ALS on the Cusp

Imagine the elation of patients with amyotrophic lateral sclerosis (ALS), popularly known as Lou Gehrig’s disease, and their families when they read the National Institute of Neurological Disorders and Stroke’s (NINDS) announcement in 2002 that a widely respected mouse model for ALS showed significant improvements when injected with the antibiotic minocycline. The investigators found neuroprotective benefits and all the pharmacological hallmarks of a promising CNS agent in minocycline. Furthermore, it worked by a different mechanism of action than the single approved agent for treating the horrifying neurodegenerative disease that destroys lower and upper motor neurons to gradually trap the alert mind within an increasingly paralyzed body before suffocating the patient to death. That sole drug, Sanofi’ riluzole (Rilutek), extends life less than three months on average. Here was the potential for a combination therapy that might in concert offer hope for staving off devastating decline and painful death more than a couple paltry months.

Pity those same people. Four and a half years later, the crushing blow from the minocycline clinical trials could not...