Project description
Tailored therapies for Parkinson’s and Huntington’s disease
Increased life expectancy has led to more age-related neurodegenerative diseases, challenging the brain’s regenerative capacity. Stem cell therapies show promise in treating neuronal loss in Parkinson’s and Huntington’s disease. Still, their benefits are limited by a generic approach that does not address individual patient needs. The ERC-funded Custom-Made project aims to develop tailored stem cell therapies. It will integrate stem cell technologies, genomics, and bioengineering to create specialised grafts of therapeutic neurons and supportive cells. It will refine graft connectivity in preclinical models and utilise a humanised 3D bioengineered system for in-depth studies on graft integration. Additionally, the project will innovate self-regulating cell products and introduce grafts with disease-modifying properties.
Objective
The rising incidence of age-associated neurodegenerative diseases, due to increased life expectancy, poses a challenge to the brain's limited regenerative capabilities. Stem cell-based therapies offer promise for addressing crucial neuron loss, particularly in Parkinson’s and Huntington’s diseases (PD and HD). Ongoing first-generation stem cell trials, replacing damaged neurons with healthy ones, represent a notable advancement. However, their impact is constrained by a ‘one-cell-fits-all’ generic therapy that does not take in account the diverse clinical needs due to variations in disease etiology, stage, patient heterogeneity, and daily fluctuations in graft requirements.
CUSTOM-MADE is a transformative initiative advancing knowledge and crafting a tailored portfolio of stem cell products for individual patients or patient subgroups, notably targeting PD and HD. Integrating stem cell technologies, genomics and bioengineering, CUSTOM-MADE develops rational graft composites of therapeutic neurons and complementary cells to meet the clinical needs of individual patients. Moving into preclinical animal models, the project refines graft connectivity and functional recovery post-transplantation. A groundbreaking leap involves modeling circuitry reconstruction in a fully humanized 3D in-vitro bioengineered system - the connectoids, allowing detailed studies at cellular and molecular levels for defining factors controlling correct and functional graft integration. For increased translatability, CUSTOM-MADE pioneers the generation of cell products whose activity can be self-regulated or tuned to the patients’ individual requirements. The project implements strategic measures to shield grafts from pathology and introduces the innovative concept of grafted cells exhibiting disease-modifying activity. In essence, CUSTOM-MADE drives the field toward customized stem-cell therapies for PD and HD, marking a paradigm shift in the treatment landscape of neurodegenerative diseases.
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
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)
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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-SYG - HORIZON ERC Synergy 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-SyG
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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.
20122 Milano
Italy
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.