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Collaborating for a Better Tomorrow: Our Partnership with Pharma and Biotech

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Collaborating for a Better Tomorrow

At the Children’s Tumor Foundation, we believe collaboration plays a critical role in groundbreaking research and innovation. And we’re passionate about accelerating the journey from discovery to real-world impact. That’s why we actively seek partnerships with pharmaceutical and biotech companies to catalyze advancements that positively impact patients’ lives.


FDA-approved Drug for NF, and more on the horizon

CTF-funded research paved the way to the first FDA-approved drug for NF, Koselugo (selumetinib).


the number of NF Clinical Trials in the past 10 years

There are currently 67 active clinical trials for NF – three times the number of trials than just ten years ago!


years of funding NF research

CTF was founded 45 years ago as the first grassroots organization to support patients with all types of NF.


have joined 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.


patients each year visit the NF Clinic Network

The NF Clinic Network (NFCN) is dedicated to improving NF clinical care and establishing best practices.

Partner With Us

When you partner with CTF, you access:

  • A seasoned team of experts who have navigated the path from drug discovery to FDA/EMA approval for NF, and are ready to do it again
  • A rich ecosystem of resources, including our Preclinical Hub, effective experimental models, robust data, and the NF Patient Registry
  • Our deep commitment to scientific innovation and agile methodologies that will result in tangible outcomes

We invite pharmaceutical and biotech companies who share our passion for pioneering research and transformative solutions to join us. Together, let’s shape the future and make a lasting difference in the lives of countless patients.

Contact Us

Click the image above to download our pitch deck.

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.

Through the NF Preclinical Hub, CTF offers:

  • Negotiated Master Service Agreements
  • Predetermined protocols and tests
  • Access to CTF models, data tools, libraries, and biomarkers
  • A unique pipeline, from concept to clinic

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

Enter the Preclinical Hub

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, click Contact Us to get started.

Why Pharma Should Invest in NF

The Children’s Tumor Foundation answers the questions pharmaceutical companies are asking about investing in a rare disease such as NF.

Are teams of scientists working on this problem?


CTF values collaboration throughout the drug discovery process. Diverse teams of experts managed by CTF share discoveries and data, increasing research efficiency.
Where can we find patients for clinical trials?

The NF Registry is a patient-entered registry structured to accelerate clinical trial recruitment and fuel knowledge of the diversity of NF manifestations.

Are there other investors working in the NF space?
CTF’s significant investment in a Phase 2b clinical trial at NFlection Therapeutics further established CTF as a co-investor with pharma in innovative NF research.
How can we standardize the endpoints of clinical trials?
This worldwide consortium of clinicians and patients develops clinical trial designs and works with the FDA on drug approval criteria.
Where do we find experts in this field?
This expert network of specialists helps to guide drug discovery and development in order to increase scientific and clinical quality in decision-making.
Where can we find new drug targets?

The NF Data Portal is a centralized repository that collects, analyzes, and releases integrated data, accelerating identification of ‘druggable’ targets.

Are there care and treatment centers?

A growing network of CTF-affiliated clinics is cultivating relationships between patients and doctors while improving and standardizing NF patient care.

Have these drugs been tested in animal models?

CTF has invested in teams of top laboratories with NF-relevant animal models, bringing promising drug treatments to the clinic quickly and efficiently.

Are there enough patients engaged in the drug discovery process?

CTF Engage, our patient engagement training program, has a team of patient representatives knowledgeable in all aspects of NF drug discovery.

Is there enough tissue available for testing?

CTF created a centralized library of openly available samples for biomarker discovery and development, managed by the Indiana University School of Medicine

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.

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:

Recruit patients for an IRB-approved clinical trial or research study.

Request de-identified data maintained in the NF registry for data analysis.

All requests are reviewed by the Data Access Committee, consisting of NF clinicians, patients, and care advocates.

For all requests, please contact Kate Kelts at

Learn More

Patient Engagement

CTF Engage is the patient engagement initiative of the Children’s Tumor Foundation, dedicated to transforming the landscape of NF research through the curation of quality, meaningful interactions between patients and families with research stakeholders. Our mission is to support scientific projects of merit that generate value for the NF community. By collaborating with Patient Representatives, your team can better understand the issues that are important to NF patients of all backgrounds – and build trust and connections with the community.

How can you engage with the NF community?  Download our toolkit to learn more.

Tools for Pharma & Industry

How Patient Representatives can lend their perspectives to your projects:

  • – Serve as study advisors or members of community advisory boards
  • – Provide input on study design, such as protocols, endpoints, and recruitment materials
  • – Guide research priorities and give insight on community perspectives for therapies
  • – Be storytellers and speak with your team about lived experiences

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.

Pharma Success Story

45 Years of Impact Video

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