The Dementia Consortium is streaming £305,000 into a project aiming to discover new therapeutics for Amyotrophic Lateral Sclerosis and Frontotemporal Lobar degeneration, the most common cause of dementia in people aged under 50.
Both conditions are characterised by the build-up of protein clumps, which can disrupt vital nerve cell communication and eventually cause nerve cell death.
The project aims to identify new agents able to clear the misfolded protein TDP-43, which has shown promise as a target in early research, potentially forming the basis of future drug development programmes.
With cash from the Dementia Consortium, a unique charity-private £3-million partnership between Alzheimer’s Research UK, MRC Technology and the pharmas Eisai and Lilly, researchers at non-profit group the International Centre for Genetic Engineering and Biotechnology in Trieste will develop assays and perform high-content screening to test libraries of potential compounds for their ability to clear clumps of TDP-43.
In partnership with MRC Technology, the researchers will then test promising drugs in cells and animals, to understand how they work and explore whether they can improve symptoms of ALS (also known as motor neurone disease or Lou Gehrig’s Disease) and FTLD.
“Our initial research into TDP-43 has showed promising results, with some compounds able to enhance aggregate clearance by more than 50% and fully restore TDP 43 function,” said Marco Baralle, Group Leader in Biotechnology Development at the ICGEB, noting that the funding “will allow us to progress our research in collaboration with MRC Technology to the next stage”.
