An Open-Label, Prospective Study Evaluating the Clinical and Immunological Effects of Higher Dose Granulocyte Colony-Stimulating Factor in ALS.

Objective: We evaluated the safety and tolerability of higher-dose granulocyte colony-stimulating factor (G-CSF) in patients with amyotrophic lateral sclerosis. In addition, rates of disease progression and serum G-CSF levels and other immunological and hematological markers were measured.

Methods: Three patients with advanced amyotrophic lateral sclerosis were treated with G-CSF subcutaneously at 5 μg/kg twice daily for 5 consecutive days monthly for 4-12 months. Patients were monitored for adverse effects, and disease progression was assessed with ALSFRS-R and other measures.

Results: Patients tolerated higher-dose G-CSF well with no serious adverse events. Adverse effects were mild to moderate with musculoskeletal pain and malaise being most often reported. No significant change in the rate of disease progression was noted for ALSFRS-R or other measures. Bone marrow progenitor cells were rapidly mobilized for a duration of approximately 9 days with transient and variable effect on cytokines.

Conclusions: Higher-dose G-CSF was well tolerated in this cohort with no apparent effect on disease progression up to 1 year.