According to the World Health Organization, to date, fewer than 6% of rare diseases (RDs) have an approved treatment option. In light of this observation, it appears crucial to develop disruptive approaches that allow to find treatments for multiple RDs. To achieve this goal, DREAMS is developing a novel approach combining iPSCs with novel AI technologies to discover drugs which target common mechanisms of rare neuromuscular disorders (NMD). The project will focus on 5 NMDs that share common pathophysiological characteristics related to dysfunctions of autophagy and desmin disorganization.
The DREAMS project is set to deliver high-impact results across short, medium, and long-term horizons in the field of neuromuscular diseases (NMDs). By Month 60, using skeletal muscle cells derived from induced pluripotent stem cells (skMC-iPSC), DREAMS will, 1) generate up to 5 novel biomarkers of the 5 diseases, 2) perform a high throughput drug screen on all 5 diseases to identify 10–50 repurposable drugs and 3) validate 3 lead compounds in animal models. It will also deliver an innovative clinical trial design and platform, engaging over 160 professionals and 400 patients clinicians with the support of a well-renowned association AFM-Telethon. Within 3–5 years post-project, DREAMS will achieve preclinical validation of at least 2 therapies across 5 NMDs, establish 3–5 industry agreements in rare NMDs, and involve more than 500 professionals and 1600 patients. In the long term (>5 years), the project aims to deliver one clinically validated therapy, influence clinical guidelines, and mobilize >85% of professionals and patients in innovation activities, contributing to a projected €50 million investment in new therapies.
Together, these technological and conceptual innovations will allow the DREAMS consortium to accelerate the development of therapies by reducing timelines for small molecule identification (from 3-5 years to 12 months) and lowering drug development costs by 40%. Its focus on the identification of common mechanisms, so it expands the numbers of pathologies and then patients who could benefit from common molecular targets and potential treatments. Through a collaborative ecosystem of researchers, patients, and regulators, DREAMS will establish a regulatory-ready framework with the potential to transform care pathways across Europe for over 430,000 patients with rare NMDs.
