MDA 2024: Most patients don’t get recommended treatments for ALS

Guidelines urge combo of 3 therapies to treat patients in clinical practice

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The letters MDA, for Muscular Dystrophy Association, are seen against a backdrop of scattered oral medications in this illustration for the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.

While the most recent guidelines for treating amyotrophic lateral sclerosis (ALS) recommend that patients receive three available treatments together, as soon as possible, a new analysis of U.S. data shows that most individuals with the progressive disease don’t get this combination in clinical practice.

The analysis found that most patients are given riluzole, an older therapy, to treat the neurodegenerative disease. However, the more recent treatments for ALS — specifically Radicava or Radicava ORS (edaravone) and Relyvrio (sodium phenylbutyrate and taurursodiol) — were less frequently taken, despite the guidelines urging their use.

Though the reasons for the lesser use of these therapies are not totally clear, the researchers suggest that insurance issues may be to blame.

“We suspect that difficulties in obtaining insurance approval for the more recently-approved compounds may be an addressable barrier to optimizing care,” the scientists wrote in an abstract summarizing their findings.

Their poster, “Exploring the ALS Treatment Landscape: FDA-Approved Therapies in Current Clinical Practice,” was presented at the Muscular Dystrophy Association (MDA) 2024 Clinical and Scientific Conference, held March 3-6 in Florida and virtually.

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Researchers investigate what’s actually happening in their clinic

Riluzole in 1995 became the first ALS treatment to win approval by the U.S. Food and Drug Administration (FDA). The medication initially was sold as a tablet formulation called Rilutek, but it now also exists in an oral film formulation called Exservan and a liquid formulation called Tiglutik.

For decades, riluzole was the only available treatment to slow the progression of ALS, but that changed in recent years with FDA approvals of Radicava in 2017 and Relyvrio in 2022.

As riluzole, Radicava, and Relyvrio all have been proven in clinical trials to slow the progression of ALS, it’s recommended that patients start on all three treatments as soon as possible.

Now, a team led by scientists at Georgetown University conducted a review of patients at their clinic to see if this was actually happening in clinical practice.

“There may be logistical, financial, or other barriers that prevent patients from utilizing FDA-approved medications in the clinical setting,” the scientists wrote.

“Studying real-world access to the three medications … may provide the most accurate picture of medication-specific challenges faced by the current population of patients with ALS,” the team wrote.

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Analysis finds many patients not taking newer treatments for ALS

The analysis included data from 39 people with ALS. All began receiving ALS-specific medical care when all three therapies were already approved and, at least theoretically, available to patients.

The results showed that six (15%) of the patients were receiving all three ALS treatments according to recommendations. By comparison, nearly half (46%) were receiving riluzole only, while four (10%) were receiving Radicava or Radicava ORS alone, and one (3%) was taking Relyvrio alone. The rest were either taking combinations of two meds or were not receiving any treatment.

In total, more than two-thirds of patients (69%) were on the older Rilutek, but less than half were on either of the newer Radicava formulations (41%) or Relyvrio (31%).

“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,” the researchers wrote.

“In particular,” they concluded, “many patients were not taking the most recently approved 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.

Though this study wasn’t designed to look into reasons why patients weren’t taking medications, the researchers said that issues getting insurance coverage for newer therapies, which carry heavy price tags, is likely a major factor.

They also noted that the most recent guidelines from the American Academy of Neurology for ALS management date back to 2009, when neither of these therapies were approved. They called for further work to identify and eliminate barriers to patients accessing the best possible care.

“Years of research have resulted in advances that can significantly benefit ALS patients. To create meaningful change, it is imperative that barriers to implementation are identified and addressed,” they wrote.