title: Safety and feasibility of long term administration of recombinant human granulocyte-colony stimulating factor in patients with amyotrophic lateral sclerosis.
abstract: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neuronal disease resulting in a loss of the upper and lower motor neurons and subsequent death within three to four years after diagnosis. Mouse models and preliminary human exposure data suggest that the treatment with granulocyte-colony stimulating factor (G-CSF) has neuro-protective effects and may delay ALS progression. As data on long-term administration of G-CSF in patients with normal bone marrow (BM) function are scarce, we initiated a compassionate use program including 6 ALS patients with monthly G-CSF treatment cycles. Here we demonstrate that G-CSF injection was safe and feasible throughout our observation period up to three years. Significant decrease of mobilization efficiency occurred in one patient and a loss of immature erythroid progenitors was observed in all six patients. These data imply that follow-up studies analyzing BM function during long-term G-CSF stimulation are required.
authors.affiliation: University Hospital Regensburg, Department of Internal Medicine III, Regensburg, Germany. Electronic address: jochen.grassinger@ukr.de.
authors.forename: Jochen
authors.initials: J
authors.lastname: Grassinger
authors.affiliation: University Hospital Regensburg, Department of Neurology, Regensburg, Germany.
authors.forename: Andrei
authors.initials: A
authors.lastname: Khomenko
authors.affiliation: University Hospital Regensburg, Department of Internal Medicine III, Regensburg, Germany.
authors.forename: Christina
authors.initials: C
authors.lastname: Hart
authors.affiliation: University Hospital Regensburg, Department of Neurology, Regensburg, Germany.
authors.forename: Dobri
authors.initials: D
authors.lastname: Baldaranov
authors.affiliation: University Hospital Regensburg, Department of Neurology, Regensburg, Germany.
authors.forename: Siw W
authors.initials: SW
authors.lastname: Johannesen
authors.affiliation: University Hospital Regensburg, Department of Internal Medicine III, Regensburg, Germany.
authors.forename: Gunnar
authors.initials: G
authors.lastname: Mueller
authors.affiliation: University Hospital Regensburg, Department of Internal Medicine III, Regensburg, Germany.
authors.forename: Roland
authors.initials: R
authors.lastname: Schelker
authors.affiliation: University Hospital Regensburg, Department of Neurology, Regensburg, Germany.
authors.forename: Wilhelm
authors.initials: W
authors.lastname: Schulte-Mattler
authors.affiliation: University Hospital Regensburg, Department of Internal Medicine III, Regensburg, Germany.
authors.forename: Reinhard
authors.initials: R
authors.lastname: Andreesen
authors.affiliation: University Hospital Regensburg, Department of Neurology, Regensburg, Germany.
authors.forename: Ulrich
authors.initials: U
authors.lastname: Bogdahn
mesh_terms: D000328:Adult; D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy*; D018450:Disease Progression; D004912:Erythrocytes / Q000187:drug effects; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000009:adverse effects* / Q000627:therapeutic use*; D006098:Granulocytes / Q000187:drug effects; D006801:Humans; D007958:Leukocyte Count; D008264:Macrophages / Q000187:drug effects; D008297:Male; D008875:Middle Aged; D018696:Neuroprotective Agents / Q000009:adverse effects / Q000627:therapeutic use; D010976:Platelet Count; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use
chemical_list: D018696:Neuroprotective Agents; D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor
keywords: ALS; Amyotrophic lateral sclerosis; G-CSF; Granulocyte-colony stimulating factor