Descripción del proyecto
Desarrollo de tratamientos innovadores para los trastornos neuromusculares raros
Hoy día, menos del 6 % de las enfermedades raras cuentan con terapias aprobadas, por lo que la Organización Mundial de la Salud destaca la necesidad urgente de desarrollar nuevos tratamientos. En el proyecto DREAMS, financiado con fondos europeos, se abordará esta cuestión mediante el diseño de terapias para cinco trastornos neuromusculares raros con rasgos fisiopatológicos parecidos. El planteamiento innovador del proyecto consiste en utilizar células musculares esqueléticas derivadas de células madre pluripotentes inducidas para descubrir biomarcadores comunes a estos trastornos. Además, se empleará la inteligencia artificial (IA) para identificar dianas terapéuticas comunes y posibles fármacos a partir de cribados fenotípicos de fármacos y datos ómicos. La plataforma resultante combinará la IA y células madre pluripotentes inducidas para desarrollar tratamientos con moléculas pequeñas aplicables a diferentes trastornos neuromusculares, estableciendo así una nueva pauta en la investigación terapéutica de las enfermedades raras.
Objetivo
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 targets shared 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 diseases that 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.
Ámbito científico
Palabras clave
Programa(s)
Tema(s)
Convocatoria de propuestas
HORIZON-HLTH-2022-DISEASE-06-two-stage
Consulte otros proyectos de esta convocatoriaRégimen de financiación
HORIZON-RIA - HORIZON Research and Innovation ActionsCoordinador
91100 Corbeil-Essonnes
Francia
Organización definida por ella misma como pequeña y mediana empresa (pyme) en el momento de la firma del acuerdo de subvención.