Research project TIRCON
TIRCON was an international research project in which Hoffnungsbaum e.V. acted 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 hospitals in Europe and North America.
The study investigated whether deferiprone can break down the iron deposits in the brain typical of PKAN by oral administration (by mouth). A possible positive effect on the course of the disease was also investigated.
2019 saw the publication of the Study results. They suggest that the active ingredient deferiprone may delay disease progression, particularly in atypical PKAN.
NBIA patient registry and biobank
Another core task of TIRCON was the establishment of an internationally merged NBIA patient registry and a NBIA biomaterial bank for NBIA patients. Both serve to collect data on the course and effects of NBIA diseases that are important for further research.
Both the patient registry and biobank will continue beyond the end of the TIRCON project. Read more at NBIA patient registry and biobank.
Other goals of TIRCON
The project also aimed to improve the treatment and care of NBIA patients by:
- Transfer of research results into clinical practice
- Knowledge sharing about diagnosis and treatment among physicians, patient advocacy groups, and health care organizations
- Promotion of drug development for PKAN/NBIA.
- Train and encourage junior scientists and clinicians in the use of PKAN/NBIA to ensure sustainable and high-level research and care in the future.
- Improve infrastructure for NBIA patients by networking NBIA clinical centers.
The role of Hoffnungsbaum e.V.
In total were thirteen project partners from eight countries, including basic researchers, clinicians, pharmaceutical company representatives, and patient organizations NBIA Disorders Association and Hoffnungsbaum e.V.. Hoffnungsbaum e.V. took on public relations and information dissemination tasks:
- Preparation of information material or support of publications on NBIA and TIRCON for various target groups.
- Maintaining the project website
- Interface with all stakeholders in the field of rare disease health policy.
- Support of education and training for young physicians and scientists
In doing so, 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.