Correcting a genetic error in the patient's blood stem cells can mean a lifetime cure for a number of rare genetic diseases. Science is currently working on several promising gene therapies, yet the majority ultimately does not reach the patient. This project aims to make this treatment better and in a permanent way available to patients. To this end, the consortium will develop a platform infrastructure that can be used for many different diseases.
Public acceptance
At the same time, CURE4LIFE tackles the blockages with regard to the regulation, reimbursement and public acceptance of these therapies in a knowledge centre for dialogue with society. For example, VU researchers will examine the changing quality of life of patients and their parents through gene therapy. Bartels: "The effects on quality of life are often ignored in medical research projects. It is a wonderful step forward that we are now going to work together, because in the end a good quality of life is the most important outcome of a possible therapy."
Different disciplines
The questions that the consortium wants to answer are not only relevant for stem cell gene therapies, but for many other regenerative treatments. Consider, for example, adapted immune cells as a treatment for leukemia. This large consortium brings together many different disciplines and aspects of gene therapy. "The intention is to bring the biomedical and social sciences together on this subject. We do this very practically by letting researchers from the social corner work for a few days in the laboratory, and vice versa." According to Staal, this connection is crucial to achieve biomedically anchored gene therapies that are accepted in society. "The goal is to bring a gene therapy treatment to the clinic for more diseases in five years so that we can offer more patients a lifelong cure," staal concludes.
The consortium consists of various patient associations and Erasmus MC, Utrecht University, Avans University of Applied Sciences, UMCU, VU University Amsterdam, Radboud University Nijmegen. The consortium also receives support from Batavia Biosciences, ISHEO, Miltenyi and Zorginstituut Nederland. The NWO award falls within the programme of the Dutch National Research Agenda Research on Routes by Consortia (NWA-ORC).