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Alexion AstraZeneca Rare Disease

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.

A major turning point for NF research occurred in 2020, with the announcement of the FDA approval of selumetinib (Koselugo), the first approved drug for NF. At that time we shared the story of Philip Moss. At the age of 6, Philip’s neck began to swell, which led to a surgery to investigate further. The revelation was life-altering: a plexiform tumor and a diagnosis of NF1. But Philip’s journey led him to a groundbreaking clinical trial for a MEK inhibitor called selumetinib - a drug that reached FDA approval as a result of CTF-funded research.

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.

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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Platform Basket Trial: INTUITT for NF2-SWN with Takeda Pharmaceuticals

The Children’s Tumor Foundation partners with Takeda Pharmaceuticals and six leading medical centers on a clinical trial called INTUITT-NF2, an innovative platform trial that evaluates multiple treatments simultaneously. This trial is currently in progress and testing brigatinib and neratinib in patients with NF2-SWN.

This initiative is a result of the landmark work of our visionary Synodos for NF2 research collaborative, the NF2-SWN Accelerator Initiative, an investment from Takeda Pharmaceuticals, the participation of scientists at the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH), and the vital Synodos NF2 leadership from Massachusetts General Hospital (MGH), Johns Hopkins University (JHU) and Indiana University (IU).

This alliance across the academic, pharma, and patient landscapes has shortened the time from initial research to active trial, thereby bringing promising treatment options to the NF2-SWN patients who need them.

Recorded live on May 3, 2023, the webinar at the link below discussed an exciting progress update on the NF2-INTUITT clinical trial, a first-of-its-kind platform trial dedicated to treatment for NF2-related schwannomatosis.

Watch Now

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.


SpringWorks Therapeutics, a Pfizer-conceived, mission-driven medicines company dedicated to developing innovative potential new treatments for underserved patient communities, launched in 2017 with news of promising investigational therapies, including one for NF. An industry partner with the Children’s Tumor Foundation since the pharmaceutical company formed, SpringWorks is making great strides into research
for new treatments for neurofibromatosis and other rare diseases.

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SpringWorks Therapeutics received the Children’s Tumor Foundation Innovation in Medicine Award at CTF’s 2021 National Gala.

Additional Progress from our Partnerships

Read more about the progress being made by CTF pharma and biotech parters below.

CTF and Healx Global Partnership

CTF has a global partnership with Healx, a leading technology company that will progress new AI (artificial intelligence)-derived therapies for NF.  The Healx team of experts discovered HLX-1502 as a potential treatment for NF1 by applying their innovative AI drug discovery platform.

CTF Funds NF2 Therapeutics Gene Therapy

The Children’s Tumor Foundation has funded NF2 Therapeutics, Inc. to develop a first-in-class gene therapy for NF2-SWN. CTF and NF2 Therapeutics Inc. are optimistic that an NF2 gene therapy will provide a disease-modifying treatment for patients.

CTF and Pharma News

Graphical abstract from The New England Journal of Medicine showing the effects of Brigatinib in NF2-related Schwannomatosis, indicating a decrease in tumor volume for various target tumors.
Collaborations & Partnerships

Brigatinib Shows Promise for NF2-SWN: A Direct Result of CTF’s Synodos Initiative

A landmark study published in the New England Journal of Medicine has revealed promising results…
Collaborations & Partnerships

Funding Opportunity: Developmental and Hyperactive Ras Tumor (DHART) SPORE Program

The Developmental and HyperActive Ras Tumor (DHART) SPORE is a National Cancer Institute (NCI) funded initiative that…
Two individuals standing in front of a banner for the "ASGCT 2024 Annual Meeting" in Baltimore, smiling at the camera.
Awards & Grants

CTF Featured at American Society of Gene & Cell Therapy Annual Meeting

Samantha Ginn and Vidya Browder The Children's Tumor Foundation (CTF) had its partnership with the…

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