Pilot study of granulocyte colony stimulating factor (G-CSF)-mobilized peripheral blood stem cells in amyotrophic lateral sclerosis (ALS).
Amyotrophic lateral sclerosis (ALS) is characterized by degeneration of upper and lower motor neurons in the brain, brainstem, and spinal cord. It has been proposed that bone marrow (BM)-derived cells might supply motor neurons and other cells with a cellular milieu more conducive to survival in ALS. Direct injection of stem cells in ALS is problematic because of the large expanse of the neuraxis that would need to be injected. We reasoned that transiently increasing the number of circulating hematopoietic stem cells might be a useful therapeutic approach. However, agents stimulating the activation and mobilization of hematopoietic stem cells may have adverse effects such as activation of microglial cells. We conducted a small pilot trial of the collection and reinfusion of granulocyte-colony stimulating factor (G-CSF)-mobilized peripheral blood stem cells (PBSC) in ALS patients and found no adverse effects, paving the way for a properly powered therapeutic trial with an optimized regimen of G-CSF.
Cashman
Neil
N
ALS Centre Brain Research Centre, University of British Columbia, Vancouver, BC, Canada.
Tan
Lih-Yeen
LY
Krieger
Charles
C
Mädler
Burkhard
B
Mackay
Alex
A
Mackenzie
Ian
I
Benny
Barrett
B
Nantel
Stephen
S
Fabros
Marife
M
Shinobu
Leslie
L
Yousefi
Masoud
M
Eisen
Andrew
A
D000690:Amyotrophic Lateral Sclerosis / Q000473:pathology / Q000503:physiopathology / Q000628:therapy*; D001344:Autopsy; D005069:Evaluation Studies as Topic; D016179:Granulocyte Colony-Stimulating Factor / Q000494:pharmacology*; D019650:Hematopoietic Stem Cell Mobilization / Q000379:methods; D006412:Hematopoietic Stem Cells / Q000187:drug effects* / Q000502:physiology*; D006801:Humans; D053580:Muscle Strength; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods*; D010865:Pilot Projects; D013997:Time Factors
D016179:Granulocyte Colony-Stimulating Factor