Humanitas will coordinate an international clinical trial of a new drug against amyotrophic lateral sclerosis (ALS). The project, winner of the Horizon 2020 call for rare diseases and orphan drugs of the European Commission, will involve a total of 26 European centers with leading experts in ALS. The research group will be led by Professor Alberto Albanese, Head of Neurology at Humanitas.
The study will evaluate the drug Tauroursodessosicolic Acid (TUDCA), an experimental drug derived from bile acids, which has demonstrated antiapoptotic properties in animal and cell models, able to counteract the degeneration of motor neurons affected by the disease.
The pilot study
A first pilot study, conducted on 60 patients in three Italian centers, has shown that taking TUDCA is able to slow down the course of the disease, thus allowing a prolongation of patient survival. The new international study will evaluate on a large scale the therapeutic efficacy of TUDCA in patients with ALS, a disease for which there is currently no cure.
Patient enrollment began in January 2019: patients diagnosed with ALS, with onset of the disease no longer than 18 months and without swallowing disorders, including patients already undergoing treatment with riluzole, will be able to join the study. Participants in this study will receive TUDCA or placebo (a placebo is a substance that does not contain any active medication but is similar in appearance to the drug being studied). This means that neither the patient nor the doctor will know what treatment they are receiving. It will be randomly assigned to the group that will receive TUDCA or placebo; therefore, for patients enrolled in the study there will be a 50% chance of receiving the drug.