title: Autologous bone marrow-derived stem cells in amyotrophic lateral sclerosis: a pilot study.
abstract: BACKGROUND
  Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder with no effective treatment. Stem cell therapy may be one of the promising treatment options for such patients.
  
  
  OBJECTIVE
  To assess the feasibility, efficacy and safety of autologous bone marrow-derived stem cells in patients of ALS.
  
  
  METHODS
  We conducted an open-label pilot study of autologous bone marrow-derived stem cells in patients with ALS attending the Neurology Clinic of a tertiary care referral centre.
  
  
  METHODS
  Ten patients with ALS with mean revised ALS Functional Rating Scale (ALSFRS-R) score of 30.2 (± 10.58) at baseline received intrathecal autologous bone marrow-derived stem cells. Primary end point was improvement in the ALSFRS-R score at 90, 180, 270 and 365 days post therapy. Secondary endpoints included ALSFRS-R subscores, time to 4-point deterioration, median survival and reported adverse events. Paired t-test was used to compare changes in ALSFRS-R from baseline and Kaplan-Meier analysis was used for survival calculations.
  
  
  RESULTS
  There was no significant deterioration in ALSFRS-R composite score from baseline at one-year follow-up (P=0.090). The median survival post procedure was 18.0 months and median time to 4-point deterioration was 16.7 months. No significant adverse events were reported.
  
  
  CONCLUSIONS
  Autologous bone marrow-derived stem cell therapy is safe and feasible in patients of ALS. Short-term follow-up of ALSFRS-R scores suggests a trend towards stabilization of disease. However, the benefit needs to be confirmed in the long-term follow-up period.
authors.affiliation: Department of Neurology, Postgraduate Institute of Medical Education and Research, Chandigarh, India. sudeshprabhakar@gmail.com
authors.forename: Sudesh
authors.initials: S
authors.lastname: Prabhakar
authors.forename: Neelam
authors.initials: N
authors.lastname: Marwaha
authors.forename: Vivek
authors.initials: V
authors.lastname: Lal
authors.forename: Ratti R
authors.initials: RR
authors.lastname: Sharma
authors.forename: Roopa
authors.initials: R
authors.lastname: Rajan
authors.forename: Niranjan
authors.initials: N
authors.lastname: Khandelwal
mesh_terms: D000328:Adult; D000368:Aged; D000690:Amyotrophic Lateral Sclerosis / Q000401:mortality / Q000601:surgery*; D001854:Bone Marrow Cells / Q000502:physiology; D016026:Bone Marrow Transplantation; D004185:Disability Evaluation; D005260:Female; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D012189:Retrospective Studies; D012720:Severity of Illness Index; D033581:Stem Cell Transplantation / Q000379:methods*; D016019:Survival Analysis