Project description
Bringing a small molecule drug against leukemia to the clinic
T-cell acute lymphoblastic leukaemia (T-ALL) is an aggressive cancer with no effective cure yet. Chemotherapy is partially effective as a first-line treatment, but has significant side effects and cannot kill the cancer stem cells, leading eventually to relapse. The Swiss biopharma company Cellestia has developed a small molecule compound, CB-103, that targets the NOTCH pathway which is aberrantly activated in T-ALL. Preclinical data have shown that CB-103 has a high therapeutic index, significantly prolongs survival rate and has no toxicity. The EU-funded CB-103 project will undertake the necessary activities to plan clinical trials of the CB-103 small molecule in T-ALL patients. Successful development through clinical validation will advance CB-103 toward market approval.
Objective
T-cell Acute Lymphoblastic Leukemia (T-ALL) is an aggressive NOTCH-dependent cancer that affects the blood and bone marrow especially in children aged 0-14 years, and currently has no effective cure. T-ALL accounts for about 25% of the total cases of Lymphoblastic Leukemia (ALL); additionally, an estimated 250,000 people are afflicted by NOTCH-dependent cancers every year. Chemotherapy, the standard-of-care treatment for T-ALL, can reduce tumor growth, but it is ineffective at killing the cancer stem cells that drive tumor progression. Moreover, it can lead to adverse toxicological effects as a result of off-target toxicity, as the drug cannot discriminate between healthy and cancerous cells. Small molecule drugs help to solve this issue thanks to their high efficiency in targeting tumor cells. Cellestia may have found the answer. Its novel approach targets a tumor development pathway, the NOTCH, which play crucial oncogenic roles in tumorigenesis, tumor expansion and survival. By inhibiting the NOTCH signaling in its most downstream part, Cellestia’s small molecule compound, CB-103, blocks the problem at the roots (i.e. specifically at the level of the NOTCH transcription complex) stopping cancer cells growth and survival. Pre-clinical results show CB-103 to have a high therapeutic index relative to conventional cancer drugs. Most importantly, it significantly prolongs survival rates (median improvement of 75%) with no adverse side effects or dose-limiting toxicity (DLT). Results indicate it may have benefit for other cancers too – any in which NOTCH pathway is highly activated, including forms of leukemia and other cancers such as lung, breast and colorectal. The Phase 1 project will be focused on establishing a complete and scalable supply chain, verifying the business model and commercialization strategy, planning new collaborations and partnerships, including the ones to implement the next clinical trials stage.
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.
- social sciences economics and business business and management business models
- medical and health sciences medical biotechnology cells technologies stem cells
- medical and health sciences clinical medicine oncology leukemia
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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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H2020-EU.2.3. - INDUSTRIAL LEADERSHIP - Innovation In SMEs
MAIN PROGRAMME
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H2020-EU.3. - PRIORITY 'Societal challenges
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H2020-EU.2.1. - INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies
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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.
SME-1 - SME instrument phase 1
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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) H2020-EIC-SMEInst-2018-2020
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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.
CH 4057 Basel
Switzerland
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
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.