In the last years research has pointed out potential mechanisms of pathogenesis in ALS
including lack of degradation of abnormally accumulated proteins inside motor neurons, and an
unbalanced function of the immune system leading to the prevalence of a neurotoxic function
over neuroprotection. These two mechanisms contribute to ALS progression hence representing
important therapeutic targets to modify disease expression.
With a phase II clinical trial the investigators aim to study the biological response in ALS
treated with Rapamycin, to obtain predictive information for a larger study.
Eight Italian Centres will enroll 63 patients; treatment will be double blinded to patients
and physicians, and will last 18 weeks.Follow up will be carried out for 36 months (total
duration: 54 weeks).
Phase:
Phase 2
Details
Lead Sponsor:
Azienda Ospedaliera - Universitaria di Modena Azienda Ospedaliero-Universitaria di Modena
Collaborators:
Azienda Ospedaliera Niguarda Cà Granda Azienda Ospedaliero Universitaria Maggiore della Carita Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta Fondazione Salvatore Maugeri IRCCS Azienda Ospedaliera Universitaria San Martino - IST Istituto Nazionale per la Ricerca sul Cancro, Genoa, Italy University of Modena and Reggio Emilia University of Padova University of Turin, Italy