Project - Translating biomarkers to the beside, a precision medicine approach to clinical trials in Frontotemporal Lobar Degeneration FTLD and Motor Neuron disease
Research Project Abstract
The proposed study aims to collate a multimodal clinical repository including clinical data, biofluid data, neurophysiological data and neuroimaging and combine this data to develop improved multimodal measures of disease progression in MND and FTD. Using a clinical dataset of over 400 unique participants we aim to identify new subgroups, and therefore improve the predictive power of these biomarkers. Importantly this will allow identification of potential cohorts of participants who may respond to particular interventions, allowing a move towards more precision medicine in these areas. In addition, we will collect prospective novel clinical neuroimaging data, neurophysiological data and clinical data to test new hypothesis with regards to the some of the earliest disease pathobiology in those with MND and FTD.
Project with a disease tag
MND, FTD, and early onset dementia
Challenges within the field
Delay to diagnosis is a major issue, resulting in significant anxiety for patients and delayed access to treatment. This work aims to develop faster ways to diagnose these life limiting conditions and improve the power within drug trials to detect a clinical benefit from novel therapies.
Research Project Description
Clinical "bedside" biomarkers: we will collect detailed data on the clinical neurological finding in those with ALS and FTD. In particular we will identify if novel clinical metrics (such as a composite score reflecting upper and lower motor clinical burden) impact disease progression
Novel clinical measures: we will develop novel measure of disease burden in ALS, with a particular emphasis on non-motor symptoms. We will devise easy to use tools to capture the impact of non-motor symptoms in ALS.
Neuroimaging measures: advanced neuroimaging will be acquired cross-sectionally and longitudinally to establish profiles of neurodegeneration in those with ALS and FTD. Novel approaches to regional disease spread will be applied to better understand the temporal clinical evolution of ALS and FTD
Multimodal analysis: we will combine clinical and neuroimaging datasets to identify the brain basis for the clinical symptoms experienced by patients, providing important insights into disease evolution in ALS and FTD, as well as validating the potential of novel neuroimaging biomarkers.
Translation research: building on the above approaches, we will integrate our research into clinical trials designs to establish the utility of these potential clinical and neuroimaging biomarkers as potential outcome measures for future clinical trial design