In 2020, the last NBIA scientists' meeting took place only online due to the pandemic. In October 2022, for the first time since 2017, numerous actors in NBIA research finally met in person again for 3 days for the 8th International NBIA Symposium in Lausanne.
Group photo with participants at the 8th NBIA Symposium 2022 in Lausanne
The conference attracted 80 official participants from 14 countries, 28 of whom were speakers. Including the guests, e.g. patient representatives, 91 people participated. Even though the 2020 online symposium had significantly more participants with 160 participants from 26 countries, a face-to-face meeting in a confidential setting offers much better conditions for individual scientists to exchange ideas with each other, even informally, about their NBIA projects and project ideas. It is not uncommon for such personal encounters to later lead to scientific collaborations.
The NBIA Disorders Association has awarded eight travel grants totaling $5,000 to early career scientists who would otherwise not have been able to afford to participate. The young researchers were able to present their project results or disease-specific information in the form of posters at the symposium.
Participant Patricia Wood, founder and president of the NBIA Disorders Association, concludes: "Several junior scientists subsequently sent me emails telling me how valuable this experience was and that it motivated them to continue working on NBIA. It also gave them ideas for research and helped them establish collaborations with other participants."
Wood reports lively discussions with many questions and ideas. Networking fostered interest in cooperation in order to further explore some topics in mutual exchange. The event focused on the more common NBIA diseases such as PKAN/CoPAN, MPAN, BPAN, PLAN/INAD and FAHN.
The main topics of discussion were:
• Disease mechanisms and therapeutic approaches for NBIA diseases
• Tailor-made animal and cell models for preclinical tests
• Specific rating scales for clinical trials, based on natural disease progression
• Clinical therapy studies
A committee of scientists led by Thomas Klopstock from the Ludwig-Maximilians-University of Munich had prepared the program of the symposium. In addition to Susan Hayflick of the Oregon Health & Science University in Portland, Valeria Tiranti of the Neurological Institute C. Besta in Milan and Agnès Rötig of the Institute Imagine in Paris, patient representatives were also HoffnungsbaumChairman Markus Nielbock and NBIA Disorders Association Chair Patricia Wood (USA) are members of this body. On site in Lausanne, the patient organisation NBIA Suisse, led by Fatemeh Mollet, organised the event, which had been postponed twice due to the pandemic, to ensure that everything ran smoothly.
Image: NBIA Alliance representatives at the symposium
L-R: Roberta Scalise, AISNAF, Fatemeh Mollet, NBIA Suisse, Joost Schimmel, Stichting Ijzersterk, Patricia Wood, NBIA Disorders Association.
A meeting of the NBIA Alliance was also held on the sidelines of the symposium. The NBIA Alliance is a network of international NBIA patient organisations that was set up as part of the EU research project TIRCON and now has 10 members. The patient representatives discussed possible joint projects for the future as well as an update of the NBIA Alliance website. The NBIA Alliance also had a conversation with representatives of the U.S. pharmaceutical company CoA Therapeutics, which is currently preparing a clinical trial for PKAN. Members of four patient associations were on site in person, with the rest attending both sessions via Zoom. For Hoffnungsbaum e.V. Chairman Markus Nielbock took part in the meetings virtually.
In addition to the NBIA Disorders Association and the Swiss Foundation for People with Rare Diseases, the three biotech sponsors Chiesi, CoA Therapeutics and Travere Therapeutics made the organization of this symposium possible with their financial support. Hoffnungssbaum e.V. had also provided 5,000 € as a further sponsor. Since these funds were not needed, they will be donated to the Research Fund of Hoffnungsbaum e.V. again.
Sources and further information:
December 2022 newsletter from the NBIA Disorders Association (also image source), pp. 2-4:
Fatemeh Mollet and NBIA Suisse website: