Authors: Jorge C. Trainini, Gabriel Volman, Noemi Lago, Javier Bordone, Oscar Ectchegoyen, Francisco Villasante, Facundo Heredia, Yanina Huamanchuco, Jorge Docampo, Bastarrica Maria Elena.
Background: Amyotrophic lateral sclerosis (ALS) is a motor neuron degenerative disease, causing progressive muscle weakness and death. Currently, there is no effective therapy to prevent its progression.
Objectives: The primary objective was to evaluate the safety and feasibility of autologous mesenchymal stem cell (MSC) transplantation in pre-rolandic motor and brain stem areas to treat ALS. A secondary objective was to evaluate its therapeutic efficacy through the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) and the functional status of the pyramidal tract using tractography neuroimages.
Material and Methods: A prospective, non-randomized study was conducted, including 12 ALS patients with autologous MSC transplantation between July 2011 and March 2013. Mean age was 57±11 years (29-67 years). Follow-up consisted of ALSFRS, tractography and a respiratory functional study at 6 and 12 months after MSC transplantation. The study assessed: a) the feasibility and safety of the procedure, b) survival and c) time until need for tracheostomy.
Results: Patients were divided into two subgroups; Group I: without manifest bulbar involvement and Group II: with evident bulbar involvement at the time of transplantation. Overall survival at one year post-transplantation was 58% (average survival: 8.8 months); in Group I 7/8 patients (86%) and in Group II, 1/4 patients (25%) survived (average survival: 12 and 4.6 months, respectively).
Conclusions: Neurotransplantation of autologous MSCs in ALS patients is feasible and safe. Patients with early or recently diagnosed disease, without manifest bulbar involvement would derive the greatest benefit, with better chance of survival and quality of life.
View/Download pdf