Today, the Bobby and Robine Foundation officially transferred the cheque for 244,000 euros to the Kinder UKE in Hamburg – Germany . With this donation, the UKE start the immune project.
The immune project will address the role of the immune system in disease development and response to therapy. The aim of the project is to help to develop better and safer therapies for children with metabolic diseases.
Goals of the Immune project
In one third of the patients with CLN2, the immune system reacts against the replacement enzyme, reducing the efficacy of the treatment. Prof dr. Tolosa: “We need to understand which parts of the enzyme are prone to trigger an immune response and in which patients. The goal is to develop new forms of the enzyme that are equally active, but less enticing for the immune system.”
The role of the immune system in the pathogenesis of the disease is suspected, but has not been addressed. Tolosa: “We plan a deep molecular analysis of the cellular pathways affected by the loss of the protein TPP1 in cells of the immune system, with the aim of finding new therapeutic targets.”
Enzyme substitution therapy for children with CLN2 is currently the best treatment option, but the administration is not easy for the patients, and it mitigates, but does not cure, the disease. The goal of this project is to explore new therapeutic pathways and to advance towards a safe gene therapy approach.
Dr. med. Angela Schulz
Prof. Dr. Eva Tolosa