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A combination of two experimental drugs appears to slow the decline of patients with ALS, or Lou Gehrig's disease. NPR's Jon Hamilton reports this is just the latest sign that researchers are closing in on better treatments for the fatal illness.
JON HAMILTON, BYLINE: Patients with ALS lose the ability to control their muscles over several years, and no treatment has been able to stop this process. But Dr. Sabrina Paganoni of Massachusetts General Hospital says that after decades of frustration, scientists are making good progress.
SABRINA PAGANONI: I'm convinced that we are at the beginning of a new era in ALS treatment discovery.
HAMILTON: Paganoni led a study of 137 people with a fast-progressing form of the disease. Some took daily doses of an experimental drug combination known as AMX0035. Paganoni says it helps protect nerve cells from two types of damage that are hallmarks of ALS.
PAGANONI: Patients who were treated with AMX0035 retain physical function longer than those who receive placebo. And this is very hopeful for people with ALS, especially because we were able to see a treatment effect in a relatively short period of time.
HAMILTON: After six months, patients who got the drug scored several points higher on a standard measure of disability. Paganoni says the difference was modest, but meaningful to patients.
PAGANONI: They want to be able to continue to use their hands so that they can cut their own food and type emails, or they want to be able to continue to walk and to climb stairs. And this is exactly what we measured in the trial.
HAMILTON: Many of the patients who got AMX0035 were already on two other drugs for ALS that have become the standard treatment. Paganoni says it appears that the new and old drugs all work in different ways to slow down the disease.
PAGANONI: So we think ultimately we will need a combination of treatments to effectively fight ALS.
HAMILTON: It's not clear yet whether AMX0035 extends life or maintains muscle strength. And ordinarily, at least one larger study would be required before the Food and Drug Administration considered approving the drug. But Neil Thakur of the ALS Association says that group and the advocacy organization I AM ALS are petitioning the FDA to make an exception.
NEIL THAKUR: In ALS, a trial like this would probably take about three years. And so the question for the whole community is, what do we gain for that three-year study?
HAMILTON: The ALS Association helped fund the research on AMX0035 and has a limited financial stake in its success. Thakur says the group's main concern, though, is patients who won't live long enough to wait for another study.
THAKUR: And so that's why we're thinking the best thing to do for the community is to make this drug available sooner and let everyone have it as a treatment option as soon as possible.
HAMILTON: AMX0035 has taken a highly unusual path toward approval. It was developed by Amylyx, a tiny company founded by a couple of college students. Josh Cohen and Justin Klee are still in their 20s, and Cohen says they were in their office very late one night when they learned the study results.
JOSH COHEN: When the statisticians called, you could hear their whole firm cheering in the background. And so we knew before they said, you know, the numbers that, you know, something good had happened.
HAMILTON: But Klee says their elation was mixed with a sense of responsibility.
JUSTIN KLEE: While these results are great, it's not a cure. And so we and others in the whole community - we need to keep pushing forward until we get cures.
HAMILTON: That's beginning to look more likely, says Kuldip Dave, the ALS Association's vice president of research.
KULDIP DAVE: What makes this time so exciting is there are over 50 different clinical trials that are enrolling and recruiting ALS patients right now. They're all going after different targets.
HAMILTON: The new study appears in The New England Journal of Medicine.
Jon Hamilton, NPR News.
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