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TRAF-STOP therapy to reduCe inflammation in athERosclerosis.

Project description

A novel oral drug for atherosclerosis is moving toward clinical application

Atherosclerosis was long considered a lipid storage disease as it is characterised by an accumulation of cholesterol and other lipids within the artery wall. It is now also recognised as a chronic inflammatory disease involving the immune system. Inhibitors of a key mediator of cell-cell communication in immunity can prevent initiation of plaque formation and retard its progression. However, in the long-term immunity is suppressed as well. TRACER scientists discovered a potentially more specific target downstream of that system that reduces existing atherosclerosis without suppressing immunity. With EU support, they are now paving the way to bring an oral drug to patients. Better success in treating atherosclerosis should help in significantly lowering the incidence of associated cardiovascular diseases.

Objective

Atherosclerosis, the underlying cause of the majority of cardiovascular diseases, is a lipid driven, inflammatory disease of
the large arteries. Despite a 25% relative risk reduction achieved by lipid-lowering treatment, the vast majority of
atherosclerosis induced cardiovascular disease risk remains unaddressed. Therefore, characterizing mediators of the
inflammatory aspect of atherosclerosis is a widely recognized scientific goal with great therapeutic implications. Blocking the
co-stimulatory CD40L-CD40 dyad reduces atherosclerosis. However, long-term inhibition of CD40L or its receptor CD40
results in suppression of the immune system and poses a risk for thromboembolic events. Therefore, we focused on the
downstream signaling pathways of CD40, and found that the interaction between CD40 and TNF-receptor-associated factor
6 (TRAF6) is the driving force for atherosclerosis. Using virtual ligand screening, we identified several small molecule
inhibitors termed TRAF-STOPs that were modeled to bind to the CD40-binding domain of TRAF6. TRAF-STOPs significantly
reduce (existing) atherosclerosis and treatment was well tolerated. The first toxicology results in mice show that there are no
side effects. Here we pursue the hypothesis that TRAF-STOPs are excellent candidates to pass the translational pipeline
towards a clinical application to treat atherosclerotic cardiovascular disease. Prof. Lutgens is one of the founders of the
recently established start-up company Cartesio Therapeutics to be able to valorise our novel TRAF-STOPs. By the end of
the PoC grant, we expect to have an oral drug available and to have completed toxicology and bio-distribution analysis in a
large animal model (mini-pig) and have tested TRAF-STOPs in a pig model of atherosclerosis. This way, we hold a solid
business case in our hands. The resulting business- and (pre-)clinical development plan and patent portfolio will then be
ready for seed investment and venture capital funding.

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Programme(s)

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Topic(s)

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Funding Scheme

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ERC-POC-LS - ERC Proof of Concept Lump Sum Pilot

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Call for proposal

Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.

(opens in new window) ERC-2019-PoC

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Host institution

ACADEMISCH MEDISCH CENTRUM BIJ DE UNIVERSITEIT VAN AMSTERDAM
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
MEIBERGDREEF 15
1105AZ Amsterdam
Netherlands

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Region
West-Nederland Noord-Holland Groot-Amsterdam
Activity type
Higher or Secondary Education Establishments
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Total cost

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Beneficiaries (1)

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