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Our Mission Our Progress

We are the Drug Discovery Engine for NF

The Children’s Tumor Foundation (CTF) is the drug discovery engine for NF. By bringing together patients, doctors, scientists, and pharma, we drive treatments, advance care, and deliver faster results for millions affected by neurofibromatosis or schwannomatosis. Our patient-first collaborative approach accelerates drug development and brings life-changing therapies to patients faster – driven by our mission to end NF.

The Impact of Your Donations

A gloved hand uses a pipette to add liquid to a multiwell plate in a laboratory setting.

First FDA-Approved Treatment for NF1: Koselugo

CTF’s research investment proved that MEK inhibitors shrink NF tumors. We spearheaded the first-ever FDA session on NF, building critical support for FDA approval.

Second NF1 Treatment Awaiting Approval: Mirdametinib

CTF helped identify and reposition this drug for NF, leading to its transfer to SpringWorks, a company spun off from Pfizer.

Phase 3 Topical Treatment for cNF: NFX-179

CTF’s early impact investment could soon lead to a gel for cutaneous neurofibroma (cNF) skin tumors.

Two scientists in lab coats and masks work near a microscope in a laboratory setting.

Breakthrough for NF2-SWN: Brigatinib

CTF’s team uncovered and fast-tracked this potential treatment in the first NF platform clinical trial, which is showing great promise.

First AI-Driven NF Drug: HLX-1502

CTF’s investment accelerates the development of Healx’s drug, which has been granted Fast Track designation by the FDA.

A dropper adds red liquid to one of several clear test tubes.

First NF1 Biomarker Study

CTF-funded blood test shows promising potential to detect early signs of NF1 tumors that could become malignant.

Pushing Boundaries, Sustaining Momentum, and Fostering New Possibilities

Triple the Number of Clinical Trials

CTF’s leadership and research funding have expanded the NF field, boosting clinical trials from 21 a decade ago to 68 today, unlocking new possibilities for treatment and care.

20+ Companies in the NF Space

CTF catalyzed unprecedented interest in NF, transforming a once-overlooked field into a hub that now attracts over 20 pharmaceutical and biotech companies.

1,000+ Experts at the NF Conference

Organized and led by CTF, this ‘must-attend’ global gathering for NF research drives innovation and collaboration among scientists, clinicians, and industry leaders.

NF Clinic Network: 20,000 Patients Annually

CTF established the NF Clinic Network to ensure access to high-quality NF care, now spanning over 70 locations across North America.

Global Patient Registry with 11,000 Patients

CTF established the NF Registry, a vital resource accelerating clinical trial recruitment and driving breakthroughs in NF research.

Impact of CTF Funding

Search the Children’s Tumor Foundation’s Award Database to discover investigators and projects driving key advances in NF research.

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years of funding NF research

200

Young Investigators were drawn to the NF field via the YIA

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NF Research Projects Funded

Explore CTF Funded Research Projects

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