PharmaTher Orphan Status for Ketamine in ALS

FDA’s orphan designation is based on data from the university that suggest ketamine has the potential to increase life expectancy of ALS patients at any stage and slow the progressive loss of muscle associated with poor outcomes, PharmaTher says. “Ketamine works by blocking the action of the ionotropic glutamate receptor, the NMDA receptor,” it says. “Thus, unlike other inhibitors of NMDA receptor function, such as riluzole, ketamine indirectly dampens NMDA receptor-related glutamate excitotoxicity. Further, ketamine can lower D-serine concentrations intracellularly and also partially activates dopamine receptors. Collectively, these mechanisms of ketamine contribute in part to the drug’s neuroprotective effects, which may extend to the motor neurons targeted in ALS.”

Ketamine was approved by FDA in 1970 as an anesthetic. It is often abused as a party drug because of its euphoric effects.