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Collaborations and Partnerships
At Hope for Hermione, we understand the importance of collaboration in the field of BPAN research. We work closely with other organizations, researchers, and alliances to pool resources and knowledge towards a common goal. Learn more about our collaborations.
In early October 2024, the NBIA Disorders Association convened the first-ever BPAN Research Roundtable, uniting 13 non-profit organizations worldwide dedicated to funding and advancing research into Beta-propeller Protein-Associated Neurodegeneration (BPAN). Since its identification in 2012, BPAN has become the most prevalent NBIA disorder, catalysing a surge of research and advocacy efforts.
The roundtable aimed to synchronize the efforts of participating organizations, ensuring they were informed about ongoing research to avoid duplication and identify areas needing further focus. Most importantly, it reinforced a shared commitment to a common goal: developing effective treatments and ultimately curing BPAN. Over the past decade, the research community has made significant strides in understanding BPAN, and participants proudly highlighted progress in key areas.
One critical focus was the status of current patient registries and natural history studies. These efforts, essential for tracking BPAN’s progression, provide valuable insights into the disease’s impact and potential treatment pathways.
Discussions also delved into major research areas, including autophagy, mitochondrial dysfunction, ferroptosis, and biomarker identification. These cellular mechanisms are central to developing therapies to alleviate symptoms and evaluate their effectiveness. Participants emphasized the need to advance small molecule research and gene therapy initiatives, both of which target BPAN’s genetic causes and offer hope for precision treatments.
Representatives from 13 organisations participated in the roundtable: Action for BPAN (UK), AISNAF (Italy), BPAN France (France), BPAN Spain (Spain), Don't Forget Morgan Foundation (USA), Duets Across America (USA), Hoffnungsbaum e.V. (Germany), Hope for Hermione (UK), Isabel's Chance (USA), NBIA Disorders Association (USA), NBIA Poland (Poland), NBIA Suisse (Switzerland), and Stichting IJzersterk (Netherlands). Many of these groups are part of the international NBIA Alliance, supporting research into BPAN and other NBIA disorders.
Another focus was repurposing FDA-approved compounds for BPAN treatment. By screening these drugs in animal models, researchers aim to identify medications that could be used off-label to improve symptoms.
The roundtable also highlighted progress on medical consensus guidelines, the establishment of NBIA Centres of Excellence, and BPAN-specific clinics. These initiatives aim to ensure families receive specialized, high-quality care while supporting advancements in BPAN research.
Collaboration emerged as the cornerstone of success. As Christian Magnet of BPAN France put it: “Act together to do better faster.” By sharing information, aligning efforts, and maximizing resources, these organizations are accelerating progress.
Looking ahead, the group committed to maintaining momentum with biannual meetings to ensure research remains focused on the most promising areas.
Christina Ftikas of the Don’t Forget Morgan Foundation echoed this sentiment: “Collaboration and research efforts across BPAN foundations will help the community get to the finish line faster and hopefully lead to the development of treatments for our beautiful children.”
The roundtable underscored the strength, unity, and determination of the BPAN community. Together, these efforts bring us closer to a future where effective treatments and a cure for BPAN become a reality.