Human umbilical cord blood-derived mononuclear cell transplantation: case series of 30 subjects with Hereditary Ataxia

Abstract

BackgroundThe differential diagnosis for hereditary ataxia encompasses a variety of diseases characterized by both autosomal dominant and recessive inheritance. There are no curative treatments available for these neurodegenerative conditions. This open label treatment study used human umbilical cord blood-derived mononuclear cells (CBMC) combined with rehabilitation training as potential disease modulators.Methods30 patients suffering from hereditary ataxia were treated with CBMCs administered systemically by intravenous infusion and intrathecally by either cervical or lumbar puncture. Primary endpoint measures were the Berg Balance Scale (BBS), serum markers of immunoglobulin and T-cell subsets, measured at baseline and pre-determined times post-treatment.ResultsA reduction of pathological symptoms and signs was shown following treatment. The BBS scores, IgG, IgA, total T cells and CD3+CD4 T cells all improved significantly compared to pre-treatment values (P < 0.01~0.001). There were no adverse events.ConclusionThe combination of CBMC infusion and rehabilitation training may be a safe and effective treatment for ataxia, which dramatically improves patients' functional symptoms. These data support expanded double blind, placebo-controlled studies for these treatment modalities.

Effects of Intravenous Administration of Human Umbilical Cord Blood Stem Cells in 3-Acetylpyridine-Lesioned Rats

“…However, to our knowledge there are only two studies using HuUCBCs for the treatment of CA [39, 40]. By using an anti-HLA-ABC antibody, we found that intravenous administered HuUCBMCs reached the cerebellum and brain stem of 3-AP-lesioned rats.…”

Machado Joseph disease: clinical and genetic aspects, and current treatment

“…There was a significant improvement in the Berg Balance Scale after 4 --6 weeks of treatment with intravenous and intrathecal human umbilical cord mononuclear cells and rehabilitation training in 30 patients with different hereditary ataxias (5 with SCA3/MJD) [90]. In a further study, 16 patients with SCAs (7 with SCA3/MJD) were treated with intravenous and intrathecal mesenchymal umbilical cord stem cell.…”

Human allogeneic AB0/Rh-identical umbilical cord blood cells in the treatment of juvenile patients with cerebral palsy

“…No serious adverse hematological, biochemical or immunological effects were reported. Later, researchers reported significant improvements (>50%, Berg Balance Scale) after 4e6 combined intrathecal/intravenous infusions of allogeneic UCB cells in 13 of 30 patients with hereditary ataxia; in 17 other patients, the effect was smaller (5e49%) [39].…”