An experimental drug to treat amyotrophic lateral sclerosis (ALS) that was dreamed up by two college students in their dorm room has been shown to moderately slow the diseases' progression, offering hope to patients with ALS, for which there is no cure and very few treatments.

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A drug dreamed up in a dorm room

In 2013, Joshua Cohen and Justin Klee—co-founders of Amylyx, which developed the experimental drug—were undergraduate students at Brown University, and one night, Cohen and Klee and were wondering what causes brain cells to die from neurodegenerative diseases and what might help mitigate that decline.

After an extensive amount of research, Cohen and Klee discovered two drugs, sodium phenylbutrate and taurursodiol, that they thought may be able to protect neurons from degeneration. Neurons in the brain are partly regulated by two structures—mitochrondria and the endoplasmic reticulum, Klee and Cohen learned—so they hypothesized that, if a combination of sodium phenylbutrate and taurursodiol could increase mitochondrial and endoplasmic reticulum function, nerves may be protected.

Klee and Cohen then contacted Rudy Tanzi, an Alzheimer's researcher at Massachusetts General Hospital, who decided to help them test their hypothesis. "When they first wrote to me in June 2013, they were just kids," Tanzi said. "I thought it was a pretty naïve idea, to be honest, but [I] thought I'd go through this exercise with them—throw them some hard-to-do science."

Together, the three designed a laboratory study to test whether the drug combination could protect against oxidative stress and damage.

"I thought, 'Let's test these two whippersnapper undergrads, show them how hard science really is,'" Tanzi said. "I was 100% sure it would fail."

When the results of the study came back a few months later, the group discovered that hydrogen peroxide had killed cultured neuron cells. However, when the drug combination of sodium phenybutrate and taurursodiol was added, 90% of the neurons were saved, Tanzi said.

Tanzi thought the results were a fluke, and he told Cohen and Klee to conduct the experiment one more time. They did—and this time, 95% of the cells were saved when the combination of sodium phenybutrate and taurursodiol was added.

Tanzi was impressed with the studies' results, and he contacted colleagues of his at Massachusetts General to discuss next steps. Those conversations eventually led to the development of clinical studies on the drug combination.

Clinical study shows positive results

Results from one of those clinical studies were published Wednesday in the New England Journal of Medicine, and they showed that the drug combination slowed ALS' progression in some patients.

The double-blinded, randomized study—known as the CENTAUR Trial—involved 137 patients with fast-progressing ALS who researchers tracked for six months. Participants were randomly assigned to receive either Amylyx's drug combination or a placebo. For every one participant who received a placebo, two received Amylyx's drug.

The researchers measured the drug's outcomes based on the ALS Functional Rating Scale, which is a questionnaire that scores how well a patient is able to complete daily activities, such as walking up stairs or swallowing food, on a 48-point scale. Sabrina Paganoni, an ALS researcher at Harvard Medical School and a principal investigator on the study, said ALS patients "want to be able to continue to use their hands so they can cut their own food and type emails, or they want to be able to walk and climb stairs. And this is exactly what we measured in the trial."

The researchers found that, on average, patients taking Amylyx's drug saw their conditions decline by 2.9 points fewer than patients who were waking a placebo—indicating that patients who were taking the drug experienced a slower progression of disease than patients who were taking a placebo.

Specific outcomes varied by patient, but the majority of patients taking Amylyx's drug saw improvements in their fine motor function, the researchers found. None of the patients saw improvements in breathing ability, however.

Small differences could be significant for ALS patients

While the measured differences in the patients' outcomes appear to be small, even minor changes in their conditions can be significant for ALS patients, according to Paganoni.

"Even a small change in a couple of points can mean a large change in what daily life looks like," she said. "A two-point change could mean the difference between eating successfully or requiring a feeding tube—or between walking and using a wheelchair."

While the results of the study don't suggest the drug is a cure for ALS, Paganoni said she's "convinced that we are at the beginning of a new era in ALS treatment discovery."

Since there is no cure for ALS and there currently are very few treatments, it's possible that FDA could approve Amylyx's drug before it enters a Phase 3 clinical trial—a study that could take several years before generating results, according to STAT News. STAT News reports that the ALS Association plans to circulate a public petition calling on FDA to fast-track the drug's approval or to allow expanded access to the drug before Phase 3 trials are completed.

Neil Thakur, chief mission officer for the ALS Association, said he believes it's important that the drug is offered to ALS patients as soon as possible. "I think this is going to be a challenge for the FDA, who will feel pressure to be both bold and conservative," he said. "We want them to take the side of ALS patients."

However, Jeffrey Rothstein, who directs an ALS research center at Johns Hopkins University and was part of the study, said more research is needed to prove that the drug works before it's widely given to patients. ALS patients should "have drugs that work, and that means you need to do two clinical trials," he said.

Merit Cudkowicz, head of the Healey & AMG Center and a senior author of the study, said she hopes there's a way FDA can "get [the drug] to patients but also … keep learning about it" (Keshavan, STAT News, 9/2; Hamilton, "Shots," NPR, 9/2; Belluck, New York Times, 9/2; Rockoff, Wall Street Journal, 9/2).