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Gamma-secretase allosteric modulation as preventive therapy in Alzheimer’s Disease

Project description

Preventive therapy for Alzheimer’s disease

Therapeutic Aβ antibodies have confirmed amyloid plaques as drug targets in Alzheimer’s disease (AD). However, these antibodies are expensive and offer limited benefits. There is a need for a safe, affordable, orally administered drug to prevent brain damage. Gamma-secretase allosteric modulators (GAM) have theoretical potential, but lack experimental evidence for clinical development. The ERC grant Cellphase-AD developed an innovative xenotransplantation model that demonstrates characteristics of AD. The ERC-funded GALOMOD project will treat this model with a newly developed compound to show that GAM can prevent AD pathology. These experiments will support the concept that GAM can be designed as a preventive medication for both sporadic and familial AD, paving the way for clinical trials of the compound.

Objective

The recent advent of therapeutic Aβ-antibodies has validated amyloid plaques as drug targets in Alzheimer’s Disease (AD). They remain expensive, prone to side effects, and clinical benefit is limited. The field needs a safe and cheap drug, preferably a small compound that can be taken orally, and in a preventative way before brain damage has occurred. Despite strong theoretical potential, further clinical development of gamma-secretase allosteric modulators (GAM), is currently hampered by the lack of strong experimental evidence that they are safe and can effectively prevent AD. In this POC we will exploit findings of our ERC grant Cellphase-AD to generate such evidence. We developed an innovative xenotransplantation model beyond the classical mouse models, as it shows not only amyloid plaques, but also the induction of neuro-inflammation, real Tau-pathology, granulovacuolar neurodegeneration, and secretion of Tau biomarkers in the blood, all characteristic of AD (PMID37708272). We will treat this model in collaboration with a contractor with a newly developed compound to demonstrate that this GAM can prevent in a safe and effective way this AD pathology. Using an innovative unbiased ʏ-secretase assay we will provide direct evidence that this GAM, in contrast to the previously failed gamma-secretase inhibitor Semagacestat, maintains normal processing and signalling and does not show mechanistic driven side effects. The experiments aim to support the concept that GAM can be developed as preventative medication in both sporadic and familial AD and to bring the compound forward to clinical trialing.

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Topic(s)

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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

VIB VZW
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
SUZANNE TASSIERSTRAAT 1
9052 ZWIJNAARDE - GENT
Belgium

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Region
Vlaams Gewest Prov. Oost-Vlaanderen Arr. Gent
Activity type
Research Organisations
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Total cost

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Beneficiaries (1)

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