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Giving researchers the tools they to accelerate treatments for NF

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Preclinical Hub

The Children’s Tumor Foundation is accelerating the path to drug discovery by constructing an NF-focused Preclinical Hub to supercharge the development of NF treatments. As our understanding of NF has grown, more potential treatments are now within our grasp, and effective preclinical testing will better predict success once the drug moves into clinical trials.

CTF’s Preclinical Hub speeds innovation and builds bridges across academia, industry, and the medical community. The Preclinical Hub will streamline access to the best preclinical models and guide each potential treatment through a swift and appropriate screening journey, saving time and money.

The CTF Preclinical Hub will catalyze drug discovery, increase testing, and speed the approval of potential treatments.

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Through the NF Preclinical Hub, CTF offers:
  • Negotiated Master Service Agreements
  • Predetermined protocols and tests
  • Access to CTF models, data tools, libraries, and biomarkers

To enter the Hub, your model must be already characterized and validated. If you and your institution are willing to contribute cell lines and/or animal models to the Hub, please email Irene Morganstern (, Director of Preclinical Initiatives.

Colorful Logo that reads NF Registry

The NF Registry represents the collaborative efforts of patients from around the world diagnosed with NF, including neurofibromatosis type 1 (NF1) and all forms of schwannomatosis (SWN), including NF2-related schwannomatosis (NF2-SWN), formerly called neurofibromatosis type 2. The database was created to accelerate research and therapy development for patients affected by NF.

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About the NF Registry

The database houses the information necessary to determine the prevalence and natural history of the disorders as well as to assist in the identification of appropriate candidate patients for a particular study, all of whom have agreed to be contacted by CTF about potential beneficial research. Identifiable information for registry participants will never be released by CTF. For centers conducting clinical trials, participants who may be candidates for studies will be contacted by CTF and provided with contact information; if the patient is interested in potential participation in a study, he or she will contact the study center.

Visit NF Registry

Available Projects

There are two main ways to use NF Registry data:

  1. Recruit patients for an IRB-approved clinical trial or research study.
  2. Request de-identified data maintained in the NF registry for data analysis.

The Data Access Committee, consisting of NF clinicians, patients, and care advocates, reviews all requests.

For all requests, please contact Kate Kelts at

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Schwannomatosis Open Research Collaborative

Banner for schwannomatosis open research collaborative, involving children's tumor foundation, nf data portal, and sage bionetworks.

Need help analyzing schwannomatosis omics or other big data? Apply for the Schwannomatosis Open Research Collaborative Computational Partnership Initiative

With support from the Children’s Tumor Foundation, our partners at Sage Bionetworks have established the Schwannomatosis Open Research Collaborative (SORC) with the goal of identifying genomic features that contribute to disease etiology and heterogeneity in schwannomatosis (SWN), including SMARCB1-related SWN, LZTR1-SWN, 22q-related SWN, SWN-not otherwise specified, and NF2-related SWN, while also expanding the NF community’s capacity for computational research.

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NF Data Portal

To maximize the benefit of data-sharing, we seek the broad participation of fellow NF funders to incentivize grantees to share their data on the NF Data Portal. We also encourage scientists conducting independent research to share their data on the portal voluntarily.


In 2018, The Children’s Tumor Foundation, together with the Neurofibromatosis Therapeutic Acceleration Program (NTAP) and Sage Bionetworks, launched the NF Data Portal, which is now available for researchers. The portal is hosted on the Synapse platform created by Sage, and contains molecular and clinical data coming from the many projects that the Foundation is managing and has managed in recent years. In particular, CTF’s Synodos consortia projects used the platform to execute their data analysis and integration, releasing their datasets during the length of the project in a timely fashion.

The Synodos projects worked with a 12-month embargo on data release, allowing their participants to share results in real-time within an exclusive space, and then releasing them progressively after the confidentiality period. Other projects that the Foundation is managing and sponsoring will use this platform as a resource for making data available to the research community fruition.

