Current Trials and Therapies for the Treatment of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

An area of health care that provides many more questions than answers includes neurodegenerative disorders. Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig\u27s disease, and frontotemporal dementia (FTD) are both diseases about which we know very little. However, ALS and FTD affect nearly 30,000 and 60,000 Americans respectively. Currently, there is not a cure for ALS or FTD and treatment options are aimed toward symptom management. Much of the pathophysiology of these diseases is unknown, but we do know there are genetic implications, specifically in SOD1, TARDBP and c90RF72. These mutations lead to cognitive deficits, muscle weakness and, eventually, paralysis. The only U.S. Food and Drug Administration (FDA) approved treatment for ALS is riluzole (Rilutek®) which helps to slow the disease progression but still lacks a curative effect. An area of interest in the treatment of ALS and FTD is antisense oligonucleotide (ASO) therapy. Current trials examining ASOs as targets for SOD1 and c90RF72 have shown promise. With these therapies still in preliminary trials, care for these patients is largely palliative. Pharmacists, as part of a multidisciplinary team, can play an integral role in the management of disease sequelae to improve the patient\u27s quality of life