ALS disease models and therapeutic drug screen (Dementia Consortium)

This project has been awarded to Marco Baralle, RNA Biology, ICGEB Trieste, Italy

While people with ALS (also known as motor neuron disease or Lou Gehrig’s Disease) and FTLD, the most common cause of dementia in people under the age of 50, experience different symptoms, both diseases are associated with the same biological processes. Both are characterised by the build-up of ‘protein clumps’ – aggregates of misfolded versions of a protein called TAR DNA-binding protein 43 (TDP-43). This build-up can disrupt vital nerve cell communication and eventually cause nerve cell death. Sadly, no treatments exist to halt the spread of damage in these diseases.  The RNA Biology group in Trieste have set out to tackle this lack of treatments and aim to identify agents capable of clearing misfolded proteins, which could form the basis of future much-needed drug development programmes.

The grant from the Dementia Consortium, in  partnership between Alzheimer’s Research UK, lifeARC  and the pharmaceutical companies Eisai and Lilly, will allowed the RNA biology group to develop assays and perform high-content screening in collaboration with LifeARc,  to test libraries of potential compounds for their ability to clear clumps of misfolded TDP-43. By uniting expertise across the continent, the two research teams will work in partnership to test promising drugs in cells and animals, to understand how they work and explore whether they can improve symptoms of ALS and FTLD.

Duration: 2015-2018

Donor/funding programme:

DEMENTIA Consortium


Alzheimer’s Research UK