Research project TIRCON
TIRCON was an international research project in which Hoffnungsbaum e.V. as an important partner.
During the four years, important research projects for the future were launched:
The TIRCON project focused on a clinical therapy study in PKAN patients with 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 can break down the iron deposits in the brain typical of PKAN by taking it orally (by mouth). A possible positive effect on the course of the disease was also investigated.
2019 saw the publication of the Results. They indicate that the active ingredient deferiprone can delay the course of the disease, especially in atypical PKAN.
Another core task of TIRCON was the establishment of an internationally merged 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 diseases, which are important for further research.
Both the patient registry and the biobank will continue to operate TIRCON beyond the end of the project. Read more at NBIA patient registry and biobank.
Other goals of TIRCON
The aim of the project was also to improve the treatment and care of NBIA patients by:
- Transfer of research results into clinical practice
- Knowledge exchange on diagnosis and therapy between physicians, patient advocacy groups and health care organizations
- Promoting drug development for PKAN/NBIA
- Training and promotion of young scientists and clinicians in the use of PKAN/NBIA to ensure sustainable and high-level research and care in the future
- Improving infrastructure for NBIA patients by connecting NBIA clinical centers
The role of Hoffnungsbaum e.V.
Overall, thirteen project partners from eight countries, including basic researchers, clinicians, representatives of pharmaceutical companies and the patient organisations NBIA Disorders Association and Hoffnungsbaum e.V.. Hoffnungsbaum e.V. took on tasks in the field of public relations and information dissemination:
- Preparation of information material or support of publications on NBIA and TIRCON for different target groups
- Maintaining the project website
- Interface to all stakeholders in the field of rare disease health policy
- Support for education and training for junior physicians and scientists
Together with the American NBIA Disorders Association, we had the opportunity to actively bring the patients' perspective into the research network and to represent their interests.