8th International Symposium on NBIA
The 8th International Symposium on NBIA attracted 160 participants from 26 countries. The record number of participants included world-renowned NBIA scientists and clinicians as well as those who only recently have become involved in NBIA research and care.
The symposium Scientific Steering Committee was led by Dr. Thomas Klopstock of Ludwig-Maximilians University Munich in Germany and included Drs. Susan Hayflick of Oregon Health & Science University in Portland, Valeria Tiranti of Foundation Neurological Institute C. Besta in Milan, Italy, and Agnès Rötig of Institute Imagine in Paris, France. Lay advocacy partners Fatemeh Mollet of NBIA Switzerland, Markus Nielbock of Hoffnungsbaum e. V. from Germany and Patricia Wood of NBIA Disorders Association in the United States were also committee members.
Hosted by NBIA Switzerland associated with Ludwig-Maximilians University Munich in Germany, the daily agenda included talks by long-standing, experienced NBIA researchers and their team members in which they provided research updates. In addition, researchers new to the NBIA community with expertise in cutting-edge therapies shared and contributed to the exchange of ideas.
Key Note Lecture
- Roland Lill, Marburg (Germany)
- Lena Burbulla, Munich (Germany)
- David Devos, Lille (France)
- Ivano Di Meo, Milan (Italy)
- Dario Finazzi, Brescia (Italy)
- Barbara Gnutti, Brescia (Italy)
- Paola Goffrini, Parma (Italy)
- Penelope Hogarth, Portland (USA)
- Arcangela Iuso, Munich (Germany)
- Suzanne Jackowski, Memphis (USA)
- Hana Kolarova, Prague (Czech Republic)
- Manju Kurian, London (UK)
- Sonia Levi, Milan (Italy)
- Mario Mauthe, Groningen (Netherlands)
- Tassula Proikas-Cezanne, Tübingen (Germany)
- Ahad Rahim, London (UK)
- Agnès Rötig, Paris (France)
- Hein Schepers, Groningen (Netherlands)
- Susanne A. Schneider, Munich (Germany)
- Sunita Venkateswaran, Ottawa (Canada)
- Zhihao Wu, Dallas (USA)
Specialized sessions each day focused on the more common NBIA disorders—PKAN, CoPAN, MPAN, BPAN, PLAN/INAD and FAHN—but the discussions of individual disorders had overarching interest for all NBIA Disorders.
Overall, five cross-disease topics crystallized as focal points for discussion:
- Deciphering the biochemical pathomechanisms of the diseases, thus obtaining fresh ideas for potential new therapies
- Exploring the potential of gene, small molecule, and cell therapies in treating NBIA disorders
- Generating appropriate animal and differentiated cell models for pre-clinical testing
- Performing natural history studies and development of appropriate subtype-specific rating scales as prerequisite for clinical trials
- Conducting clinical trials, upcoming or already underway, and requirements to implement them.
Everyone agreed that sufficient funding remains a major obstacle when considering the various scientific approaches that still need to be explored. Regardless of the therapeutic approach chosen for treatment, researchers shared the view that natural history studies cannot begin early enough in parallel to other research to have meaningful clinical endpoints available to achieve clinical trial readiness.
Patient representatives of the 10 NBIA Alliance members attended, along with seven organizations dedicated to a specific NBIA disorder. Biotech and pharmaceutical companies were also represented. They heard 37 scientific talks and six flash talks and could ask questions after each presentation. In addition, each day opened with a patient representative giving a welcome address that shared the patient perspective and acknowledged and encouraged the work of the NBIA scientific community.
Each day ended with a lively discussion open to all participants on urgent needs and next steps in the NBIA subtypes covered in the presentations. These roundtables attracted between 50 and 75 participants.
A big THANK YOU to our sponsors for this event!
- Chiesi Global Rare Diseases
- CoA Therapeutics
- Hoffnungsbaum e.V.
- Travere Therapeutics