An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich’s ataxia

Kevin C. Kemp (), Anastasia Georgievskaya, Kelly Hares, Juliana Redondo, Steven Bailey, Claire M. Rice, Neil J. Scolding, Chris Metcalfe and Alastair Wilkins
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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

Nature Communications, 2022, vol. 13, issue 1, 1-8

Abstract: 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.

Date: 2022
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DOI: 10.1038/s41467-022-31450-w

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