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CTF Progress in NF2-SWN

Platform Basket Trials: INTUITT for NF2-SWN

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 for NF2-SWN simultaneously. This initiative is a result of the landmark work of our visionary Synodos for NF2 research collaborative, our NF2 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).

A direct outcome of this collaborative partnership and research initiative, spearheaded by the Children’s Tumor Foundation (CTF), found promising evidence that brigatinib can help shrink the tumors of patients with NF2-related schwannomatosis. Driven by CTF’s mission to End NF, 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.

Brigatinib: Breaking News

In 2024, the New England Journal of Medicine published promising results from the INTUITT-NF2 trial on the use of brigatinib in treating NF2-related schwannomatosis. In this highly innovative phase II platform-basket trial, the multi-center team found that brigatinib shrunk 10% of growing tumors and 23% of all tumors. Some patients also experienced better hearing and said they had less pain.

Read the full press release at this link.

Recorded live on May 3, 2023, this webinar discussed an exciting progress update on the INTUITT-NF2 clinical trial dedicated to treatment for NF2-related schwannomatosis.

Watch Now.

Read more about CTF platform basket trials

CTF is a funding partner with EU-PEARL, 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.

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.

What is a Platform Trial?

Selumetinib

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.

Currently, selumetinib is in a Phase 2 clinical trial for tumors in NF2-related schwannomatosis.

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.

Read more about Selumetinib

CTF Grant Funding

At CTF, we are expanding our research portfolio across multiple fronts. Part of our strategy is to double research funding and create more “shots on goal” in our fight to find treatments for NF. Our Discovery Fund supports vital research discovery by funding individual researchers as well as collaborations, and encompasses the Young Investigators Award (YIA), Drug Discovery Initiative (DDI), Clinical Research Awards (CRA), Contract Awards, Gene Therapy awards, and other designated research investigations.

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

Explore CTF-Funded Research Projects

Impact of CTF Funding

47

years of funding NF research

200

Young Investigators were drawn to the NF field via the YIA

800

NF Research Projects Funded

NF2-SWN Research Highlights

NF2-SWN YIA & DDI Research Awards

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NF2 Gene Therapy Funding

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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.

NF2-related schwannomatosis (NF2-SWN) was referred to as “neurofibromatosis type 2” or NF2 until May 2022. For the time being, we suggest searching ClinicalTrials.gov for both of these terms.

Search forNF2-Related Schwannomatosis Clinical Trials
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 www.nfregistry.org 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 www.clinicaltrialsregister.eu.

CTF Team Science and Data Sharing

IMPACT OF TEAM SCIENCE

1931

Drugs screened in Synodos for NF2

8

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

10

Pharma/biotech companies drawn toward NF

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.

NF2 Accelerator Initiative

The Children’s Tumor Foundation NF2-SWN Accelerator Initiative was dedicated to finding effective treatments for NF2-related schwannomatosis, or NF2-SWN (previously called neurofibromatosis type 2, or NF2). This powerful initiative brought treatments to the clinic and patients by expanding the clinical drug pipeline for NF2-SWN, improving drug selection through innovative testing models, and developing gene therapy options that address the underlying genetic causes of NF2-SWN.

Read our NF2 Accelerator Impact Report Here. 

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.

Read More

Synodos for NF2

The first of the Synodos models established by CTF, Synodos for NF2 launched in 2014 to provide clarity to patients about available drug options, and completed its work in 2018. This consortium of multidisciplinary investigators from 12 world-class labs and medical centers performed rigorous drug testing that laid the groundwork for further research and clinical trials currently in progress, including the INTUITT-NF2 platform trial.

Synodos has represented a significant financial commitment on the part of the Foundation. In each Synodos collaboration, CTF assembles “dream teams” of doctors, scientists, and patients who work together to solve problems that are too complex for any individual lab or researcher to solve. Our goal is to speed the drug discovery process through innovative research methods, collective knowledge, and data shared openly through CTF’s NF Data Portal. This data is analyzed by our partners at Sage Bionetworks and is then made available to the rest of the world. This approach is expanding interest in NF research to additional fields, including cancer.

Read more about the Progress of Synodos for NF2 here. 

Following Synodos for NF2, the Foundation launched Synodos for NF1 and Synodos for Schwannmatosis.

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.

Read More

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 is as simple as filling out a survey about your symptoms and experiences.

Join the NF Registry

Donate

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.

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