Mesenchymal Stem Cell (MSC) Transplantation in Patients with Amyotrophic Lateral Sclerosis (ALS): Is there a “Responder Population”?

Abstract

Objective: To analyze the safety and clinical effect of stem cell therapy in ALS. Methods: In phase I of the trial, ALS subjects have been intrathecally transplanted with autologous bone marrow-derived mesenchymal stem cells (MSCs) using a surgical procedure. Results: We present the results of a 6-month interim analysis of the ongoing study. Intrathecal administration of MSCs into ALS patients was feasible and safe. We showed a clinical benefit evident for the entire group of patients (n=25). The mean rate of ALSFRS-R score change (decrease) pre-transplant was 1.76 ± 1.36 points/period whereas the mean post-transplant rate was 1.06 ± 0.9 points/period (p=0.014). The key finding of our study is that there appears to be a group of patients, whom we call "responders" whose reaction to the treatment was different from the reaction of other patients we call "non-responders". Conclusion: In our study the "responders" progressed faster prior to the treatment than "non-responders". Hence, we hypothesize that the pre-treatment progression rate may play a role as a predictive factor and a criterion for selecting ALS patients for cell-based therapies.