Tel Aviv Hospital Administers ‘Game-changing’ ALS Medicine

Patients in Israel have become some of the first globally to get a new ALS drug, which is aimed at slowing down the progress of this disease.

Two patients were administered the new drug named Tofersen for the degenerative disease and it was injected into the spine in the same way as an epidural.

The patient

Hailing from Nahariya, one of the patients is Narkis Moshe, who lost her brother to ALS before being diagnosed with the same six months earlier.

She said that since starting the new drug, she was not that scared of her fate. The 58-year old said that she had been afraid when she had first been diagnosed.

Moshe said that she did not want to go through the same as her brother, but now she felt better because a new era of treatment and research had begun.

She stated that she was excited about the new treatment and hoped it would help her have a good quality of life and a lot more years with her family.

The drug

Biogen, based in Massachusetts, has developed the new drug named Tofersen, but it is not developed for all patients with ALS.

This is due to the fact that there are different underlying causes of the disease. The drug is for patients who have a faulty SOD1 gene.

The Food and Drug Administration (FDA) has given its approval for the use of the drug for a ‘compassionate use’ program, which means it can be used for specific patients before fully regulatory approval is given.

Since Israel adjusts its drug regulation policies in accordance with the US, it allowed the new drug to be used at the Tel Aviv Sourasky Medical Center.

The treatment was administered by Prof. Vivian Dror at Sourasky and she said that only a few dozen patients were getting the drug globally.

She also said that she was pleased that Israel had adopted it early on because it was a game-changing treatment.

She said that the patients who were being given the drug were able to communicate, swallow and still had use of their limbs, so they hoped to slow down their deterioration and help them retain their abilities.

She added that as compared to the other limited treatments that exist for ALS, the new drug brings something significant to the table.

More details

Different patients have different underlying causes that result in an ALS diagnosis. Tofersen has been developed primarily for patients who suffer from the disease due to hSOD1’s overproduction.

Everyone produces this protein, but it can have a toxic impact on the motor neurons when it is produced in excess.

The problem occurs because the SOD1 gene has a defect and this leads to excess production of hSOD1. Tofersen tries to reduce the production of this protein in order to offset the defect.

Dror stated that even though only a small number of ALS cases happen because of the SOD1 gene, it was expected that they would adapt the underlying technology and use it for patients suffering from the disease due to other genes.