Developing an innovative gene therapy for central nervous system disorders

Clévio Nóbrega is the director of and a researcher at the Biomedical Research Centre (CBMR). Within the centre, he also manages the Laboratory for Molecular Neuroscience and Gene Therapy, where several research projects are currently underway, including PolyQ-ACT.

This project aims to investigate, develop and test an innovative gene therapy treatment for a group of incurable diseases that affect the central nervous system and, more specifically, the brain.

No treatments currently exist to delay or stop the progression of these terminal diseases. The available treatments merely address the symptoms, which means there is an urgent need to develop innovative therapies.

“Our laboratory has identified a new therapeutic target related to the progression of these diseases, which are known as polyglutamine diseases,” says the researcher. So far, we’ve already seen results for three of the nine diseases in the group, demonstrating that gene therapy treatment focussed on this new target can significantly delay the progression of the disease. The findings of the PolyQ-ACT project may pave the way to a standard treatment being found for most polyglutamine diseases, thus contributing to improving patient quality of life.

Although it is difficult to predict the future and the impact of these projects, Clévio Nóbrega and his team are hopeful that the findings of the PolyQ-ACT will enable a treatment to be developed that clinics will be able to treat their patients with in the future.

“We are confident, not least because we were one of the 10 finalists nationwide in an idea accelerator programme run by Roche (Building Tomorrow Together), which aims to bring innovative neuroscience ideas into the marketplace,” he confides.

According to the World Health Organisation, there will be over 2 billion people aged 60 or over in 2050. This increase will also lead to an exponential growth in age-related diseases, specifically neurodegenerative diseases.

As such, this project is aligned with United Nations Sustainable Development Goal 3 of the 2030 Agenda; Good Health and Wellbeing.

Clévio Nóbrega has a Bachelor’s Degree in Biology a PhD in Molecular Biology and Cytogenetics and is the director of and a researcher at the Biomedical Research Centre (CBMR) of the University of Algarve.