MND is a group of diseases with multiple causes, rather than a single disease with a single target. Tailoring treatments for different subtypes of MND may result in more effective therapies than only looking for a ‘one size fits all’ solution.
Over the short and medium term we aim to:
- Group patients into known subtypes of MND that align with more targeted treatments already in the development pipeline
- Develop biomarkers (chemical or other measurable signatures of processes inside the body) that can tell us whether a treatment is hitting the desired target
- Advance a candidate therapy through an experimental medicine trial. Robust biomarkers and well defined ‘strata’ of MND will help us to do this.
Establishing standardised ways to collect the clinical and biological information needed in order to subtype patients (or ‘stratify the disease’) is already underway with a study called AMBRoSIA systematically collecting patient samples and tracking clinical symptoms over time. Patients coming into the AMBRoSIA study are genetically tested against a panel of known genes that cause MND. Partnering with our Genomic Medicine and Bioinformatics team could help us to define more categories of MND by extending the panel of known genetic risk factors and by understanding how groups of different genes interact with each other. In the longer term, this knowledge may help us uncover new therapeutic approaches for MND as well.
At least 15 Principle Investigators based at the Sheffield Institute for Translational Neuroscience (SITraN) lead their own research team primarily focused on Motor Neurone Disease. SITraN and the Sheffield BRC work together to pull through new discoveries in the basic biology of diseases of neurodegeneration into promising therapies and treatments. Scientists working at the earlier stages of the pipeline aim to uncover new targets for therapy development, and even entirely new strategies for treatment by finding out what is unknown in the fundamental biology of a disease. Partnering with collaborators all the way through the pipeline to Clinicians working at the point of delivering a new treatment to patients aims to streamline the process of translating findings from basic science ‘at the bench’ into clinical improvements ‘at the bedside’.