A potential new gene therapy for Alzheimer’s has been developed by University of Bristol researchers, thanks to funding from Bristol-based charity BRACE Dementia Research. The research breakthrough could help address a condition that affects millions of people worldwide.
Dr Kevin Kemp and his research team at the University have been able to convert the high-risk Alzheimer’s gene, APOE4, into the lower-risk APOE3 gene.
By targeting these genes, the hope is that the therapy could prevent or slow Alzheimer’s in people who carry the high-risk APOE4 gene, which is found in around 1 in 4 individuals.
Dr Kemp’s revolutionary new research used human immune blood cells. Importantly, these immune cells were derived from people living with Alzheimer’s disease who also had the high-risk APOE4 gene.
By using human immune cells, researchers were able to observe responses that were as close to real-life human responses as possible.
Importantly, this innovative approach showed little impact on the rest of the cells’ DNA, suggesting the therapy could be safe with minimal unwanted effects.
These promising results suggest the experimental gene therapy could be suitable for clinical use and would be administered through the bloodstream, bypassing the blood-brain barrier and targeting the whole brain.
It marks a significant development, as some Alzheimer’s treatments have been partially or totally prevented from entering all parts of the brain by this barrier, making them less effective.
Dr Kevin Kemp, Associate Professor in the School of Psychology and Neuroscience at the University of Bristol, said: “Our research is still at an early stage, but we’re really excited by the results we’ve seen so far. By targeting the high-risk APOE4 gene, we hope to tackle one of the key risk factors for Alzheimer’s and ultimately develop a new treatment for the disease.”
Liberty Harrison, CEO of BRACE Dementia Research, added “We welcome this exciting development from Dr Kemp, therapies that can reduce or prevent APOE4 from contributing to the onset of Alzheimer’s represent a major unmet clinical need and are urgently required. BRACE is a small charity, and we are proud of the impact we have had on this crucial area of research.”
Further information
September is World Alzheimer’s Month
- Alzheimer’s disease is the most common type of dementia, causing 60-70% of all dementia cases. NHS Alzheimer’s
- Alzheimer's is not caused by a single gene. NIH’s National Institute on Aging (NIA)
- The APOE gene helps the brain to clear the toxic build-up of amyloid protein, which is found in the brains of people with Alzheimer's. ScienceDirect
- APOE4 is the most significant known genetic risk factor, especially for late-onset Alzheimer's. NIH’s National Institute on Aging (NIA)
- Everyone has two copies of the APOE gene, with one copy inherited from each parent.
- One in four people carry one copy of the APOE4 gene and one in fifty carry two copies. NIH’s National Institute on Aging (NIA)
- Having two copies of APOE4 makes the chances of developing Alzheimer’s ten to fifteen times higher than for someone without them. Science direct
- Having one or two copies of the APOE4 gene does not mean a person will definitely develop Alzheimer’s disease.
- The NHS only offers APOE4 gene testing for a small number of people who have a family history of early-onset Alzheimer’s. NHS Genetic and genomic testing
- Early-onset Alzheimer’s is when the condition develops before the age of 65 and can affect people as young those in their 30s and 40s. Johns Hopkins Medicine
About BRACE Dementia Research
BRACE Dementia Research is a small charity funding world-class clinical and laboratory research into Alzheimer's and other forms of dementia to understand the causes, achieve earlier diagnosis, find and develop better treatments and, one day, a cure.
The charity is based in the Bristol Brain Centre at Southmead Hospital, an NHS hospital.
Since 2000, the charity has:
- Funded more than £12 million pounds of research
- Contributed to more than 140 different projects
- Supported more than 50 PhD students
- Invested £600,000 to enable the Bristol Brain Centre to be built
- Funded a Dementia Research Nurse
- Supported a Consultant Senior Lecturer at the University of Bristol
- Provided core support to the South West Dementia Brain Bank
- Given ongoing funding to the development of Fastball, an early Alzheimer’s test, currently being investigated for NHS use
