Canada pharmaceutical company PharmaTher is partnering with the University of Kansas to develop and possibly commercialize the dissociative anesthetic ketamine for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
“We are pleased to have added the Lou Gehrig’s disease program to our already impressive development pipeline that focuses on novel uses, formulations and delivery of ketamine in the treatment of neuropsychiatric, neurological and neuromuscular diseases,” said Fabio Chianelli, CEO of PharmaTher.
ALS is a progressive neuromuscular disease that affects about 50,000 people in the U.S. and Europe. As ALS progresses, motor neurons die causing the brain to lose the ability to control muscle movements. Eventually, patients lose the ability to speak, eat, move, or breathe. Most patients succumb to the disease within 2 to 6 years of diagnosis.
There is no cure for ALS, and only three medications have been approved to treat symptoms. And while effective at symptom reduction, these treatments fail to have any measurable effects on slowing disease progression or improving survival.
But, a team of University of Kansas Medical Center researchers believe, based on their preclinical studies, that ketamine may be capable of stopping or slowing the muscle decline associated with ALS and extend the life expectancy for patients if given in the early stages of muscle decline.
According to PharmaTher, ketamine works by blocking the action of the ionotropic glutamate receptor, the NMDA receptor. “Unlike other inhibitors of NMDA receptor function, such as riluzole, ketamine dampens NMDA receptor-related glutamate excitotoxicity indirectly. Further, ketamine can lower D-serine concentrations intracellularly and also partially activates dopamine receptors,” the company said.
“Collectively, these mechanisms of ketamine contribute in part to the drug’s neuroprotective effects which may extend to the motor neurons targeted in ALS,” the company added.
Ketamine is in lower doses an anesthetic used in medical and veterinary practice. At higher doses, the therapy has dissociative effects. Ketamine is also used off-label to treat chronic pain and depression.