Gathering in Brussels for a week, a diverse group of leading experts from across the world – ranging from research scientists, medical professionals, pharmaceutical and biotech companies, public and private funders, and patient representatives – exchanged views on the scientific and medical future of the family of genetic conditions neurofibromatosis and schwannomatosis (known collectively as NF). Affecting 4 million people worldwide of all populations equally, including approximately 375,000 Europeans, these conditions cause tumors to grow on nerves throughout the body.
The 2024 Global NF Conference, hosted by Children’s Tumor Foundation (CTF) Europe, was concluded on 25 June, connecting nearly 1,000 global experts with 160 presentations to share up-to-date knowledge and insights in the growing fields of NF research and care.
Conference Highlights:
- Advancements in Gene Therapy: Researchers presented groundbreaking work in gene therapy aimed at correcting the genetic mutations responsible for NF, offering hope for more effective treatments.
- Comprehensive Care Models: new models of comprehensive care were discussed, emphasizing the importance of holistic approaches to improve patient outcomes and quality of life.
- Novel Therapeutics: Several novel therapeutics were presented, including innovative drug candidates that show promise in targeting NF-related tumors more effectively and with fewer side effects.
- Artificial Intelligence in NF Research: AI and machine learning applications were showcased, highlighting their potential in accelerating drug discovery and improving diagnostic accuracy.
- Biomarker Discovery: Significant progress in biomarker discovery was reported, paving the way for better disease monitoring, methods that allow earlier detection of malignancy, and more precise treatment strategies
- Public-Private Partnerships: Success stories of public-private partnerships, such as the EU-PEARL project, demonstrated the power of collaboration in driving forward NF research and clinical trials.
Embarking on a New Era for NF Research and Care
With the goal of redefining the future of NF research and care, accelerating the path to effective therapies, and improving patient outcomes, the NF Conference underscored the timeliness of making NF research and innovation more visible. As experts from across the globe explored areas such as gene therapy, comprehensive care, novel therapeutics, artificial intelligence, and biomarker discovery, the importance of facilitating the translation of science into therapies became evident.
Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA), Nathalie Moll emphasized that EFPIA can play a key role in linking the research and patient communities to pharmaceutical companies by supporting Public Private Partnerships (PPP) like the Innovative Health Initiative (IHI). These initiatives bring together industry, EU funding, researchers, patients, regulators, and other relevant stakeholders to drive forward science and innovation. The Director General emphasized that NF is the perfect example for showing policy makers how important it is to take big plans down to what truly matters, therapies for patients.
The Executive Director of the Innovative Health Initiative (IHI), Niklas Blomberg, stressed that the IHI helps to “identify interesting, important, and challenging problems which are too difficult for individual actors or sectors to tackle alone” and by extension, strives for more collaboration in the broader healthcare ecosystem. The Executive Director of Science Policy and Regulatory Affairs at EFPIA, Magda Chlebus, emphasized that collaboration is not just about doing more of the same things; it is about doing things differently. This collaborative spirit is familiar to CTF, which harnessed the opportunity in 2019 with its participation in the strategic partnership between the public and private sectors forming the future of clinical trials through the EU-PEARL project.
Global experts discussed the success story of EU-PEARL for NF, especially as CTF and the Global Coalition for Adaptive Research are currently working to set up a platform trial for NF1 and schwannomatosis (NF2-SWN), based on the clinical platform trial template established by EU-PEARL. This illustrates that pioneering ideas can accelerate the development of effective treatments for patients, particularly when public-private collaborations materialize and targeted investments follow.
Annette Bakker, Chief Executive Officer of the Children’s Tumor Foundation and Board Chair of CTF Europe, said, “This collective effort is pivotal in accelerating breakthroughs for NF and other rare diseases. By fostering collaboration among diverse stakeholders, we can turn scientific discoveries into tangible therapies that significantly improve patients’ lives. The pilots spearheaded by CTF – accelerating R&D by utilizing preclinical hubs and platform trials, and repositioning shelved assets back into commercial application for NF2-SWN – have worked. We are ready to scale but we need more believers – drug companies and investors – who see what we are building for rare diseases, and to join us in the construction of unique public private partnerships that will lead to life-changing advancements for those affected by rare diseases, including NF. This is not just about science; it’s about building a better future for the millions of patients and their families.”
Ultimately, foundations such as CTF Europe act as connectors; bringing together industry, patients, scientists, regulators, and policy makers in such large-scale gatherings. It is through the right collaborations established within PPP’s that NF’s scientific potential can be fully realized. Echoing EFPIA’s Director General Nathalie Moll’s words, “NF is the perfect example that should be leveraged in the policy discussions at the European level; to remind policy makers that health matters”. And it does matter. Let’s not waste the opportunity to invest in the right research – the research that matters.
