International scientific NBIA conference in Poland

On October 19 and 20, 2023, a BPAN workshop, an NBIA Alliance meeting and an international scientific NBIA conference took place in Warsaw, Poland. Researchers, doctors and patient representatives from many countries came together to exchange information about research projects and results. Possible new research approaches were also discussed and collaborations explored. You can read the conference agenda here: https://nbia-polska.org/data/files/agenda.pdf

 Participants of the conference, picture: NBIA Poland

Unfortunately, Markus Nielbock and Noah Rusch from Hoffnungsbaum e.V. were only able to attend part of the event online for professional reasons. Patricia Wood from the NBIA Disorders Association (NBIA DA) was present the whole time and reported on the weekend in the NBIA DA's December newsletter:

As NBIA DA's Research Program Manager, I have had the privilege of attending valuable events: a BPAN workshop, NBIA Poland's first conference, and an NBIA Alliance meeting. Attending these events has been very rewarding as I have witnessed the passion and commitment of our dedicated researchers, clinicians and patient organization 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 organized by our sister organization, 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 pioneering 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 major barriers to basic, translational and clinical research on BPAN and to promote networking and resource sharing among scientists. Discussions covered 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, increasing international collaboration and promoting the careers of young scientists.
Joost Schimmel, board member of Stichting Ijzersterk, commented: "The scientists will draw up a list of available resources for research into BPAN, which will accelerate our understanding of the disease, and I think 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 the BPAN landscape analysis is currently being finalized by the consulting firm Science Compass. Once available, it will be shared with the BPAN community by the patient organizations that funded the analysis. This study will serve as the basis for future BPAN grant initiatives.

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

This was followed by an NBIA Alliance meeting where each organization presented their ongoing projects and paved the way for discussions on potential research funding collaborations aligned with our common goals. A major goal on our agenda was to determine the total number of NBIA families served by Alliance members without 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 ties and reaffirm our commitment to a global NBIA community that strives for common goals.

On Thursday and Friday, NBIA Poland hosted the scientific conference "Scientists for the NBIA Community", which included comprehensive scientific presentations on various NBIA diseases. The aim of the conference was to bring together patient families, doctors and researchers to learn about the need for therapies and to establish international cooperation between research centers, associations and the NBIA community.
There were around 150 participants, including families from Poland. Thanks to the simultaneous translation of 22 research presentations into English or Polish, everyone present was able to understand all the information. Some researchers presented unpublished work 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.

At the end of the meeting on Friday evening, researchers and representatives of patient organizations came together to discuss other interesting topics and reach a consensus on future steps. All were in favor of Dr. Yapici Zuhal from Istanbul University in Turkey to host the 9th Scientific Symposium on NBIA Diseases 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 from around the world to come together and share their work, ideas and friendships. 

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

The Polish patient organization NBIA Poland warmly thanked the participants with an open letter. A German translation of the letter follows:

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 honor for us that you have placed your trust in us and honored us with your presence. The scientific advances in the field of NBIA presented at the conference were impressive. We received a lot of hope for effective treatment and an expansion of knowledge to help 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. We thank you for your smile and commitment to explaining disease mechanisms and for your complex research. Many of the results presented were presented before publication, mostly not yet patented or just newly achieved in your labs. This is 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 decided to fight NBIA diseases, because this is the most valuable thing we could have dreamed of. You have given us hope for the progress of medicine, which is so important for patients and their families.

[...]
We hope that you spent valuable time at our conference. We believe that the entire NBIA community experienced a pleasant atmosphere as well as a warm and cordial atmosphere. Supporting each other and working together to overcome NBIA gives us strength that no one can take away. 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 who attended the conference. We hope that 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