GCSF for Friedreich Ataxia
Research type
Research Study
Full title
The pharmacological effects of granulocyte-colony stimulating factor (GCSF) on frataxin expression in patients with Friedreich Ataxia
IRAS ID
220928
Contact name
Alastair Wilkins
Contact email
Sponsor organisation
University of Bristol
Eudract number
2017-003084-34
Duration of Study in the UK
0 years, 7 months, 30 days
Research summary
Friedreich ataxia (FRDA) is an incurable neurological disorder, typically presenting in late childhood, which causes significant disability. There has been much excitement and hope that stem cell therapies might provide an effective treatment for a variety of neurodegenerative diseases including FRDA, and bone marrow stem cells hold significant promise, not least as they have been used extensively for many years for other conditions and thus have the best safety profile. Bone marrow cells can be mobilised and help neurological repair by using cell mobilising drugs, such as GCSF (granulocyte colony stimulating factor) which is in common usage in haematological conditions, as well as in healthy people prior to bone marrow donation. This is a drug that activates stem cells within the bone marrow and induces them to circulate around the body including the brain.
Experimental studies undertaken within our laboratories clearly indicate GCSF protects mice from neurological damage. These drugs have huge potential in neurodegenerative conditions and represent a novel therapeutic possibility for FRDA.
The next stage in our research is to further understand whether GCSF induces similar changes in humans with FRDA. We will study a small number of patients with the condition who have been given GCSF (at identical doses to those given to ‘healthy’ people prior to bone bone marrow donation) for a short period of time. We will define whether administration of the drug leads to changes in blood markers which would indicate a positive response to the drug. The study will also allow us to decide what blood markers we can monitor in the subsequent trial. This has not been studied before and is a vital step in the development of a stem cell research trial. Once information has been obtained from this study, a larger trial of GCSF in FRDA can be developed.
REC name
East of England - Cambridge East Research Ethics Committee
REC reference
17/EE/0486
Date of REC Opinion
22 Jan 2018
REC opinion
Further Information Favourable Opinion