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It has been over 80 years since the disease named after baseball legend Lou Gehrig claimed his life. In all that time, a cure has remained elusive and treatment options are very limited.
Also known as amyotrophic lateral sclerosis, or ALS, this neurodegenerative disease attacks nerve cells in the brain and spinal cord that control muscles. It progressively takes people’s ability to move, talk and, eventually, breathe. No matter what we call it, this disease is devastating.
“It is a brutal disease because it’s loss after loss after loss,” Karin Tilberg of Orono told Maine Public. Her husband Chris Smith died from the disease in January 2021.
Tilberg brought the idea of a statewide ALS registry to Gov. Janet Mills, and the governor submitted a bill that would require health care providers to report diagnoses of this disease to the Maine Center for Disease Control and Prevention. Having a better understanding of who has ALS and where they live could provide more insight into what causes it.
“I hope it’s a sign of hope and encouragement that more information will be gathered about this disease to find solutions,” Tilberg said. “You know, there aren’t any now.”
Much remains unknown about the cause and treatment of ALS. Anything that can add progress and hope to an often hopeless situation is welcome, even incremental steps forward. The Maine Legislature has a chance to take such a step with the creation of this registry.
“What’s the registry good for? Well, you can’t treat what you can’t measure,” Dr. John Taylor, a neurologist at Mid Coast Hospital and the director of Maine’s only ALS clinic, told Maine Public.
You also can’t treat a disease without treatments, and right now, only a small handful exist for ALS. In yet another blow to ALS patients and advocates, a new treatment with research funded in part by money raised during the “Ice Bucket Challenge” failed to get the recommendation of a U.S. Food and Drug Administration (FDA) advisory panel last week.
By a non-binding vote of 6-4, this FDA advisory panel found that there was not enough evidence in the single study presented by Amylyx Pharmaceuticals that its treatment, AMX0035, is effective against ALS. That study showed a slower decline for patients receiving the drug, but a majority of panel members wanted to see further trial results. The FDA has also raised concerns about the existing study.
“There’s no question of the burdensome nature of the disease and a huge unmet need for safe and effective treatment,” said panel member Dr. Kenneth Fischbeck, an investigator at the National Institute of Neurological Disorders and Stroke, according to the New York Times. “On the other hand… we were asked to look for substantial evidence with persuasiveness and robustness. And I think this one trial doesn’t quite meet that bar.”
We understand the arguments about process and scientific integrity involved here. But we can’t help but focus on the people involved. It could take multiple years to conduct more study on this drug. Many of these patients won’t have that long. Just listen to the people who have already been receiving the treatment as part of the trial.
“I implore you with literal life and death urgency to recommend approval of AMX0035,” Becky Mourey told the panel, as reported by the Times.
Importantly, the panel’s patient representative Mark Weston, who has ALS, voted in favor of the treatment. And he emphasized that both the manufacturer and the FDA have agreed on its safety. The FDA, for example, said AM0035 “appears to be relatively safe and well-tolerated.”
Last year, the FDA approved a drug to treat Alzheimer’s despite an overwhelming vote (10 out of 11 members) against it from the advisory panel. The move was controversial, and again, we understand why. But given that precedent, the devastating impacts of this disease coupled with few options, a much closer panel vote and the initial trial results, the agency should take a hard look at doing the same when it decides whether to approve this ALS treatment in the coming months.
“This is a uniformly fatal disease so any drug that shows this benefit — and even if that benefit is modest — is fantastic for patients, who have one path forward with this disease: death,” Dr. Jeffrey Rothstein of Johns Hopkins University, who helped patients participate in the Amylyx study, told the Associated Press.
We understand why some experts would like to see more study. We’re also quite sure the people living with ALS would like to see more time with their families.
In the fight against ALS, bureaucracy at both the state and federal levels needs to help marshal resources, better understanding and paths to treatment — not stand in the way.