The Children’s Tumor Foundation (CTF) has released its latest impact report, detailing significant progress in the fight to end NF. The report provides a comprehensive overview of the foundation’s strategic initiatives and research impact through 2024.
“We stand at a critical moment in our fight against NF,” notes CTF CEO Annette Bakker, PhD. “With two drugs already approved and multiple treatments on the horizon, the momentum is unprecedented. This report demonstrates how far we’ve come and outlines our strategic roadmap for the future.”
The full impact report is available here, providing detailed insights into the foundation’s strategic plans for accelerating NF research and improving patient care.
Treatment Landscape: Breakthrough Approvals and Pipeline
A major highlight of the report is the recent FDA approval of Gomekli (mirdametinib), marking the second approved treatment for NF1. This follows the 2020 approval of Koselugo, demonstrating significant momentum in the NF treatment landscape.
Several promising treatments are also in advanced stages of development:
- NFX-179 topical therapy for cutaneous neurofibromas has progressed to Phase 3 trials
- Brigatinib has shown encouraging results in the INTUITT-NF2 platform trial for NF2-related schwannomatosis
- A groundbreaking biomarker blood test for early detection of malignant peripheral nerve sheath tumors (MPNSTs) is under development
Accelerating Discovery: Strategic Research Investments
The report details CTF’s commitment to expanding the research field with significant investments in:
- Optic pathway glioma studies
- NF2-SWN gene therapy research
- Schwannomatosis research at the Sylvester Comprehensive Cancer Center
- 16 new research awards granted in 2024, supporting young investigators and innovative approaches
Accelerating Treatments: Infrastructure and Partnerships
CTF continues to drive therapy development through strategic initiatives:
- The CTF Preclinical Hub, which provides access to models, expertise, and resources to expedite drug discovery
- Platform and basket trials that efficiently test multiple treatments
- Strategic commercial investments in promising therapies
- Partnerships with innovative companies like CureAge Therapeutics and Healx, which is leveraging AI for drug discovery
Foundations of Progress: Community and Care
The foundation’s work extends beyond research to create a comprehensive ecosystem:
- The 2024 Global NF Conference in Brussels brought together 849 participants from 43 countries
- The NF Registry has grown to over 11,000 participants, accelerating research and clinical trial recruitment
- Implementation of the NF Clinic Network Designation System to improve the quality of care
- Enhanced patient engagement initiatives through CTF Engage
Click the image below to read this issue online, or CLICK HERE to download it as a PDF.
