TIRCON research project
TIRCON was an international research project in which Hoffnungsbaum e.V. acted as an important partner.
During the four years, important research projects were initiated for the future:
Deferiprone Study
The TIRCON project focused on a clinical therapy study involving PKAN patients and the iron-binding substance Deferiprone. PKAN patients had the opportunity to participate in the study until March 2015. The study lasted 18 months and was conducted at five different clinics in Europe and North America.
The study investigated whether Deferiprone, when taken orally, could reduce the iron deposits in the brain that are typical of PKAN. It also examined whether it had a positive effect on the course of the disease.
The publication of the study resultsoccurred in 2019. These results suggest that the active substance Deferiprone can slow down the progression of the disease, especially in atypical PKAN.
NBIA Patient Registry and Biobank
Another core task of TIRCON was to establish an internationally integrated NBIA patient registry and an NBIA biomaterial bank for NBIA patients. Both serve to collect data on the course of the disease and the effects of NBIA, which is important for further research.
Both the patient registry and the biobank will continue to be maintained beyond the end of the TIRCON project. Read more under NBIA Patient Registry and Biobank.
Further goals of TIRCON
The project also aimed to improve the treatment and care of NBIA patients by:
- Transferring research results into clinical practice
- Exchanging knowledge about diagnosis and therapy between doctors, patient representatives and healthcare organizations
- Promoting drug development for PKAN/NBIA
- Training and supporting young scientists and clinicians in dealing with PKAN/NBIA to ensure sustainable and high-quality research and care in the future
- Improving the infrastructure for NBIA patients by networking clinical NBIA centers
The role of Hoffnungsbaum e.V.
In total, thirteen project partners from eight countries were involved, including basic researchers, clinicians, representatives from pharmaceutical companies and the patient organizations NBIA Disorders Association and Hoffnungsbaum e.V.. Hoffnungsbaum e.V. took on tasks in the area of public relations and information dissemination:
- Creating information material or supporting publications on NBIA and TIRCON for various target groups
- Maintaining the project website
- Acting as an interface to all stakeholders in the field of health policy for rare diseases
- Supporting the training and further education of young doctors and scientists
Together with the American NBIA Disorders Association, we had the opportunity to actively bring the patients' perspective into the research network and represent their interests.
