Currently, there are only two approved medications to treat Lou Gehrig's disease (also known as ALS), a condition popularized by the Ice Bucket Challenge. With neither of these medications being particularly effective, a two-drug combination developed by two college students from Brown University seven years ago has been found to allow patients to live several months longer.
Known as AMX0035, the drug combination consists of an existing supplement and a medication for a pediatric urea disorder.
For the study, researchers recruited 137 patients who had developed symptoms of ALS at least 18 months before the trial, were affected in at least three parts of the body, and had signs of the disease progressing rapidly. Most were already taking one or both of the approved ALS medications.
All in all, it was found that the new drug combination managed to slow the progression of ALS paralysis by around 25% more than the placebo. Moreover, the researchers behind the study found that those receiving the placebo declined in 18 weeks to a level that patients receiving the treatment reached only after 24 weeks.
One weakness of the study, however, is that it was only conducted for 24 weeks. Thereafter, those given the placebo were given the option to take the therapy for up to 30 months. As such, the researchers do not have full results for patients who only received the placebo for 30 months to compare with those receiving the treatment from Day 1.
Nevertheless, the study reported that people who received AMX0035 during the trial and afterward lived an average of 6.5 months longer than those originally o the placebo- or 25 months compared to 18.5 months.
While promising results, Dr. Walter Koroshetz, director of the National Institute of Neurological Disorders and Stroke, who wasn't involved in the trial, said that it wasn't clear how much the treatment benefited those who received it only after the 24-week trial.
"It could mean that the drug is really effective and people who got the drug late really would have been dead at 12 months instead of 18," he said. "Or, the other way of thinking about it is that the drug is not effective unless you get it early. There's no clue here to which one of those is true."