Cooperation and technological transfer for the development of a new platform precision medicine for Amyotrophic Lateral Sclerosis

Overview

Project Summary

Amyotrophic lateral sclerosis (ALS) is a rare, progressive, and degenerative neurological disease for which there is no cure, leading to paralysis and death on average between 2 to 5 years after diagnosis. There are significant gaps in the understanding of the pathophysiology of ALS, not only due to incomplete knowledge of the biological mechanisms but also resulting from biological models with limited translation to humans. The diversity of possible causes for the onset of the disease and the pathophysiological heterogeneity among patients result in greater difficulty in finding precise and effective therapies. In this sense, designing therapeutic platforms aimed at precision, according to the particularities of each patient, is of utmost importance. To promote effective advancements in the treatment of ALS, with a strong socio-economic impact on populations, and in compliance with European goals, specifically in line with the common cross-border development strategy, an interdisciplinary consortium of researchers from two world-renowned universities, the University of Coimbra (UC) and the University of Salamanca (USAL), has been established. This consortium benefits not only from the recognized expertise in innovation and knowledge transfer but also from the strong relationship with neurologists from the two specialized ALS hospitals that centralize patients from the Centro region of Portugal and Castilla y León. This project is based on the innovative development of a new 3D model of ALS, using human motor neurons (HMN) co-cultured with human skeletal muscle cells (HSkMC) derived from the same patient, originating from stem cells derived from urine. This is an interdisciplinary project with a strong innovative potential for precision diagnosis and treatment of ALS, using a non-invasive cellular platform. The joint action of the entities involved in the consortium is essential for the execution of this project, not only for the technological and scientific contributions of the researchers from these institutions but especially for access to ALS patients. The geographical proximity provides a cross-border dimension with a strong impact on the success of the project. The technology transfer from UC's laboratories to USAL's will allow the development of a more ambitious cross-border project.

Project Details