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Development of brain-permeable masked nanobody-drug conjugates to eliminate glioma stem cells

Objective

Glioblastoma multiforme (GBM) is one of the most prevalent brain tumours and is currently incurable. Patients die from tumour recurrence after resection and chemotherapy. New treatments need to overcome two great challenges: reaching the tumour margins, where the blood-brain barrier (BBB) is intact, and eradicating glioma stem cells (GSCs), which cause tumour relapse. Targeting receptors overexpressed on cancer cells with antibody-drug conjugates (ADC) has shown great promise in cancer treatment. However, most receptors on GSCs are also present in normal stem cells. Thus, targeting these receptors with antibody-drug conjugates may lead to severe side effects. The main objective of this proposal is to develop a masked nanobody-drug conjugate with the capacity to overcome the BBB and to be activated only in the tumour to selectively target GSCs. Nanobodies have increased capacity to penetrate tissues and biological barriers but none is yet available to target the GSC marker CD133. Therefore, I will generate new nanobodies against this receptor. Subsequently, I will set up a phage display platform to develop masked nanobodies that can be selectively unmasked by tumour proteases. The masked nanobody will be conjugated to a drug and then to a BBB-shuttle peptide. A panel of conjugates will be screened in vitro for target selectivity, efficient activation and BBB transport. Finally, the efficacy of the most promising candidates will be tested in a GBM mouse model. Our unique combination will generate the first bispecific nanobody-drug conjugates targeting GSCs to treat GBM. The concept of GSC-targeting with activatable ADC may change the current paradigm for the treatment of brain tumours. Furthermore, our workflow may be applied to dramatically enhance the efficacy of other biotherapeutics with on-target off-site dose-limiting effects.

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Keywords

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Programme(s)

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Topic(s)

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Funding Scheme

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HORIZON-TMA-MSCA-PF-EF - HORIZON TMA MSCA Postdoctoral Fellowships - European Fellowships

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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) HORIZON-MSCA-2021-PF-01

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Coordinator

INSTITUT QUIMIC DE SARRIA
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.

€ 165 312,96
Address
CALLE VIA AUGUSTA 384-394
08017 Barcelona
Spain

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Region
Este Cataluña Barcelona
Activity type
Higher or Secondary Education Establishments
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Total cost

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.

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