Gnome Sciences, a molecular pathology and translational research services company, announced a significant advance in the fight against ALS (amyotrophic lateral sclerosis). In a newly published study, its researchers found that a drug already approved to treat cancer—called venetoclax—was able to prevent the progression of ALS in 100% of treated mice that would otherwise develop and die from the disease.

The study showed that mice treated with venetoclax maintained their ability to move, avoided paralysis, and had dramatically less damage to their nerves and brain tissue. The drug also reduced inflammation in the nervous system, which is known to play a role in ALS progression. There are no effective treatments today for ALS, a uniformly fatal disease.

Importantly, the research team also studied brain tissue from people who had died of ALS. They found that certain proteins—called BCL2 family proteins—were much more active in ALS-affected neurons than in healthy ones. These proteins were found in the same areas as other known markers of ALS, suggesting they may play a key role in the disease and treatment. These BCL2 proteins have important roles in key cellular pathways, including apoptosis, autophagy, mitophagy, and clearing of misfolded and aggregated proteins.

“This is a major step forward,” said Gerard Nuovo, MD, the lead investigator at Gnome Sciences. “We’ve shown that a drug already approved for cancer can stop ALS before it starts, in animals—and we see the same molecular signatures in human brain tissue. That’s incredibly promising. I filed the patent for use of BCL2-targeting drugs like venetoclax to treat ALS and other neurodegenerative conditions to enable a critical path forward.”

“We have begun a clinical trial to evaluate the drug in human ALS.” said Dr. A. Mikhail, CEO of Gnome Sciences, “We have guarded optimism, but it is too early at this stage to make clinical recommendations, especially because this class of drugs is not without risks. We are in discussions with several companies to evaluate BCL2 inhibitors in ALS patients.”