Using stem cells to understand and treat toxic proteins in frontotemporal dementia.

Frontotemporal dementia (FTD) is a relatively rare form of dementia that is thought to account for around one in 20 dementia cases.

One of the key hallmarks of FTD is the build-up of protein into toxic clumps in the brain. In many cases of FTD the clumps are made up of a protein known as TDP-43.

In normal conditions, many proteins in the body, including TDP-43, can “autoregulate”. This means they have internal systems that change how much protein is present, depending on the environment.

Early research has suggested that the regulation of TDP-43 becomes disrupted in FTD.

Dr Matthew White’s Alzheimer’s Research UK Fellowship will further our understanding of this process. He will use cutting edge stem cell techniques to grow nerve cells in the lab and investigate how they change when the regulation of TDP-43 is disrupted.

His research will also be extended to using the lab-grown nerve cells to study how potential drugs could reverse some of those effects.

This Fellowship will not only build our understanding of a vital process that occurs during FTD but may also identify approaches that could be used in the treatment of the disease.