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CTF Progress in NF1

Approved Treatments

In the spring of 2020, the U.S. Food and Drug Administration (FDA) announced the approval of Koselugo (selumetinib) for use in patients with inoperable plexiform neurofibromas, a common manifestation in neurofibromatosis type one (NF1). 

Koselugo is the first and only approved treatment for NF and has paved the way for the development of more treatment options for all types of NF.

The FDA’s approval of AstraZeneca’s and MSD (Merck)’s submission was a major milestone for patients living with NF. We are at this game-changing moment because of the support of CTF donors, who funded the pioneering research that led to FDA approval.

One step closer fda approves first drug for neurofibromatosis.

The Road to The First Approved Drug for NF


CTF’s NF Preclinical Initiative (NFPI) demonstrated that MEK inhibitors have a massive result on plexiform neurofibroma tumor volume. The NFPI was a $7 million, 5-year CTF team-science initiative that concluded in 2013.

Brigitte Widemann, MD, reported at CTF's 2015 NF Conference that clinical trial data showed meaningful decreases in tumor volume. In 2020, CTF shared the FDA announcement of the approval of selumetinib, now marketed as Koselugo.

Now, Koselugo has been approved for the treatment of NF1 patients in 32 countries, and is now in clinical trials for other tumor types that occur in NF2-related schwannomatosis and other types of schwannomatosis.

CTF Grant Funding

The Children’s Tumor Foundation is dedicated to funding research to prevent, reverse, or manage complications of all types of NF. CTF has played an important role in providing funding to early-career NF researchers and to early-stage NF research.

Our Discovery Fund supports individual researchers and clinicians engaged in NF investigations. The Discovery Fund encompasses the Young Investigators Award (YIA), Drug Discovery Initiative (DDI), and Clinical Research Awards (CRA), as well as Contract Awards and Gene Therapy awards.

Impact of CTF Funding


years of funding NF research


Young Investigators were drawn to the NF field via the YIA


NF Research Projects Funded

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


Latest NF1 Research Awards

NF1 Gene Therapy Initiative

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NF1 YIA & DDI Research Awards

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Clinical Research Award

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Clinical Trials

Clinical trials help identify potential new treatments that will benefit patients living with NF. The number of clinical trials for NF has increased to three times the number of trials from just a decade ago. Many of these potential treatments are directly due to research sponsored by the Children's Tumor Foundation.

Click here to search for NF1
Two people in lab coats working on a microscope.

Tips for your Clinical Trials search

“Targets” are the part of the disease process that the drug tries to stop.

  • “Agents” are the drug names.
  • “Phases” are the current stage of research. The higher the number of the Phase, the closer the treatment is to being approved for use in NF patients.
    • Phase 0 is very early stage testing
    • Phase 1 tests for drug safety
    • Phase 2 tests whether the treatment works.
    • Phase 3 tests whether the new treatment is better than existing treatments

You can also find more information at by clicking on “Clinical Trials” on the menu bar, or reading CTF’s resource for patients, What is a Clinical Trial?

For clinical trials in Europe, search

Platform Basket Trials

CTF is a funding partner with EU-PEARL (EU Patient-cEntric clinicAl tRial pLatforms), a joint project under a European Innovative Medicine Initiative (IMI). This pursuit is building platform trials in which multiple drugs are tested in parallel under the same clinical protocol. This approach allows more efficient identification of potential treatments.

Through this initiative, an NF1 platform trial protocol is being defined and finalized and will be operationalized through CTF and the Global Coalition for Adaptive Research. It will initially focus on treatments for plexiform neurofibromas, cutaneous neurofibromas, optic pathway gliomas (OPG), and non-OPG low-grade gliomas.

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Read more about CTF platform basket trials

Thanks to CTF’s advocacy efforts in Europe, NF was chosen as a prototype for rare diseases by the IMI. The NF component of EU-PEARL is co-lead by CTF and the Erasmus Medical Center in Rotterdam, Netherlands. While a network of European clinics will be the first of many to execute these trials, the established protocols will enable trials such as this to run globally.

In addition to the NF1 protocol, a schwannomatosis platform trial protocol is being developed that will enroll schwannomatosis patients, including NF2-related schwannomatosis patients, and will test the ability of drugs to shrink tumors.

Separately, CTF is funding the INTUITT-NF2 trial, which is currently in progress and testing brigatinib and neratinib in patients with NF2-SWN. This initiative is a result of the landmark work of CTF’s visionary Synodos for NF2 research collaboration. Read more about these trials on the Progress in NF2-SWN page.

