No cure: But new treatments are being developed

There is no cure for ALS. Currently, a single drug called riluzole is the only drug that is FDA-approved for use in ALS patients but, at best, it modestly slows the disease. Members of the ALS Therapy Alliance’s scientific board are pursuing exciting new therapies, which are currently in trial.

Biogen Idec and Knopp Neurosciences recently entered into an exclusive worldwide license agreement to try pramipexole, a novel oral neuroprotective therapy, to treat ALS. This promising new drug, conducted by the Northeast ALS Consortium (NEALS), had potentially beneficial effects in recent pilot studies. In a tremendous breakthrough, it showed a favorable dose-related effect in extending survival in a phase two study. The drug is entering a phase three study, funded by Biogen Idec.

In addition to very promising small molecules, which are traditional drugs, researchers are investigating therapies that silence expression of toxic genes, such as the SOD1 gene, whose mutations can cause ALS. This is an innovative new approach, which represents tremendous progress.

Although there are no therapies that slow the basic process of degeneration of motor nerves, there are other treatments, such as feeding and breathing aids, that relieve some ALS symptoms. There are also drugs available for problems that many ALS patients experience, such as fatigue, muscle cramps, depression and sleep disturbance.