Authors of the English original article: Patricia Wood and Angelika Klucken
160 participants from 26 countries attended the 7th International Symposium on NBIA and Related Diseases, which was held as a virtual event for the first time. With online access making it easier to meet in virtual space during the COVID-19 pandemic, this record number of participants was made possible, including the world's most renowned NBIA scientists and clinicians, as well as those professionals who have only recently become involved in NBIA research and care.
The Scientific Steering Committee of the symposium was chaired by Prof. Thomas Klopstock, head of the NBIA research team at the Friedrich Baur Institute of the Neurological University Hospital Munich (LMU). The preparatory committee also included Prof. Susan Hayflick from Oregon Health & Science University in Portland, USA, Prof. Valeria Tiranti from the C. Besta Neurological Institute in Milan, Italy, and Dr. Agnès Rötig from the Imagine Institute in Paris, France. The patient representatives Fatemeh Mollet from NBIA Suisse, Angelika Klucken from Hoffnungsbaum e. V. and Patricia Wood from the NBIA Disorders Association in the USA were also committee members.
The picture shows the virtual lobby of the symposium. Participants were able to click on the meetings or offers they wanted to attend.
The program of the conference, which was hosted by the NBIA Disorders Association in the USA, included presentations in which long-standing, experienced NBIA researchers and their colleagues reported on current research findings. In addition, researchers who are new to the NBIA community but have special expertise in the latest treatment methods shared their knowledge and contributed to the exchange of ideas.
The meeting program each day focused on one or two of the more common NBIA diseases in particular - PKAN, CoPAN, BPAN, MPAN, PLAN and FAHN, but the discussions on individual diseases were of overarching interest to all NBIA forms. Overall, five cross-disease topics emerged as focal points for discussion:
- Deciphering the pathological biochemical mechanisms of diseases and thus gaining new ideas for possible new therapies
- Exploring the potential of gene, small molecule drug and cell therapies in the treatment of NBIA diseases
- Generation of suitable animal models and differentiated cell models for preclinical tests
- Conducting studies on the natural course of disease and developing suitable subtype-specific assessment scales as a prerequisite for clinical studies
- Implementation of upcoming or ongoing clinical trials and requirements for their implementation.
All agreed that sufficient funding remains a major obstacle when it comes to the various scientific approaches that still need to be explored. Regardless of the therapeutic approach chosen for a treatment, the researchers shared the view that, in parallel with other research, natural history studies cannot start soon enough to have meaningful clinical endpoints at hand to be well prepared for clinical trials, i.e. to achieve trial readiness.
Patient representatives from 10 NBIA Alliance member organizations attended the conference, as well as representatives from the seven organizations dedicated to a specific NBIA disease. Biotech and pharmaceutical companies were also represented.
Participants listened to 37 scientific presentations and six lightning talks and were able to ask the speakers questions after each presentation. In addition, each day opened with a welcome address from a patient representative who acknowledged and encouraged the commitment of the NBIA scientific community from a patient perspective.
Each day ended with a lively discussion, open to all participants, on urgent needs and next steps for the NBIA diseases covered in the presentations. Between 50 and 75 people participated in these discussions. Additional networking opportunities and virtual exhibit booths were also offered during the symposium.
Susan Hayflick, medical geneticist and director of NBIA research in Portland, Oregon (USA), suggested that researchers should meet virtually more often in addition to in-person symposia to improve scientific exchange and accelerate NBIA research. Many agreed and rated the quality of the virtual platform used at the symposium as very good.
Most of the presentations were recorded and are now available at the following link:
Summaries of the daily presentations in layman's terms are being prepared and will soon be made available by the non-profit NBIA patient organizations. In 2021, the 8th International Symposium on NBIA and Related Disorders will be held as an in-person meeting in Lausanne, Switzerland from October 14-16, 2021.
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