Retinitis pigmentosa is an inherited disease characterised by the progressive loss of retinal photoreceptors and, consequently, of vision. Currently, patients affected by this condition do not have effective treatments to stop the disease, which has a significant impact on quality of life. With the aim of identifying new therapeutic strategies to address this pathology, the Fundación Lucha contra la Ceguera (FUNDALUCE) has awarded a grant to a project led by Dr. Maria Llorián-Salvador, principal investigator of the Diabetes and Metabolism research group at Vall d’Hebron Research Institute (VHIR).

Although retinitis pigmentosa is caused by more than 80 genetic mutations that trigger the death of retinal photoreceptors, recent studies suggest that sustained inflammation plays a key role in the progression of the disease. This observation opens the door to new therapeutic approaches based on modulation of the immune system.

A new strategy based on inflammation regulation

The project funded by FUNDALUCE focuses on a specific type of immune cell: regulatory T cells (Treg). These cells play a crucial role in controlling the inflammatory response and are currently being studied as a therapeutic tool in neurodegenerative diseases such as Alzheimer’s disease or multiple sclerosis. The VHIR team has previously obtained preliminary results in animal models*, showing that the absence of Treg cells accelerates retinal damage, while their reintroduction can reduce inflammation and help preserve neurons.

The research will explore two complementary intervention strategies: the systemic expansion of Treg cells through treatments that stimulate their proliferation across the body, and a local therapy approach using viral vectors designed to increase the presence of Treg cells specifically in the retina and the optic nerve. In addition, the team will study the mechanisms by which Treg cells prevent retinal degeneration.

As Dr. Maria Llorián-Salvador explains, “this project explores an innovative therapeutic approach based on inflammation regulation, which is already being studied in clinical trials for other diseases. This will help accelerate the clinical implementation of this potential new treatment for retinitis pigmentosa, with the aim of preserving vision and improving the quality of life of those affected”.

FUNDALUCE 2024 Grant

The FUNDALUCE Grant is an annual recognition of talent and excellence in biomedical research, particularly in the field of inherited retinal dystrophies. This award seeks to support high-impact scientific projects that contribute significantly to the understanding, treatment or prevention of these diseases.

The project was selected following a rigorous evaluation process by the State Research Agency (AEI), which is part of the Ministry of Science, Innovation and Universities, as well as by the Foundation’s Expert Advisory Committee (CAE), and was ultimately ratified by the FUNDALUCE Board of Trustees.

*Institutional Statement on the Use of Research Animals