NFX-179 Impact Investment

The Children’s Tumor Foundation entered a new phase in its strategic NF research model by announcing a significant impact investment in a Phase 2b clinical trial at NFlection Therapeutics, a biotechnology company focused on the discovery and development of effective, targeted therapies for rare diseases. The trial involves NFX-179, a topical (on the skin) treatment which has successfully passed a Phase 1/2a (safety and first signs of efficacy) trial. The NFX-179 treatment is for cutaneous neurofibromas (cNF), which are tumors that grow in the skin or right underneath the skin, and result in severe, disfiguring bumps on the skin. CTF’s investment strategy in NF drug development is a diversified portfolio approach to tackling all forms of NF, from early-stage investments in young investigators, open data collaboratives such as Synodos. This first-inits-history impact investment further establishes CTF as a co-investor with pharma/biotech in innovative NF research, expanding opportunities to increase the NF drug development pipeline.

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CTF Team Science and Data Sharing



Experts collaborating in three NF1 research consortia


Team science Initiatives (leading to selumetinib for NF1 and brigatinib for NF2-SWN)


Pharma/biotech companies drawn toward NF (10 years ago there were none)

The CTF research model values collaboration in every aspect of the drug discovery process. Diverse teams of experts managed by the Foundation collaborate and share their discoveries and data. By working together instead of in silos, we greatly increase the efficiency of research into problems that are too complex to be solved by individual scientists.

Click the titles of the past and present CTF team-science initiatives below to learn more about CTF progress in open data sharing.

Year of Open Science

The Children’s Tumor Foundation’s latest partnership is out of this world! CTF is euphoric to be part of the #YearofOpenScience, a collaboration spearheaded by the Center for Open Science, with support from NASA. Together with 15 additional organizations, these groups will convene for a series of working sessions to align collective action, culminating with a Year of Open Science conference in 2024 to showcase outputs, coalition-building, and ongoing work from these joint efforts.

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The NF Data Portal

Through our partnership with Sage Bionetworks, data is available and ready to use in the NF Data Portal. This centralized data repository is managed by specialists who collect, analyze, and release integrated data, accelerating the understanding of all types of neurofibromatosis and schwannomatosis, and the identification of “druggable” targets.

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Biomarker Project for NF1

In 2022, the Children’s Tumor Foundation announced a groundbreaking three-year study, which it will fund for nearly $2 million, to determine if a DNA-based blood test can offer better understanding and ultimately earlier diagnosis of malignant peripheral nerve sheath tumor (MPNST) predisposition in NF1 patients. CTF has committed its support to fund the study in its entirety. The project format is inspired by CTF’s highly effective Synodos model, which brings top researchers together to solve complex problems in an open and collaborative environment.

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Optic Pathway Glioma Multicenter Study

The Children’s Tumor Foundation and the Children’s Hospital of Philadelphia (CHOP) have renewed their five-year study of optic pathway glioma (OPG) in children with NF1. The study involves 25 NF clinics, and aims to provide clinicians with clear criteria that will help them decide when a patient should be treated, and when treatment (such as chemotherapy) should be avoided. OPG develops in 15–20% of children with NF1, and can cause significant health issues. In 2022, the study was renewed, and renamed to memorialize late CTF ambassador Jeffrey Owen Hanson, who suffered from OPG.

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Synodos for NF1

CTF sought out the concerns of NF1 patients when establishing Synodos for NF1, a team-science initiative that began its work in 2016 and concluded in 2020. Patient concerns for better treatment options for NF1, as well as answers for low-grade glioma patients, prompted CTF to initiate three separate consortia, bringing together 24 investigators from eight leading institutions and two companies.

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NF Preclinical Initiative

The NF Preclinical Initiative (NFPI) began in 2008 as the NF Preclinical Consortium (NFPC), a five-year, $7 million program that concluded in 2013. It expanded as the NF Therapeutic Consortium (NFTC), which continued the work of the NFPC, building on its infrastructure and discoveries.

Traditionally, it takes more than 15 years and costs hundreds of millions of dollars to translate a new discovery into one clinical treatment. The impact of the NFPI was clear: these teams completed 116 preclinical trials in 8 years, at a total cost of $11 million. The preclinical studies led to multiple clinical trials, many of which are currently underway. One of those clinical trials included the MEK inhibitor selumetinib registration trial.

Make an Impact

Join the NF Registry

The NF Registry is a secure website where people with NF can take an active role in the search for better treatments for all forms of NF. Joining the Registry involves filling out a survey about your symptoms and experiences.

Join the NF Registry


The Children’s Tumor Foundation’s mission is to end NF through research. The funding directed toward this critical research comes from donors like you. Your generosity makes real progress possible.