Building on the strength of data provided by DPUK, the clinical studies register will provide a critical link to much-needed experimental medicine studies to better understand the disease and potential treatments.
The case for a new generation of highly targeted and highly informative clinical studies in dementia is strong.
Although dementia is the disorder with the greatest unmet need, over the last 20 years there has been a near 100% failure in the development of new treatments. While many reasons underlie this failure, they centre on an inability to recruit well characterised individuals – such as those from cohorts, who we know so much more about than the general public – to experimental medicine programmes and early phase trials.
To make possible a new generation of highly targeted clinical studies, DPUK proposes to establish a register of highly characterised individuals who have agreed to join the DPUK clinical studies register (CSR). It will provide increased academic opportunity and renewed pharma interest in developing new dementia therapies.
How it will work
The three key design elements of the CSR are:
- Providing fine-grained risk stratification: Although most studies will need to access individuals at increased risk of dementia, for comparison, access to low risk individuals will also be required. Of greatest interest are individuals with brain imaging, particularly if the brain imaging has been repeated. However, individuals with existing cognitive data are also highly informative.
- Access to existing data: The CSR will request from cohorts a limited number of variables relevant to dementia risk. These will include age and family history of disease (including dementia), selected genetic information, selected lifestyle data (exercise, smoking, and alcohol), MRI, mood and cognition. These will be used to inform risk stratification algorithms and implement inclusion and exclusion criteria for proposed clinical studies.
- Web-based enhancement of existing data: The value of the CSR for experimental medicine lies in its ability to characterise early disease status. The rate of progression from prodromal disease (the period between the appearance of first symptoms and the full development of the disease) through mild cognitive impairment to dementia is highly variable and frequent measurement is required to capture individual trajectories. The CSR will use six monthly web-based repeat assessment to assess changes in cognition, mood and everyday functioning. The data will be made available to cohorts.