NBIA International Scientific Conference in Poland

On October 19 and 20, 2023, a BPAN workshop, an NBIA Alliance meeting and an international NBIA scientific conference were held in Warsaw, Poland. Researchers, physicians and patient representatives from many countries came together to discuss research projects and results. In addition, possible new research approaches were discussed and collaborations were explored. You can read the agenda of the conference here: https://nbia-polska.org/data/files/agenda.pdf

 Participants of the conference, Image: NBIA Poland

From Hoffnungsbaum e.V. Markus Nielbock and Noah Rusch for professional reasons, unfortunately only part of the online experience. Patricia Wood of the NBIA Disorders Association (NBIA DA) was present the entire time and reported on the weekend in the NBIA DA's December newsletter:

As head of the research program at NBIA DA, I have had the privilege of participating in valuable events: a BPAN workshop, the first conference of NBIA Poland, and a meeting of the NBIA Alliance. Attending these events has proven to be very rewarding, as I have been able to witness the passion and dedication of our dedicated researchers, clinicians, and patient advocacy leaders.

The aim of the BPAN workshop was to identify and discuss possibilities for the development of therapies for beta-propeller protein-associated neurodegeneration (BPAN). The workshop was organised by our sister organisation, Stichting Ijzersterk, in collaboration with three European BPAN researchers: Rachel Wise from Ludwig-Maximilians-Universität in Germany, Mario Mauthe from University Medical Center Groningen in the Netherlands and Apostolos Papandreou from University College London in the UK.
This landmark initiative was funded by the European Joint Program on Rare Diseases (EJP-RD) Networking Support Scheme and attracted thirty participants from ten countries. Unlike traditional scientific meetings, this event focused on interactive discussions between all participants to identify the main barriers to basic, translational and clinical research on BPAN and to promote networking and sharing of resources among scientists. Discussions focused on the inclusion of BPAN patient data in the TIRCON registry, the search for a BPAN biomarker that is essential for future clinical trials, strategies to expand the BPAN research network, intensify international cooperation and promote the careers of young scientists.
Joost Schimmel, board member of Stichting Ijzersterk, noted: "The scientists will draw up a list of available resources to study BPAN, which will accelerate our understanding of the disease, and I think that this networking event could be a good starting point for a joint European funding application for research into BPAN."
An official report on the workshop and a study on BPAN landscape analysis is currently being finalised by the consultancy Science Compass. Once it is available, it will be shared with the BPAN community by the patient organisations that funded the analysis. This study will serve as a basis for future BPAN grant initiatives.

The BPAN workshop ended at 2:00 p.m. on Wednesday, and at 4:30 p.m., representatives from CoA Therapeutics met with six members of the NBIA Alliance (some of the participants were connected via Zoom) to get an update on their upcoming clinical trial, which is scheduled to begin in 2024.

This was followed by a meeting of the NBIA Alliance, where each organization presented their ongoing projects and paved the way for discussions on potential collaborations in the field of research funding aligned with our shared goals. A key objective on our agenda was to determine the total number of NBIA families served by the members of the alliance without any overlap, while maintaining family privacy. Work is also underway to update the NBIA Alliance website.
These face-to-face meetings serve as an important platform for Alliance members to strengthen connections and reaffirm our commitment to a global NBIA community that shares common goals.

On Thursday and Friday, the scientific conference "Scientists for the NBIA Community" hosted by NBIA Poland, which included comprehensive scientific presentations on various NBIA diseases. The aim of the conference was to bring together patient families, physicians and researchers to learn about the need for therapies and to build international collaboration between research centers, associations and the NBIA community.
About 150 participants were present, including families from Poland. Thanks to the simultaneous translation of 22 research presentations into English or Polish, all those present were able to understand all the information. Some researchers presented work that had not yet been published, and the presentations led to lively discussions with the audience, who asked many questions about the scientific information. There was also plenty of time for researchers and clinicians to socialize, discuss possible future collaborations, and share their work more informally.

Following the conclusion of the meeting on Friday evening, researchers and representatives of patient organisations came together to discuss further topics of interest and to reach consensus on future steps. Everyone was in favor of Dr. Yapici Zuhal from the University of Istanbul in Turkey to host the 9th Scientific Symposium on NBIA Disorders next year. Beyond the scientific discourse, NBIA Poland was a friendly and welcoming host, organizing a tour of the Royal Castle in Warsaw's Old Town on Saturday, followed by a traditional Polish lunch.
It was an important week for so many members of the NBIA community who came together from all over the world and shared their work, ideas and friendships. 

Translation of an article by Patricia Wood from the NBIA DA's December newsletter:
https://www.nbiadisorders.org/images/newsletters/NBIA_Newsletter_December_2023.pdf

The Polish patient organisation NBIA Poland thanked the participants with an open letter. The following is a German translation of the letter:

Dear guests, speakers and members of the entire NBIA community in Poland and around the world,

Following the conference "Scientists for the NBIA Community, Warsaw 2023", we would like to thank you very much for your participation, your presentations and your scientific research contributions. It is a great honour for us that you have placed your trust in us and honoured us with your presence. The scientific advances in the field of NBIA presented at the conference were impressive. We have received a lot of hope for effective treatment and an increase in knowledge to support patients. The entire NBIA community has seen wonderful, dedicated scientists, clinicians, physicians, and involved professionals working on a wide range of projects. Their hard work is both impressive and worthy of recognition. Despite many difficulties, despite so many activities, you have managed to come and stay with us. Thank you for your smile and dedication to explaining disease mechanisms, as well as for your complex research. Many of the presented results have already been presented before publication, most of them have not yet been patented or have just been achieved in your laboratories. It's very touching. It is impossible to describe the gratitude we have for you, the researchers and the entire clinical and scientific teams – it is the gratitude of the entire NBIA community in Poland and around the world. We are very proud that people like you have chosen to fight NBIA diseases, because this is the most valuable thing we could have dreamed of. They have given us hope for the progress of medicine, which is so important for patients and their relatives.

[...]
We hope you spent valuable time at our conference. We feel that the entire NBIA community has experienced a pleasant atmosphere, as well as a warm and welcoming atmosphere. Mutual support and cooperation with the goal of overcoming NBIA gives us strength that no one can take away from us. Everything we do is for our loved ones. We will try to implement and support all the findings and research proposals with the NBIA sister organizations from around the world that participated in the conference. We hope you will think of us. We invite you to Poland again, you are always welcome.
Together we can achieve more!

Board of Directors of NBIA Poland

Abridged translation of the open letter from NBIA Poland:
https://nbia-polska.org/i-konferencja