Human umbilical cord blood-derived mononuclear cell transplantation: case series of 30 subjects with hereditary ataxia.

BACKGROUND: The 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. METHODS: 30 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. RESULTS: A 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. CONCLUSION: The 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.

Human cord blood-derived mononuclear cell transplantation for viral encephalitis-associated cognitive impairment: a case report.

INTRODUCTION: Herpes simplex virus is the most common cause of sporadic viral encephalitis. Cognitive impairments persist in most patients who survive herpes simplex virus-caused encephalitis after undergoing currently available treatments. This is the first report on the development of human cord blood-derived mononuclear cell transplantation as a new treatment intervention to improve the prognosis of sequelae of viral encephalitis. CASE PRESENTATION: An 11-year-old Han Chinese boy developed sequelae of viral encephalitis with cognitive, mental and motor impairments in the 8 months following routine treatments. Since receiving allogeneic cord blood-derived mononuclear cell transplantation combined with comprehensive rehabilitation therapies 7 years ago, the patient's health has significantly improved and remained stable. CONCLUSIONS: Human cord blood-derived mononuclear cell transplantation may be a potential therapeutic strategy for treating the neuropsychiatric and neurobehavioral sequelae of viral encephalitis.