Kinetics-Based Drug Discovery for Protein Misfolding Diseases


Seminario Generale - Chisesi- Tomassoni Prize
by Michele Vendruscolo (University of Cambridge)

Tuesday 19 Oct 2021, 16:00 → 18:00 Europe/Rome
Aula Conversi (Dipartimento di Fisica - Ed. G.Marconi)

The phenomenon of protein misfolding and aggregation is associated with a wide range of human disorders, including Alzheimer’s and Parkinson’s diseases. A central role in these conditions is played by protein misfolded oligomers, which are among the most cytotoxic products resulting from the process of aggregation. It has been very challenging, however, to target these oligomers with therapeutic compounds, because of their dynamic and transient nature. To overcome this problem, I will describe a kinetic-based approach, which enables the discovery and systematic optimization of compounds that reduce the number of oligomers produced during an aggregation reaction. I will illustrate this strategy for the amyloid beta peptide, which is closely linked to Alzheimer's disease. As this strategy is general, it can be applied to oligomers of any protein in drug discovery programmes. 

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