Each cell in our body constantly digests its unwanted or damaged components via the autophagosome-lysosome pathway. When lysosomal function is perturbed, for example in monogenic lysosomal storage disorders, in common familial and sporadic neurodegenerative diseases or as a part of normal ageing process, this leads to the back log in the pathway, accumulation of cellular garbage and eventually cell death. This talk will describe our journey so far, from the identification that autophagy deficit contributes to lysosomal storage Neimann-Pick disease, through the design and validation of a reporter system, to the high throughput screening and identification of FDA approved drugs that can relieve the block in autophagy. Both technical and organisational challenges in coordinating this collaborative drug discovery programme will be discussed.
Scientific Background