Project description
Taking a new leukaemia drug to the clinic
Hypomethylating agents are used in the treatment of leukaemia, particularly acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS), to reverse abnormal DNA methylation patterns that contribute to disease progression. Despite their clinical utility, these agents often face instability issues and limited efficacy, especially in elderly or medically frail patients, highlighting the need for more stable and effective therapeutic options. The ERC-funded leuCAB project will support the preclinical development of carbacitabin (CAB), a novel compound that exhibits improved results and reduced toxicity. The study will undertake structure, safety and efficacy optimisation of CAB, scaling up its production for clinical trials.
Objective
The current treatment landscape for leukemia, particularly concerning AML (acute myeloid leukemia) and MDS (myelodysplastic syndrome), is constrained by the instability of hypomethylating agents which are regularly used in elderly, medically non-fit patients (high-risk). This instability restricts their spectrum of application, leaving a significant gap in effective treatment options. Despite research into alternative therapeutic approaches the need for a mild treatment option with high success rates in this patient population remains unmet.
Carbacitabin (CAB) emerges as a promising solution to these challenges. This novel epigenetically active compound exhibits enhanced stability against hydrolytic cleavage and enzymatic metabolization. In AML PDX mouse models, CAB demonstrates significantly improved efficacy and vastly reduced toxicity, making it an optimal candidate for leukemia treatment, particularly for those high-risk patients.
Funding from the ERC PoC aims to support essential preclinical steps towards fully realizing the potential of CAB. Those include, further optimization of the lead structure of CAB to enhance targeting and thorough preclinical studies to evaluate the safety and efficacy of CAB. Additionally, scaling up the synthesis of CAB is crucial to meet the demand for large-scale production required for preclinical and subsequent clinical trials. Furthermore, efforts to solidify intellectual property (IP) protection and conduct an in-depth freedom-to-operate (FTO) analysis are necessary to safeguard CAB's market position. Establishing a spin-off company dedicated to the clinical studies of CAB will require engagement with essential stakeholders, including potential investors, pharmaceutical partners, legal advisors, and regulatory consultants during leuCAB. This comprehensive approach will ensure the effective translation of CAB from bench to bedside, bringing new hope to patients, especially those battling AML and MDS.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
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Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.1.1 - European Research Council (ERC)
MAIN PROGRAMME
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Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) ERC-2024-POC
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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.
80539 MUNCHEN
Germany
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.