Dementia and neurodegenerative disorders
Key areas of focus
- Natural history studies and patient stratification
- Novel therapeutics
- Biomarker development
Dementia and Neurodegenerative Disorders’ combines and builds on the strengths of the previous theme and will now be part of the Brain and Mind Health super-theme, which will also include ‘the Neuroscience’ and ‘Mental Health’.
The number of patients developing chronic degenerative diseases of the brain leading to dementia and major physical disability is likely to rise as the population ages. Thus, we will conduct research on patients with a wide range of conditions including Alzheimer’s Disease (AD); Vascular dementia (VD); Parkinson’s Disease (PD); Huntington’s Disease (HD); FrontoTemporal Dementia (FTD) as well as a range of other rarer disorders. All of these disorders are characterised by the slow loss of nerve cells in the brain, possibly due to additional problems within the supporting glial cells of the brain as well as local inflammation.
The theme will bring together many different research groups and institutions in Cambridge, many of whom have worked together over years around these diseases.
In particular, we have set ourselves specific goals, which in the short term are:
- To better understand why patients with apparently the same disease do not always follow the same clinical course. Thus, some patients progress quickly while others follow a more benign course. The ability to identify such groups of patients from disease onset is not only critical in helping us stratify individuals into “subtypes” of disease, but also has implications as to how to best treat them. In addition, the ability to classify patients in this way will also enable us to explore why this happens- e.g. what genetic variations underlie these subtypes of disease and how can we exploit this information in developing new therapies?
- To develop and implement novel therapies targeting unmet symptomatic aspects of these disorders. Sadly, we have few drugs that we can use in these types of diseases, but to date some aspects of disease have not been thoroughly explored. So for example, many patients have sleep problems – how can we best investigate and treat this?
- We are striving to better understand how these diseases come about in the first place by studying them across a range of model systems. This work will involve looking at patient derived cells with the hope that we can find new ways to treat them based on a clearer understanding of what fundamentally goes wrong in the cells from patients that actually have gone on to get that condition.
In the medium term, we are hoping to:
- Develop more reliable ways by which to diagnose and follow patients with these diseases. The development of these new biomarkers will also allow us to better subtype patients as well as help in assessing whether new drugs are really impacting on the disease rather than just the symptoms. Furthermore, it may even lead us to diagnose patients ahead of them getting obvious disease, which if possible could allow us to prevent the disease from developing in the first place!
Deliver new experimental therapies to patients that will include;
- (i) Drug repurposing – where a drug that is used in a clinic could be used for another disease e.g. treating a neurodegenerative disorder of the brain
- (ii) The development of new small molecules and drugs that have never been tried in humans before but nevertheless have been shown pre-clinically to safely target the problem in these conditions
- (iii) Gene therapies to replace or correct problems linked to these diseases
- (iv) Cell therapies designed to replace lost cells and help repair the brain, including transplants made from stem cells
- (v) Therapies that interact with the immune systems which may also be driving aspects of the disease process