Immunotherapy for HCHWA-D
Research type
Research Study
Full title
Immunotherapy for HCHWA-D
IRAS ID
247978
Contact name
L Kelly
Contact email
Sponsor organisation
University of Southampton
Duration of Study in the UK
1 years, 6 months, 22 days
Research summary
Our brains can be damaged in various ways, including stroke and haemorrhage. These can lead to the death of brain cells, reducing significantly the quality of life of the patient and death. One cause of stroke and haemorrhage in aged people is cerebral amyloid angiopathy (CAA), where waste products under the name amyloid build up in the walls of blood vessels and makes them more likely to bleed.
The cause of CAA is largely unknown. However, there are some genetic mutations which we know cause hereditary CAA. One of these mutations is the hereditary cerebral haemorrhage with amyloidosis, Dutch type (HCHWA-D), known as the Dutch mutation. People who carry this mutation will develop CAA and subsequently stroke/ haemorrhage regardless of lifestyle and environmental factors, tragically under the age of 50. No treatment, either preventative or curative, currently exists.
United Neuroscience have developed an anti-amyloid beta vaccine (UB311) which causes the body to produce antibodies. The antibodies break down amyloid beta and allow it to be cleared from the brain. This could potentially treat those with CAA, including those carrying the Dutch mutation. The antibodies would break down amyloid beta which is built up in the walls of the blood vessels and clear it from the brain; this would reduce the risk of stroke of bleeding.
We will use post mortem brain tissue donated from people carrying the Dutch mutation and ethically sourced by neuropathologists in The Netherlands to test whether the antibodies produced from the vaccine are specific to the amyloid beta built up in these patients. Knowing the treatment is specific would allow further studies to test the drug in people.
REC name
HSC REC B
REC reference
18/NI/0161
Date of REC Opinion
29 Aug 2018
REC opinion
Favourable Opinion