Delivering new treatments safely across the brain’s protective barriers effectively

Cristina Fornaguera

Universitat Ramon Llull, Spain

The brain is protected by barriers that prevent harmful substances from entering. While these barriers are essential for brain health, they also make it difficult for many treatments to reach the brain. This project aims to develop new delivery systems capable of crossing these barriers and transporting therapeutic instructions to brain cells. The project will first focus on Lafora disease, a rare childhood neurological disorder for which no effective treatment currently exists.

A team with expertise in nanomedicine, controlled drug delivery, neuroscience and artificial intelligence will design tiny carriers that can travel through the body, reach the brain and deliver their therapeutic cargo safely and efficiently. Computational tools will help prioritise the most promising designs, whose safety and therapeutic potential will be assessed in laboratory testing and animal models.

The project could help develop a treatment for Lafora disease and create a versatile platform for other neurological conditions. In a broader sense, the results from this project would help overcome one of the major obstacles in brain medicine: delivering treatments where they are needed.

• María José Alonso, Universidad de Santiago de Compostela, Spain

• Jordi Duran, Fundació Institut de Bioenginyeria de Catalunya, Spain

• Chelsea's Hope Lafora Children Research Foundation, United States of America

Targeting the brain: mRNA nanotherapeutics for neurological diseases

€998,898.20