DREAMS

Acrónimo: DREAMS

Objetivo temático: According to World Health Organization, to date, fewer than 6% of rare diseases (RDs) have an approved treatment option. In the light of this observation, it appears crucial to develop disruptive approaches that allow to find treatments for multiple RDs. To achieve this goal, DREAMS will start from 5 rare neuromuscular disorders (NMD) that share common pathophysiological characteristics related to dysfunctions of autophagy and desmin disorganization

Área Científica: Induced pluripotent stem cells, artificial intelligence, neuromuscular disorders, autophagy, phenotypic drug screening, virtual drug screening, adaptive clinical trial

Síntese do Projeto: According to World Health Organization, to date, fewer than 6% of rare diseases (RDs) have an approved treatment option. In the light of this observation, it appears crucial to develop disruptive approaches that allow to find treatments for multiple RDs. To achieve this goal, DREAMS will start from 5 rare neuromuscular disorders (NMD) that share common pathophysiological characteristics related to dysfunctions of autophagy and desmin disorganization. Using skeletal muscle cells derived from induced pluripotent stem cells (skMC iPSC), DREAMS will i) identify shared biomarkers of the 5 diseases and ii) perform a high throughput drug screen on all 5 diseases. Through a first Artificial Intelligence (AI) based solution, the output of the drug screening will be used to find drug targetsshared between the diseases. A second AI based solution will be used to find drug candidates (repurposable drugs and new molecular entities) for these shared drug targets. A third AI-based solution will be used to find additional diseasesthat could be treated through the shared drug targets, in order to extend the indications for the most promising drug candidates. Following their discoveries, the promising drug candidates will be tested for efficacy and safety in both iPSC and mouse models of the 5 NMDs and additional diseases. In parallel, DREAMS will design an adaptive clinical trial to prepare the final stages of drug development of these novel drug candidates with multiple indications. Together, these technological and conceptual innovations will allow the DREAMS consortium to i) develop therapies to treat multiple NMDs and related diseases, ii) to create a novel clinical design that generates a regulatory pathway for drugs that treat heterogeneous groups of patients suffering from RDs iii) to further the scientific understanding of commonalities in RDs, and iv) to create a reusable drug development platform to discover safe and effective treatments for RDs in general.

Área de intervenção: Induced pluripotent stem cells, artificial intelligence, neuromuscular disorders, autophagy, phenotypic drug screening, virtual drug screening, adaptive clinical trial

Investigador Responsável na UC: Lino Ferreira

Unidade Orgânica UC: Reitoria - CNC

Instituições participantes no Projeto:

1. CECS (Centre d’Etude des Cellules Souches) - Proponente
2. Universidade de Coimbra
3. KANTIFY (Meetsies SA)
4. INSERM (Institut National de la Santé et de la Recherché Médicale)
5. APHP (Assistance Publique - Hôpitaux de Paris)
6. TECHNION (Technion - Israel Institute of Technology)
7. SAMSARA (Samsara Therapeutics Ltd)
8. AFM Téléthon (Association Française contre les Myopathies)
9. ZABALA (Zabala Innovation Consulting, S.A.)

Instituição Financiadora/Gestora: Comissão Europeia

Programa de Financiamento: Horizon Europe Programme, call "Tackling diseases - HORIZON HLTH-2022-DISEASE-06-two-stage

Período de execução: De 01/11/2023 a 31/10/2028 (60 meses)

Custo total elegível (EUR): 6 952 110,00 €

Apoio financeiro da UE: 6 952 110,00 €

Técnico do Projeto: Maria Joana Dias