Translating cellular therapies from bench to bedside for amyotrophic lateral sclerosis

The last decade has witnessed an increasing number of biologic (e.g., cell- or viral vector-based) therapeutics supported by preclinical efficacy data for the treatment of afflictions to the CNS. While some international investigators have undertaken preliminary clinical safety studies, published literature indicate varying degrees of rigor with respect to study design and technical approach. To our knowledge, ours is the first group to have systematically generated preclinical validation data for a delivery approach and translated this into a Phase I trial attempting to covalidate the safety of a direct, targeted delivery approach, as well as a cell-based therapeutic. This article discusses the rationale for cell-based therapy in amyotrophic lateral sclerosis and several of the unique considerations encountered during this process.