Biomarkers of aHSCT
Research type
Research Study
Full title
Identifying Immune Biomarkers of Disease and Disease Control in Autoimmune Neurological Disease using Autologous Haematopoietic Stem Cell Transplantation
IRAS ID
317556
Contact name
Gavin Brittain
Contact email
Sponsor organisation
Sheffield Teaching Hospitals NHS Foundation Trust
Duration of Study in the UK
3 years, 5 months, 31 days
Research summary
Several neurological diseases are thought to be caused by the patient’s own immune system mistakenly attacking the nervous system resulting in profound disability. Conditions include multiple sclerosis (MS), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), myasthenia gravis (MG), stiff person syndrome (SPS) and neuromyelitis optica (ON). No cure exists and profound disability can occur.
MS is the most common condition and whilst there are several highly effective disease modifying therapies (DMTs), they are effective in stopping the disease in only some of the patients. Significant side effects, which accumulate over time, and treatment failure can occur.
Autologous haematopoietic stem cell transplantation (aHSCT) has been increasingly used for these conditions, particularly MS, as a highly successful one-off treatment for select patients. The faulty immune system is ‘deleted’ with chemotherapy and then ‘rebooted’ using patients’ own stem cells (a cell with the unique ability of being a building block to create many different cells) to stop further damage. It may be more successful than DMT but unfortunately, a small portion of people do not respond optimally and continue to accumulate disability. There is an initial increased risk of side effects, compared to DMT, meaning treatment should be used only in appropriate patients.
We do not appreciate the mechanism of aHSCT, how to detect which patients will benefit, or how to monitor response. We propose to investigate the immune system which is found in the stem cells, the fluid surrounding the brain and spinal cord, blood and stool of patients who are already undergoing aHSCT or receiving DMT and compare them to clinical markers as part of this observational study. This will improve understanding of the mechanism of action of aHSCT, how it can be refined, and suggest markers (biomarkers) which can potentially predict treatment success or failure before patients are exposed to the risks.
REC name
South Central - Hampshire B Research Ethics Committee
REC reference
23/SC/0020
Date of REC Opinion
24 Feb 2023
REC opinion
Further Information Favourable Opinion