Author
Listed:
- Kevin C. Kemp
(University of Bristol, Southmead Hospital)
- Anastasia Georgievskaya
(University of Bristol, Southmead Hospital)
- Kelly Hares
(University of Bristol, Southmead Hospital)
- Juliana Redondo
(University of Bristol, Southmead Hospital)
- Steven Bailey
(University of Bristol, Southmead Hospital)
- Claire M. Rice
(University of Bristol, Southmead Hospital)
- Neil J. Scolding
(University of Bristol, Southmead Hospital)
- Chris Metcalfe
(University of Bristol)
- Alastair Wilkins
(University of Bristol, Southmead Hospital)
Abstract
Friedreich’s ataxia (FA) is an inherited progressive neurodegenerative disease for which there is no proven disease-modifying treatment. Here we perform an open‐label, pilot study of recombinant human granulocyte-colony stimulating factor (G-CSF) administration in seven people with FA (EudraCT: 2017-003084-34); each participant receiving a single course of G-CSF (Lenograstim; 1.28 million units per kg per day for 5 days). The primary outcome is peripheral blood mononuclear cell frataxin levels over a 19-day period. The secondary outcomes include safety, haematopoietic stem cell (HSC) mobilisation, antioxidant levels and mitochondrial enzyme activity. The trial meets pre-specified endpoints. We show that administration of G-CSF to people with FA is safe. Mobilisation of HSCs in response to G-CSF is comparable to that of healthy individuals. Notably, sustained increases in cellular frataxin concentrations and raised PGC-1α and Nrf2 expression are detected. Our findings show potential for G-CSF therapy to have a clinical impact in people with FA.
Suggested Citation
Kevin C. Kemp & Anastasia Georgievskaya & Kelly Hares & Juliana Redondo & Steven Bailey & Claire M. Rice & Neil J. Scolding & Chris Metcalfe & Alastair Wilkins, 2022.
"An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich’s ataxia,"
Nature Communications, Nature, vol. 13(1), pages 1-8, December.
Handle:
RePEc:nat:natcom:v:13:y:2022:i:1:d:10.1038_s41467-022-31450-w
DOI: 10.1038/s41467-022-31450-w
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