Qalsody offers hope to Israeli patient Tarek Murad.
- Murad has a rare genetic form of ALS caused by a SOD1 mutation.
- The treatment aims to halt disease progression by reducing toxic protein.
- Early results show slowed progression and potential for functional recovery.
- Research is exploring preventative treatment for mutation carriers.
Tarek Murad, a 56-year-old father of three from Beit Jann, Israel, has shared his challenging journey after being diagnosed with Amyotrophic Lateral Sclerosis (ALS) in 2022. The former bank employee and IDF reservist first noticed subtle leg twitches in 2022, which gradually progressed to a limp and then significant weakness in his limbs. The diagnostic process was lengthy and difficult, involving numerous specialists and tests over several months before ALS was suspected.
Murad's condition worsened, impacting his ability to perform daily tasks like counting money or opening doors. He was eventually diagnosed with ALS, a progressive neurodegenerative disease that affects motor neurons. The diagnosis was devastating, especially after reading about the disease's typically short life expectancy. He also faced a nearly year-long wait for an appointment at a specialized ALS clinic, eventually securing an earlier slot through connections.
A turning point came in March 2025 when Dr. Tahani Sheich-Sakar, his neurologist at Ichilov Medical Center, informed him that a genetic test revealed he had a specific genetic form of ALS. This opened the door to a new treatment, Qalsody (tofersen), designed for patients with a SOD1 gene mutation. Murad described this news as "winning the lottery" and began the treatment in May 2025.
While the drug does not cure ALS, it aims to halt its progression by reducing the production of a toxic SOD1 protein. After initial skepticism, Murad reports that over a year into treatment, he feels the disease's progression has slowed. Dr. Sheich-Sakar explained that Qalsody is a revolutionary treatment that targets the root cause of SOD1-ALS and has shown promising results in clinical trials, with some patients experiencing stabilization or even a return of lost motor function.
Murad acknowledges that he knows "the end is near" but finds hope in the new treatment. He chose to share his story to offer hope to other ALS patients. The article also highlights ongoing research, including a study testing Qalsody in individuals who carry the SOD1 mutation but have not yet developed symptoms, aiming to prevent or delay the onset of the disease.