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Exploiting Cellular Metabolism to Develop the Next Generation of mTOR Inhibitors

Project description

Synthetic malonyl-CoA derivatives as advanced mTORC1 inhibitors

The mTOR kinase regulates cellular physiology through the mTORC1 and mTORC2 complexes. Dysregulation of mTORC1 is associated with diseases and ageing. Current mTOR inhibitors have limited efficacy and adverse effects, highlighting the need for better alternatives. Most mTORC1 targets are resistant to rapamycin, and catalytic inhibitors can increase toxicity. Recent research has identified malonyl-CoA as a novel and selective mTORC1 inhibitor that effectively blocks all mTORC1 target phosphorylation without affecting mTORC2 activity. The ERC-funded Inhibitor Project aims to develop synthetic malonyl-CoA derivatives as a new class of mTORC1 inhibitors with enhanced specificity and effectiveness. These inhibitors, which leverage biochemical, structural, and computational data, are expected to have significant scientific, clinical, and commercial applications due to mTORC1’s role in ageing and disease.

Objective

The mTOR (mammalian/mechanistic Target of Rapamycin) kinase, as part of the mTORC1 and mTORC2 protein complexes, is the master controller of cellular and organismal physiology. Dysregulation of mTORC1 activity is tightly linked to human disease and ageing. Consequently, mTOR inhibitors have been tested in various clinical settings, however with little success and limited applicability so far, mainly due to low efficacy or adverse effects, thus highlighting the need for more potent and more specific compounds. For instance, the phosphorylation of most mTORC1 targets is resistant to rapamycin and its analogs (rapalogs). Furthermore, catalytic mTOR inhibitors demonstrate increased toxicity and cause unwanted metabolic effects mainly because they also block mTORC2 activity. We have recently identified malonyl-CoA, a metabolic intermediate of fatty acid biosynthesis, as a novel specific mTORC1 inhibitor in cells that shows improved characteristics over existing compounds as it blocks phosphorylation of all mTORC1 targets without affecting the activity of mTORC2 or other related kinases. Using our biochemical, structural and computational data as the basis for targeted compound development, we aim to develop synthetic malonyl-CoA derivatives as a new class of mTORC1 inhibitors with improved specificity and effectiveness. Because of the well-established role of mTORC1 in ageing and disease, these inhibitors are expected to be of profound scientific, clinical, and commercial interest.

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Topic(s)

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Funding Scheme

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HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants

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Call for proposal

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(opens in new window) ERC-2023-POC

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Host institution

MAX-PLANCK-GESELLSCHAFT ZUR FORDERUNG DER WISSENSCHAFTEN EV
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 150 000,00
Address
HOFGARTENSTRASSE 8
80539 MUNCHEN
Germany

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Region
Bayern Oberbayern München, Kreisfreie Stadt
Activity type
Research Organisations
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Total cost

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Beneficiaries (1)

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