Date: November, 2015
Over 65 researchers, clinicians, NBIA family members and representatives of patient organizations were on hand for the final TIRCON meeting Oct. 15 and 16 in Munich, Germany, to take stock of all that has been accomplished over the past four years.
Treat Iron-Related Childhood-Onset Neurodegeneration, or TIRCON, was a European Union- funded research project for 5.2 million euros, or approximately $7 million. The grant started on Nov. 1, 2011, and ended Oct. 31, 2015 with a focus of advancing research into Neurodegeneration with Brain Iron Accumulation (NBIA) disorders.
The European Union considered TIRCON a model of collaboration for others to follow. This four-year project had 13 partners from eight countries, consisting of nine research institutions, a pharmaceutical and small biotech company, our organization and Hoffnungsbaum e.V., the German NBIA patient organization. All of the partners were in Munich.
Over the four years, partners have held monthly conference calls, eight in-person meetings, and published over 30 scientific publications on their TIRCON-funded work. There were presentations on each of the eight work packages during the two-day meeting and also scientific presentations sharing results of work performed under this project.
TIRCON has accomplished the following goals:
- Created an international patient registry and biobank. The registry has more than 280 patients and more than 500 blood and tissue(??) samples for a biomarker study.
- Conducted a multicenter clinical trial for deferiprone in four countries, with 89 PKAN individuals.
- Generated a framework for future studies of a pantethine derivative as a therapy for PKAN, the most common form of NBIA.
- Created a single global network of NBIA researchers who will continue to work together.
- United the NBIA patient advocacy organizations around the world as the NBIA Alliance.
- Held five workshops on NBIA disorders to educate clinicians on these rare disorders.
- Created clinical centers in Hungary, Serbia, Czech Republic, Spain, Iran and Turkey to enroll their NBIA patients in the deferiprone clinical trial and have these centers contribute to the patient registry and biobank.
The expected impact of these accomplishments is a translation of research results into clinical practice, clinical trial readiness for all NBIA disorders, an expanding NBIA clinical network of centers, and increased knowledge, with continued dissemination of TIRCON research results.
Even though the grant has ended, the deferiprone clinical trial will continue until the last patient has completed the 18-month trial in fall 2016. After that, the work to interpret the results of the trial will begin.
The international NBIA patient registry and biobank will also continue, with the goal to continually add NBIA patients and collect natural history studies at six-month intervals. The NBIA Alliance is committed to this important project and is working on joint funding by Alliance members as one way to make this feasible. Additional grant funding for this purpose is also a goal that the TIRCON partners are pursuing.
TIRCON has strengthened the ties of everyone in the NBIA community and helped build a strong foundation for the future. Everyone attending agreed nothing was “final” about the meeting. The first post-TIRCON meeting preparations already are underway.