Targeting Metabolic Reprogramming in Polycystic Kidney Disease

What is it about?

Polycystic Kidney Disease is an important inherited disorder of the kidney in urgent need of novel therapies. We discovered that PKD tissues manifest profound metabolic reprogramming to meet their energy demand. The rewiring of energy generation from different sources resembles the one observed in cancer. Based on our observations we have designed two possible therapeutic approaches that work effectively and that can be combined. On the one hand we are trying to repurpose a compound, called 2DG, that competes with glucose and can impair its utilization for energy production by the cells; on the other we are trying to develop a new inhibitor for an enzyme called asparagine synthetase. The first compound has been used in the past on humans and proven to be quite safe. The work with this molecule is quite advanced and we should be able shortly to move onto patients affected by PKD with a first safety study. The second compound has been only utilized in proof of principle studies in the mouse and will need to be fully developed.

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