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 marrowderived 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 “nonresponders”. 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.
Highlights
Amyotrophic lateral sclerosis is a disease which still remains untreatable [1,2]
We hypothesize that the pre-treatment progression rate may play a role as a predictive factor and a criterion for selecting Amyotrophic lateral sclerosis (ALS) patients for cell-based therapies
No immediate surgical complications have been observed after the cells-CSF suspension was injected: one patient developed post-dural puncture headache (PDPH)
Summary
Amyotrophic lateral sclerosis is a disease which still remains untreatable [1,2]. It is usually fatal disease of the upper and lower motoneuron. In recent years potential benefits of stem cell-based approaches have been demonstrated making stem cells an interesting candidate for new ALS therapy [4]. Mesenchymal stem cells (MSCs) have several attributes that make them good candidates for cell-based therapies too [6]. By using MSCs we can avoid the ethical issues of embryonic or fetal derived stem cells. In several recent pre-clinical and clinical trials MSCs-based approaches have been shown to be safe and feasible
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