Breakthrough SMA Drug Gets First Hospital Tryout

Israel is pioneering a “breakthrough experimental drug” against spinal muscular atrophy (SMA), The Jerusalem Post reported on Wednesday.

Tel Aviv Sourasky Medical Center’s Dana-Dwek Children’s Hospital hailed the new treatment as “a breakthrough in all the treatments of genetic diseases of this kind that in the past could be regarded as science fiction.”

The disease occurs in Israel in one per 6,000 to 11,000 births.

The hospital’s pediatric neurology unit began offering Sprinraza, the first to do so after approval by the U.S. Food and Drug Administration. The first patient was a six-month-old baby girl who “successfully received her first dose. We all hope that in fact, the treatment will bring about a significant improvement in her condition,” said Prof. Aviva Fattal-Valvsky, head of the unit.

Sprinraza was authorized for use on an experimental basis to a limited number of patients, pending approval for non-experimental use by the Health Ministry.
The drug will be given in the pediatric intensive-care unit of the hospital run by Dr. Efraim Sadot. It is injected into the spinal canal or subarachnoid space so that it reaches the cerebrospinal fluid.