The combination was christened AMX0035 because 3 and 5 are the favorite numbers of Mr. Cohen’s fiancée. During YMCA basketball sessions with Dr. Tanzi, they discussed trying it for Alzheimer’s. But investors weren’t interested.
Dr. Tanzi introduced the young men to Dr. Cudkowicz, who had once studied sodium phenylbutyrate and convinced them to test it for A.L.S. It’s now also in an Alzheimer’s trial.
The A.L.S. study, called Centaur, conducted across the country by leading A.L.S. researchers, involved patients who developed symptoms within 18 months before the trial and were affected in at least three body regions, generally signs of fast-progressing disease. Two-thirds received AMX0035, a bitter-tasting powder they mixed with water to drink or ingest through a feeding tube twice daily.
The primary goal was slowing decline on a 48-point A.L.S. scale rating 12 physical abilities, including walking, speech, swallowing, dressing, handwriting and breathing. Over 24 weeks, patients on placebo declined 2.32 points more than those taking the drug combination. Fine motor skills benefited most.
“The data that we see here indicates there may be some beneficial effect but it doesn’t look like what you’d call a home run,” Dr. Koroshetz said.
Some patients experienced gastrointestinal side effects like nausea and diarrhea, but after three weeks those effects largely subsided, and overall, the drug was safe, researchers said.
In most secondary measures, including muscle strength, respiratory ability and whether patients were hospitalized, AMX0035 appeared better than placebo, although it wasn’t statistically significant. Another measure, a biomarker of neurodegeneration, didn’t seem significantly affected. A few patients died in both groups, but experts said identifying the impact on mortality would require evaluation over a longer period.