Molecular Basis of Allogeneic Pluripotent Stem Cell Treatment of a Patient with Sporadic ALS

Abstract

There are no curative strategies for ALS, and as a result the alternative therapies are gaining ground, in particular the stem cell-based therapies which hold a promising future. Here we describe an ALS patient, who by choice, received allogeneic human cord-blood derived multipotent stem cells (MSC) intravenously and intrathecally. This is the first snapshot of pre-and post-stem cell transplantation in an ALS patient through transcriptomic, epigenomic, and proteomic follow-up elucidating the effect of stem cells in ALS. Although new CSF proteins (FGA, B2M, FN1 and IGFBP3) were observed, but more work is needed to fully elucidate the effect based on larger cohorts in a clinical trial setting. These analyses may serve as a future guide to both scientists and clinicians alike in defining the utility of such cells in ALS treatment.