By late 2018, over 55 very diverse neurofibromatosis and schwannomatosis studies were available to explore. In addition to data, researchers generated tools, publications, and other types of research that could be linked back to the original studies.

Available Projects

Click below for a list of all CTF-sponsored projects on the NF Data Portal.

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Patient Engagement

CTF Engage bridges the gap between government regulators, researchers, and patients in neurofibromatosis. By collaborating with Patient Representatives, researchers can improve their studies and accelerate the research process.

How Patient Representatives can contribute to your research

  • • Guide research priorities and help you narrow down a research question
  • • Review specific study materials, such as protocols and recruitment strategies
  • • Serve as study advisors (i.e., community advisory boards) and co-investigators
  • • Help disseminate your results to the community (write lay summaries, communicate findings)

By including Patient Representatives, you can make vital connections among the NF community, educating more patients on the value of participating in research.

Past examples of the work of CTF Patient Representatives

  • • Shared their experiences of living with NF at the first-ever NF Listening Session with the U.S. Food and Drug Administration (FDA)
  • • Collaborated to maintain and improve the CTF Patient Registry for clinical trial recruitment and research studies
  • • Acted as patient “mentors” that guided teams during the NF Hackathon initiative
  • • Served as lay reviewers in our grantmaking process
  • • Acted as advisors to CTF research teams such as Synodos, where they shared their stories about what it’s like to live with NF
  • • Assisted the University of Alabama in recruiting patients to participate in the initial review of an intimacy study survey that will be sent out to the patient community
  • • Gone on to serve in REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) working groups


If you are looking to work in collaboration with Children’s Tumor Foundation Patient Representatives, please reach out to to discuss how we can work together.


REiNS is an international effort to develop new standardized response criteria for determining treatment response in patients with all types of neurofibromatosis and schwannomatosis. Response criteria will continue to be modified as we gain experience in clinical trials for NF. We hope these criteria will be incorporated into future clinical trials and will improve our ability to determine and compare treatment efficacy.


The Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) international collaboration was established in 2011 at the Children’s Tumor Foundation annual NF conference to achieve consensus within the NF community about the design of future clinical trials, with a specific emphasis on endpoints.

The REiNS Collaboration includes nine working groups that focus on specific areas including: imaging of tumor response; functional, visual, neurocognitive, and patient-reported outcomes; disease biomarkers; cutaneous neurofibromas; patient representatives; and the newest working group, gene therapy.

The hope is that the recommendations from this collaborative effort will be used by all researchers to standardize the measurement of outcomes and thus improve clinical trials and other outcome measures for individuals with NF. Children’s Tumor Foundation provides ongoing support for many REiNS efforts.

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The Children’s Tumor Foundation human tissue biobank is an open collection of tissue samples donated by patients who want to create a meaningful legacy by accelerating NF research.


Collecting began in 2013 as a way to accelerate research by ensuring that high-quality NF-related human tissue resources are widely available. The first project was a collection of surgical samples of cutaneous neurofibromas, which have since been used better to understand the genomic and structural characteristics of these tumors. In 2014, we broadened the collection to include post-mortem tissue donated by NF patients and their families. It now includes a variety of tumor and non-tumor tissue and organs from people with neurofibromatosis type 1 (NF1) and schwannomatosis (SWN), including NF2-related schwannomatosis (NF2-SWN).


As our biobank grew, CTF sought a non-profit partner to house the collection. We were fortunate to partner with Indiana University’s Developmental and HyperActive Ras Tumor Specialized Programs of Research Excellence (DHART SPORE) Biorepository. The CTF-Indiana agreement strengthens the sustainability of our collections. Under its terms, CTF retains a decision-making role in allocating the tissue we collect and has ensured that the material remains openly available to researchers.

Sample Inventory

For information on sample inventory and how to request access, please visit the CTF Biobank within the DHART SPORE Biorepository Core.

Clinical Trials Pipeline

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.

We suggest searching for past and present clinical trials for all forms of neurofibromatosis and schwannomatosis.

Search US NF clinical trialsSearch European NF Clinical Trials
Two people in lab coats working on a microscope.

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