An Overview of ALS Treatment Developments for ALS Awareness Month

Matthew Harms, a medical consultant and care center director at the MDA and associate professor of neurology at Columbia University Medical Center, to discuss new treatments and anticipated developments for ALS over the next year.

May is amyotrophic lateral sclerosis (ALS) Awareness Month, so Pharmacy Times® interviewed Matthew Harms, a medical consultant and care center director at the Muscular Dystrophy Association (MDA) and associate professor of neurology at Columbia University Medical Center, to discuss new treatments and anticipated developments over the next year.

During the discussion, Harms detailed where the ALS treatment field is currently in terms of treatment developments.

“I think there are 2 fronts that are moving forward pretty rapidly. One is the reformulation of FDA-approved medications into formats that are easier for patients with ALS swallowing problems to be able to take,” Harms said.

Harms noted that due to this rapid progression in treatment development, there are now a couple of additional routes of administration for riluzole, a medication that was approved many years ago. Additionally, there is research underway regarding the potential for an oral formulation of edaravone, which is currently an infusion therapeutic, may be bioequivalent and have a similar effect.

“The other route is to brand new therapies, and that is where we're seeing lots of robust advances,” Harms said.

Harms explained further that when he first started taking care of patients with ALS, there were only about 5 drugs in the pipeline for treatment.

“That has ballooned to over 50. The main driver of that is the really intensive study of patients

with genetic forms of ALS. Those genes that have been identified now number almost 3 dozen, and with the support of the MDA and funding to identify those genes, and then study the downstream pathways, we have a really firm handle on 5 or 6 key driving pathways in all of ALS, not just the genetic forms,” Harms said. “Trials are being designed for idiopathic ALS, hinging on these pathways.”

The discussion also included details regarding some larger ALS clinical trials that are expected to have results in the coming year, why investigational therapies have a broader application for nongenetic ALS, how big data has helped researchers to better understand ALS, and the value of the pharmacist in providing caring for patients with ALS.