Israeli patients have become among the first in the world to start receiving a new ALS drug intended to slow the progression of the degenerative disease.
Tofersen, which is injected into the spine as an epidural, gives new hope to two patients at Tel Aviv Sourasky Medical Center. One of them, Narkis Moshe from Nahariya, lost a brother to ALS, and was later diagnosed with the disease six months ago. She said she was less afraid of her fate now that she had started taking drugs.
“When I was diagnosed with the disease, I was very scared because I didn’t want what happened to my brother to happen to me,” said Moshe, 58. “But it seems that today we are entering a new era of research and treatment.
“I am very excited about the new treatment and hope it will give me a good quality of life and many years with my family.”
Tofersen, developed by Massachusetts-based Biogen, is not suitable for all ALS patients because the disease has different underlying causes; rather, it is designed to help patients whose disease is caused by a faulty SOD1 gene.
The drug has been cleared for a “compassionate use” program by the Food and Drug Administration, which allows it for specific patients before full regulatory approval. Israel tends to follow the United States in drug regulation and therefore allows Sourasky to use the drug.
Professor Vivian Dror, Sourasky’s doctor administering the treatment, said that with only a few dozen patients worldwide receiving the drug so far, she is delighted that Israel is becoming one of the first to adopt.
“It is a potentially revolutionary treatment,” she told The Times of Israel. “Patients receiving it are still communicative, able to swallow and with some use of their limbs, and it is hoped that the drug will slow their deterioration and enable them to retain these abilities for longer.
“What it does is significant compared to the limited treatments that exist today for this disease.”
There are different underlying causes favoring the development of ALS in different patients. Tofersen is designed specifically for those who suffer from hSOD1 overproduction. It is a protein produced by everyone, but when produced in excess, it seems to have toxic effects on the motor neurons of the disease. A defect in the SOD1 gene, which produces hSOD1, is the cause of the problem.
Tofersen works by inhibiting the production of hSOD1, attempting to compensate for the effect of the SOD1 defect.
Dror said that although only a small minority of ALS cases are caused by the SOD1 gene, she believes the underlying technology behind Tofersen will be suitable for patients who suffer from other genes.
“It is important to note that this is a technological breakthrough not only for patients with this specific SOD1 defect, but also for other patients, because with the same technology it is possible to develop and to create specific drugs for additional gene defects,” she added. said. “It gives us hope.”