TIRCON (Treat Iron-Related Childhood-Onset Neurodegeneration) is an Integrated Project in the health sector of the Seventh Framework Programme of the European Union (FP7).
TIRCON is dedicated to a group of disorders called Neurodegeneration with Brain Iron Accumulation (NBIA), which are characterized by high iron levels in the brain. This neurological disease usually has a very severe course, with onset in childhood in the majority of cases. There is still poor knowledge about NBIA primarily because it is so rare that collecting significant data is a challenge. Currently, there is no proven therapy to halt or reverse Pantothenate kinase-associated neurodegeneration (PKAN) or any other form of NBIA. This is especially unfortunate as both the iron accumulation in NBIA and the biochemical defect in PKAN are predicted to be amenable to drug-based treatment. Thus, the absence of adequately powered randomized clinical trials is not due to a lack of therapeutic options but to the rarity of the disease, the lack of patient registries, and the fragmentation of therapeutic research worldwide. The grant from FP7 allows TIRCON to address these issues and explore new treatment options for this rare disease.
The project, which is coordinated by the Ludwig-Maximilians-Universität München (LMU), includes 12 other partners from around the world. It aims to set up a structured network for the diagnosis and treatment of NBIA disorders. This is accomplished by bringing together the existing outstanding, but scattered expertise in NBIA research and care throughout Europe and on the international level.
Project aims and fields of work
Through eight complimentary project work packages, TIRCON aims to achieve:
1. the setup of an international NBIA registry
2. the implementation of a biobank
3. the development of biomarkers for the disease
4. the conduction of a randomized clinical trial of the iron-chelating drug Deferiprone in PKAN
5. the preclinical development of new compounds for the treatment of PKAN
TIRCON aims to make an impact on the NBIA community at the international level. This includes doctors and clinicians involved in NBIA care and treatment, basic scientists researching new therapies, patient advocacy groups that provide support to families suffering from this disease, and research companies that can develop the drugs to treat NBIA.
TIRCON connects these groups together to create a comprehensive network, with the end goal of
- providing a virtual community where NBIA clinicians can be connected with patients and provide telemedicine support
- developing a joint education programme to teach young, as well as experienced medical professionals about NBIA
- building an interface with key stakeholders in the national health care systems
As a result, TIRCON will provide better access to NBIA diagnosis and therapy and concurrently contribute to a harmonization of care standards.
BayFOR as a partner
BayFOR, specifically the Scientific Coordination Office Bavaria- Québec/Alberta/International (WKS), provided support throughout the entire application process of TIRCON, such as finding partners for the consortium and contributing essential expertise in the proposal submission process. As management partner in the consortium, BayFOR continually provides administrative, legal and financial support and in addition contributes to the ethical work and dissemination of the project results.
TIRCON has received funding for four years in the amount of €5.215.223. It officially runs from November 2011 to October 2015. In total, TIRCON partners in Bavaria have received €2.823.131.
TIRCON is composed of 13 partner institutes from eight countries around the world. The consortium’s experience and expertise spans across a wide range of areas including clinical science, basic science, biotechnology research, management, and patient advocacy and support.
The participating partner institutes are:
Ludwig-Maximilians-Universität München (LMU), Germany, Coordinator
Technische Universität München (TUM-MED), Germany
Children’s Memorial Health Institute (CMHI), Poland
Foundation Neurological Institute Besta (FINCB), Italy
University Medical Centre Groningen (UMCG), Netherlands
Oregon Health & Science University (OHSU), USA
Children’s Hospital Oakland (CHRCO), USA
Newcastle University (UNEW), England
ACIES BIO (ACIES BIO), Slovenia
NBIA Disorders Association (NBIADA), USA
Hoffnungsbaum e. V. (HoBa), Germany
Bayerische Forschungsallianz GmbH (BayFOR), Germany
ApoPharma Incorporated (ApoPharma), Canada
Prof. Dr. med. Thomas Klopstock
Friedrich-Baur-Institut an der Neurologischen Klinik
Klinikum der Ludwig-Maximilians-Universität München
Phone: +49 (0) 89 5160-7400