UAB research into gene therapy for SPG52 receives €100,000 donation
The Irina Poate Association from Romania has donated €100,000 to the UAB's ongoing crowdfunding campaign to investigate a gene therapy that can treat type 52 spastic paraplegia (SPG52). With this new donation, the study carried out by the Gene Therapy Group for the Central Nervous System, in collaboration with the La Lucha de Abril association, has received over €750,000 in donations, 80% of the target currently set to advance the project.
Irina Calacean is one year old and is the only child in Romania diagnosed with SPG52. The Irina Poate Association was created by her parents to raise funds to help the UAB research team that is investigating a gene therapy treatment that could cure or slow down the disease.
According to the association, the donation comes from a large campaign to publicise the girl's case on social networks and the media, driven by Irina's parents and a group of volunteers who helped them share her story. With this contribution, the association "reaffirms its mission to support research into this ultra-rare disease and provide hope to affected children, and demonstrates that solidarity and participation can pave the way for treatments that transform lives".
Irina, only child in Romania to be diagnosed with SPG 52, with her parents.
The crowdfunding campaign so far has raised €762,699, 80% of the necessary funds set to advance the research, which amount to €950,000. The project has obtained the majority of its financial resources from donations made by the La Lucha de Abril association, which has raised a total of €468,751.10 to date.
The study to find a gene therapy for SPG52 was launched by the UAB at the end of 2021 with the collaboration of the La Lucha de Abril association. It is led by researchers Miguel Chillón, ICREA researcher at the Institute of Neurosciences (INc-UAB) and at the Vall d'Hebron Research Institute (VHIR), and Assumpció Bosch, professor at the Department of Biochemistry and Molecular Biology at the UAB. It also included the collaboration of the ”la Caixa” Foundation, which granted student Laura Rodríguez an INPhINIT Retaining predoctoral fellowship.
Last year the first preclinical trials of the study were completed. The study continues to advance with the hope of being able to help these affected children, who are very few in the world and only three in Spain, so that they can gain in quality of life by improving their autonomy, memory, and learning capacity.
"The solidarity of the entities created by the families of the affected children and the selfless donations of many other people is what allows us to carry out the research, which is long and very expensive for diseases as rare as SPG52," explains Miguel Chillón, who showed his appreciation for the support received over these past few years.