CALABASAS, Calif., March 15 (UPI) -- The Amyotrophic Lateral Sclerosis Association is funding a study in monkeys of a possible Lou Gehrig's disease drug.
The treatment to be tested -- developed in collaboration with Isis -- is a so-called antisense drug, which lowers the production of a mutant protein that is linked to some inherited cases of ALS, commonly known as Lou Gehrig's disease.
Researchers have found that, in some inherited cases of the disease, a protein called copper-zinc superoxide dismutase has mutated, which then kills motor neurons in these patients, ALSA said.
The drug, dubbed ISIS 333611, looks promising as a treatment for the debilitating disorder based on the results of previous rodent studies, the group said.
The monkey study -- called TREAT ALS -- is also aimed at assessing new genes linked to the disease. The trial will be conducted over 90 days, with researchers assessing dose tolerance and safety.
"The potential of this therapeutic approach is very exciting for ALS patients carrying the SOD1 mutation," said Lucie Bruijn, ALSA science director and vice president. "We are pleased that through TREAT ALS we are able to support such important efforts and move them rapidly from the lab to the clinic."
