The mission of our Curing NF team is to accelerate a cure for neurofibromatosis type one (NF1), and the path to finding cures for this complex disease is paved through scientific collaboration, innovation, and shared purpose. This spring, our Curing NF team hosted two groundbreaking meetings that exemplify this collaborative approach: our second annual Brain Tumor Initiative (BTI) and our inaugural Next Generation Models Initiative (NGMI). These convenings brought together brilliant minds from across the globe, all united in accelerating treatments and ultimately finding a cure for NF1.

 

 

Brain Tumor Initiative: Building on Momentum

The second annual BTI meeting convened nearly 50 leading clinicians and scientists in Detroit this March, focusing specifically on NF1-associated high-grade gliomas. Over two intensive days, participants shared insights through 20 presentations organized into three critical areas: clinical studies, preclinical modeling, and therapeutic development.

Clinical sessions explored how molecular and clinical features impact patient survival and highlighted upcoming clinical trials for NF1-associated high-grade gliomas. The preclinical sessions demonstrated significant progress in developing robust models that accurately replicate the disease, with discussions about conducting parallel preclinical trials alongside clinical studies to accelerate therapeutic development. The tools and therapeutics sessions showcased innovative treatment strategies, including CAR T cell therapy and bispecific antibodies, as well as advanced computational tools to expedite discovery.

Dr. Poornima Venkat, Scientific Program Manager for BTI, said, “The exciting advances in clinical trials, along with emerging preclinical models and the development of targeted therapies, offer hope for improved outcomes in patients with NF1-associated high-grade gliomas.”

 

Next Generation Models Initiative: A Landmark First Meeting

The inaugural NGMI meeting showcased true scientific collaboration at its finest. Beginning with keynote presentations from organoid research pioneers Juergen Knoblich and Sergiu Pasca, the meeting successfully united leaders in NF research with experts in advanced modeling techniques.

We were proud that participants demonstrated remarkable openness to adopting the best available models regardless of origin. The presentations showcased impressive technological advances, including microfluidic devices, immune-competent brain organoids, and AI-based image analysis. With 18 projects representing various NF1 manifestations, the diversity of next-generation preclinical systems was remarkable.

During the meeting, we also introduced NF Research Tools Central, a resource on NF1 tools and reagents created through collaboration between the Gilbert Family Foundation and Sage Bionetworks. A special townhall discussion led by Dr. Ivan Baines, Chief Operating Officer of the forthcoming Nick Gilbert Neurofibromatosis Research Institute (NGNRI), provided updates on this groundbreaking facility and specifically sought input on which models the scientific community would like to see hosted at the institute. As the first-ever brick-and-mortar facility dedicated exclusively to neurofibromatosis research, NGNRI represents a transformative development that will centralize resources, accelerate research timelines, and provide unprecedented infrastructure for developing treatments and cures. Perhaps most encouraging was what Dr. Kalyan Vinnakota, Gilbert Family Foundation’s Director of Curing NF described as “unanimous enthusiasm in the community to share their tools and resources, and to work together to identify the best model systems for specific NF1 manifestations.”

 

The Power of Collaboration

These annual meetings represent so much more than scientific conferences—they are catalysts for accelerating progress. By fostering environments where researchers can share findings, discuss challenges, and form new collaborative relationships, we’re creating the conditions necessary for breakthrough discoveries.

The Gilbert Family Foundation – and our Curing NF team – remains committed to this collaborative approach. We believe that by bringing together diverse expertise and encouraging open exchange of ideas and resources, we’re moving the needle towards our ultimate goal: finding effective treatments and eventually a cure for neurofibromatosis type 1.

As we look toward future meetings, we’re inspired by the dedication and scientific ingenuity displayed by all participants. Together, we’re not just advancing science; we’re advancing hope for all those affected by NF1.