A pivotal chapter in the story of drug discovery for NF2-related schwannomatosis (formerly called neurofibromatosis type 2) has been written – both figuratively and literally – with a new publication highlighting the critical role of collaboration in the journey of Brigatinib, a drug now offering hope to patients with this rare condition. The chapter, co-authored by a multidisciplinary team of researchers including Dr. Annette Bakker, CEO of the Children’s Tumor Foundation, underscores the groundbreaking work that made Brigatinib a reality for NF2-SWN.
The Synodos for NF2 Legacy
Brigatinib’s story begins with Synodos for NF2, an unprecedented collaborative research initiative that united scientists, clinicians, and industry partners under a common goal: accelerating treatments for NF2-related schwannomatosis. At the heart of Synodos was an ethos of sharing data, resources, and expertise to overcome the barriers that have long slowed progress in rare disease research.
Through cutting-edge tools and technologies, the team screened a wide array of potential drug candidates. Brigatinib—a drug originally developed to treat lung cancer – emerged as an unexpected but powerful contender. This discovery represented a paradigm shift, demonstrating the potential of repositioning existing drugs for rare conditions like NF2-SWN.
Revolutionizing Clinical Trials
The innovation didn’t stop with drug discovery. To test Brigatinib’s effectiveness for NF2-SWN patients, a pioneering platform trial model was used – a groundbreaking approach that allows multiple therapies to be tested simultaneously. This design significantly reduces the time and costs traditionally associated with clinical trials, enabling faster access to potential treatments for patients.
The results, published in the New England Journal of Medicine, showed promising outcomes, bringing hope to individuals living with NF2-related schwannomatosis and validating the collaborative research model that made it possible.
Collaboration as the Catalyst
The recently published chapter on Brigatinib is more than a recounting of scientific achievements; it is a testament to the power of collaboration in tackling complex challenges. It reflects how a united effort can achieve breakthroughs that might otherwise remain out of reach, especially for rare diseases like NF2-SWN.
Brigatinib’s journey—from a lung cancer therapy to a promising treatment for NF2-SWN – is proof that with the right tools, partnerships, and vision, even the most daunting medical puzzles can be solved. This is why the Children’s Tumor Foundation continues to champion innovative research models and collaborations that turn possibility into reality for the NF community.
What’s Next?
As Brigatinib advances through trials, it serves as a beacon of hope for the NF2-SWN community and a blueprint for accelerating treatments for other conditions. The Children’s Tumor Foundation remains committed to fostering bold, collaborative efforts that create more opportunities to change lives.
This milestone is not just about one drug; it’s about transforming the way we tackle rare diseases – together.
