Effect of autologous bone marrow mononuclear cell transplantation coupled with rehabilitation in limb girdle muscular dystrophy-A case report

Cell transplantation is emerging as a potential therapeutic option for LGMD due to its ability to aid in repair and regeneration of dystrophic muscles. Our aim was to study the benefit of autologous bone marrow mononuclear cells (BMMNCs) in LGMD. We administered a 39 year old LGMD male with autologous BMMNCs intramuscularly and intrathecally twice followed by rehabilitation. His muscle power in all limbs was below functional level with proximal weakness more than distal. Functional Independence Measure (FIM) score was 114. Over a period of 18 months, muscle power increased gradually with improvements in functional activities. FIM score was maintained indicating a halt in the progression of disease. 36 months after second dose of transplantation, patient's condition was maintained with no deterioration in quality of life. This report provides early evidence of beneficial effects of autologous BMMNCs coupled with rehabilitation in halting the disease progression and functional improvement in LGMD.