Israeli patients have become some of the first in the world to begin receiving a new ALS drug intended to slow the progress of the degenerative disease.
Tofersen, which is injected into the spine like an epidural, is giving new hope to two patients at Tel Aviv Sourasky Medical Center. One of them, Narkis Moshe of Nahariya, lost a brother to ALS, and was then diagnosed with the disease six months ago. She said that she is less scared about her fate now that she has started with the drug.
“When I was diagnosed with the disease I was very afraid because I didn’t want what happened to my brother to happen to me,” said Moshe, 58. “But it seems that today we’re entering a new era of research and treatment.
“I am very excited about the new treatment and I am hopeful that it will give me a good quality of life and many more years with my family.”
Tofersen, developed by Massachusetts-based Biogen, isn’t suitable for all ALS patients, as the disease has different underlying causes; rather, it is designed to help patients whose illness is cause by a faulty SOD1 gene.
The drug has received the green light for a “compassionate use” program from the Food and Drug Administration, which permits it for specific patients in advance of full regulatory approval. Israel tends to follow the US in drug regulation, and is therefore allowing the drug to be used at Sourasky.
Prof. Vivian Dror, the Sourasky doctor who is administering the treatment, said that with only a few dozen patients worldwide receiving the drug so far, she is pleased that Israel is becoming an early adopter.
“It’s a potentially game-changing treatment,” she told The Times of Israel. “The patients receiving it are still communicative, able to swallow and with some use of their limbs, and it’s 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 promoting the onset of ALS in different patients. Tofersen is designed specifically for those who suffer as a result of overproduction of hSOD1. It’s a protein produced by everyone but when produced in excess appears to have toxic effects on the motor neurons in 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 offset the effect of the SOD1 defect.
Dror said that while only a small minority of ALS cases are caused by the SOD1 gene, she believes that the underlying technology behind Tofersen will be adapted for patients who suffer as a result of 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 create specific drugs for defects in additional genes,” she said. “This gives us hope.”