Gene therapy research for a rare disease affecting children receives over 1,400 donations
The study of a gene therapy for the treatment of spastic paraplegia type 52 (SPG52) being carried out by researchers at the UAB has so far raised €263,000 through a crowdfunding project of the University and the association La lucha de Abril. The research is currently analysing the therapeutic effects of the treatment on mouse models of the disease and on neurons obtained from the stem cells of Abril, a Spanish girl affected by the disease. The association has so far made four donations of €50,000, the last of which was recently given to the project.
06/02/2023
In May 2020, the Universitat Autònoma de Barcelona (UAB) and the association La lucha de Abril began a crowdfunding campaign to research a gene therapy for the treatment of an ultra-rare disease: spastic paraplegia type 52 (SPG52). This disease affects children and there is currently no known cure. Gene therapy research, which has a very high cost and little to no public or private funding, made it difficult to raise the €250,000 needed to conduct the first two study phases. But two and a half years later, this amount has been collected thanks tothe support of 1,430 donations made by 1,230 donors. So far, the association La lucha de Abril has been able to make four donations of €50,000, the last of which was recently given to the project. A total amount of €263,000 has been raised until now. The corwdfunding campaign will continue to be active until the research study reaches its end.
Social collaboration, key to the project
The support of society has been key to continuing with the research. A support that has been administered mainly by the association La lucha de Abril, thanks to countless sports, cultural and social activities organinsed, as well as products sold, and to which thousands of anonymous people have contributed. To carry out these actions, the association had the help of several entities, including members of the Mossos d’Esquadra, the Badalona City Council and the Ochotumbao Foundation, created by actors Clara Lago and Dani Rovira, as well as different private companies. The amount of money donated to this research study so far has been €200,000.
The association La lucha de Abril was created in 2019 by Abril's parents, Jesús Merino and Cristina Crespo, when doctors from the University Hospital Germans Trias i Pujol doagnosed their daughter with SPG52. They decided to speak with a research team in Gene Therapy for the Central Nervous System at the UAB and find a possible treatment capable of curing or slowing down the disease.
This information is related to the following SDG
Good health and well-being