Exploring the ALS Treatment Landscape: FDA-Approved Therapies in Current Clinical Practice


Clinical Management

Poster Number: M192


Gabriella Law, Georgetown University Medical Center, Micah Hamilton, Georgetown University Medical Center, Daniel Bustamante, Medstar Georgetown University Hospital, Samuel Calabria, DO, Medstar Georgetown University Hospital, Nicholas Streicher, MD, Medstar Georgetown University Hospital, Shakti Nayar, MD, Medstar Georgetown University Hospital

The past few years have seen an increase in treatment options from one to three FDA-approved disease-modifying medications. ALS practice guidelines recommend patients take all three: riluzole, edaravone, and combination sodium phenylbutyrate/taurursodiol (PB-TURSO) (Goyal 2023). Given the demonstrated benefit to early treatment initiation in ALS, it is important to assess whether this recommendation is achieved in clinical practice, to start to address whether barriers to implementation need to be addressed.

We sought to outline how many of our ALS clinic patients were taking the recommended combination of three FDA-approved ALS medications, as well as how many were taking a different combination or monotherapies.

We examined data from a review of the electronic medical record for patients within our mid-Atlantic hospital network carrying a diagnosis of ALS. We assessed the number of patients receiving each of these three medications as monotherapy and any combination of these medications based on medication lists. We excluded patients first seen before PB-TURSO was able to be prescribed to our clinic patients, which was December 2022, and excluded patients who were only seen once and were not on any medication. After these exclusions, thirty-nine ALS patients were suitable for study.

Riluzole monotherapy was the most common treatment, with eighteen patients (46.15%) receiving this. Combination therapy with all three medications was the next most frequent, with six patients (15.38%) receiving this. The same number of patients received edaravone alone and edaravone/PB-TURSO, with four patients (10.26%) in each group. One patient (2.56%) received PB-TURSO alone. Two patients (5.12%) were taking riluzole and edaravone, and one patient (2.56%) was taking riluzole and PB-TURSO. Three patients (7.69%) were taking none of these therapies.

Our results suggest that most patients recently establishing care with our ALS clinic are not accessing all of the current FDA-approved, disease-modifying ALS medications quickly, despite our goal of early initiation of all three treatments. In particular, many patients were not taking the most recently approved therapies. Riluzole was FDA-approved in 1995, edaravone in 2017, and PB-TURSO in 2022. We suspect that difficulties in obtaining insurance approval for the more recently-approved compounds may be an addressable barrier to optimizing care.