Safety and Efficacy of Autologous Bone Marrow Derived Mononuclear Cell Transplant in the Management of Various Neurological Disorders.

Background: Cerebral palsy (CP), traumatic spinal cord injury (SCI), and muscular dystrophy (MD), among the various other neurological disorders, are major global health problems because they are chronic disorders with no curative treatments at present. Current interventions aim to relieve symptoms alone and therefore emphasize the necessity for new approaches.

Objective: This study aims to assess the safety and efficacy of autologous bone marrow-derived mononuclear cell (BM-MNC) therapy in patients with CP, traumatic SCI, and MD. Functional improvement and safety are the primary outcomes, while secondary outcomes include patient-reported improvement in quality of life.

Methods: This was a single-arm, open-label prospective study conducted on 100 patients with CP, SCI, and MD at the GSVM Medical College, Kanpur, India. Bone marrow aspirates were processed via centrifugation, and autologous BM-MNCs were administered intrathecally or intramuscularly. Gross motor function classification system (GMFCS) for CP, the American spinal injury association (ASIA) motor score for SCI, and the north star assessment for limb girdle type dystrophies (NSAD) for MD were used for functional outcome assessment at baseline and at post-treatment cycles. Informed consent was obtained, and the study was approved by the ethics committee. Paired t-tests were used to analyze statistical significance (p<0.05).

Results: Functional improvements were significant with autologous BM-MNC therapy. Improved motor and cognitive function were shown in CP patients with reduced GMFCS scores (p<0.001). Upper and lower extremity ASIA motor scores improved markedly (p<0.001) in SCI patients. Stabilized muscle strength was seen in MD patients, with increased NSAD and activities of daily living (ADL) scores, suggesting slowing of the disease progression (p<0.019). Side effects were mild and transient.

Conclusion: Autologous BM-MNC therapy appears to be a promising, minimally invasive option for patients with CP, SCI, and MD, which appears to markedly improve functional outcomes and quality of life and may therefore be relevant to clinical practice.