Granulocyte Colony-Stimulating Factor for Amyotrophic Lateral Sclerosis: A Randomized, Double-Blind, Placebo-Controlled Study of Iranian Patients
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Background and Purpose Te aim of this study was to determine the efcacy and tolerability of granulocyte colony-stimulating factor (G-CSF) in subjects with amyotrophic lateralsclerosis (ALS). Methods Forty subjects with ALS were randomly assigned to two groups, which received either subcutaneous G-CSF (5 μg/kg/q12h) or placebo for 5 days. Te subjects were then followed up for 3 months using the ALS Functional Rating Scale-Revised (ALSFRS-R), manualmuscle testing, ALS Assessment Questionnaire-40, and nerve conduction studies. CD34+/CD133+ cell count and monocyte chemoattractant protein-1 (MCP-1) levels were evaluated atbaseline. Results Te rate of disease progression did not difer signifcantly between the two groups. Te reduction in ALSFRS-R scores was greater in female subjects in the G-CSF group than intheir counterparts in the placebo group. Tere was a trend toward a positive correlation between baseline CSF MCP-1 levels and the change in ALSFRS-R scores in both groups (Spearman’s ρ=0.370, p=0.070). Conclusions With the protocol implemented in this study, G-CSF is not a promising optionfor the treatment of ALS. Furthermore, it may accelerate disease progression in females.
Abstract
Background and Purpose Te aim of this study was to determine the efcacy and tolerability of granulocyte colony-stimulating factor (G-CSF) in subjects with amyotrophic lateralsclerosis (ALS). Methods Forty subjects with ALS were randomly assigned to two groups, which received either subcutaneous G-CSF (5 μg/kg/q12h) or placebo for 5 days. Te subjects were then followed up for 3 months using the ALS Functional Rating Scale-Revised (ALSFRS-R), manualmuscle testing, ALS Assessment Questionnaire-40, and nerve conduction studies. CD34+/CD133+ cell count and monocyte chemoattractant protein-1 (MCP-1) levels were evaluated atbaseline. Results Te rate of disease progression did not difer signifcantly between the two groups. Te reduction in ALSFRS-R scores was greater in female subjects in the G-CSF group than intheir counterparts in the placebo group. Tere was a trend toward a positive correlation between baseline CSF MCP-1 levels and the change in ALSFRS-R scores in both groups (Spearman’s ρ=0.370, p=0.070). Conclusions With the protocol implemented in this study, G-CSF is not a promising optionfor the treatment of ALS. Furthermore, it may accelerate disease progression in females.
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