Two New Compounds Target Cell Death By Mitochondria

Trophos, a biotechnology company in Marseille, France, recently announced that it plans to launch a phase 2-3 study of its experimental compound TRO19622 in amyotrophic lateral sclerosis (ALS) in 10 centers in Europe, pending regulatory approvals.

TRO19622 is given orally and is thought to interfere with an early step in the so-called “cell death cascade.”

The cholesterol-like compound apparently prevents microscopic pores in the mitochondria of nerve cells from opening. The mitochondria, which resemble miniature cells, are present in great numbers inside nerve and muscle cells, where they produce needed energy but can also trigger a cell’s self-destruction.

In a phase 1 trial involving 36 people with ALS, TRO19622 was safe and well tolerated. The phase 2-3 trial will determine whether the drug affects survival or function.

Also in December, Maas Biolab, of Albuquerque, N.M., announced it would begin safety testing in animals of its experimental compound Mitogard, with the eventual goal of conducting a clinical trial in ALS patients.

Like TRO19622, Mitogard is thought to interfere with the opening of pores in the mitochondria. It’s based on a much older drug, cyclosporine, which is used to suppress the immune system. It has to be delivered via the spinal fluid to reach the nervous system.