DISCONTINUED: Stages of Development for GCSF

The drug development process can be thought of as a series of stages, and drugs must successfully pass through each stage to become available to patients. This treatment has been evaluated, and the program has been discontinued. Thus, it is not in the pipeline.

Link to previous study

GCSF can cross the blood–brain barrier and has multiple trophic effects on CNS cells including direct neuroprotective and neuro-regenerative effects on neural cells.

Treatment with G-CSF or combined G-CSF/SCF revealed a transcriptional frataxin upregulation in spinal cord and cerebellum of YG8R animals.

The Wilkins lab at the University of Bristol completed a study of a mouse model of FA in which GCSF protected mice from neurological damage.

2017: An open-label, pilot study of recombinant human granulocyte-colony stimulating factor (G-CSF) was conducted 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 was peripheral blood mononuclear cell frataxin levels over a 19-day period. The secondary outcomes include safety, hematopoietic stem cell (HSC) mobilization, antioxidant levels and mitochondrial enzyme activity.

2019: The study demonstrated that administration of G-CSF to people with FA is safe.

Mobilization of HSCs in response to G-CSF was comparable to healthy individuals.

Increases in cellular frataxin concentrations and PGC-1α and Nrf2 expression were detected.

The long-term safety of sustained G-CSF administration in people with FA also unknown.

Clinical benefit was not assessed in this small, short-term trial.