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CTF’s R&D Enabling Platform Attracts Pharma and Biotech to the NF Field

By May 13, 2022December 18th, 2023Science & Research

The Children’s Tumor Foundation is making available its innovative R&D enabling platform to pharma and biotech companies looking to enter the NF field (neurofibromatosis and schwannomatosis).

The platform was developed by CTF with a key aim of attracting new partners into the NF space, and by providing solutions to the questions most asked by industry partners.

Read below for those questions and the CTF solution:


Does CTF have access to NF patients?  The Children’s Tumor Foundation launched the NF Registry in 2014, the largest of its kind for NF, and which now contains over 10,000+ registrants. It is available in English, French, Italian, Spanish, and soon in German. Visit for more information. Contact Pamela Knight at or Kate Kelts at

Additionally, the Foundation hosts the largest annual gathering of NF patients, both in-person and virtually. Learn more at Contact Traceann Rose at


Are NF patients involved in clinical trial design? Patient engagement is a key priority (and program) of the Children’s Tumor foundation. It includes training classes, participation in endpoint development, clinical network evaluation, grant review and FDA listening sessions. Visit to learn more. Contact Traceann Rose at


Where does one access key opinion leaders in the NF field? CTF is organizer and host of the world’s largest annual scientific meeting in NF, called the NF Conference. Over 500+ experts (researchers, clinicians and others) attend to learn, network, and develop the future of NF research.  Visit Contact Patrice Pancza at


Does CTF have a clinic network?  The Foundation has an active Clinical Care Advisory Board in the United States and in Europe as well, CTF Europe Clinical Care Advisory Board.

CTF also has extensive clinic networks that are audited on an annual basis. In the United States and in Europe.


How can one determine drugs that could be amenable for NF?  CTF offers a custom-made target scan platform through which CTF staff analyze the literature and connects targets to potentially existing drugs. For more information contact CTF Europe’s Chief Scientific Officer Marco Nievo at


Is there NF tissue available for target validation purposes? In collaboration with Indiana University, CTF guarantees an open biobank with tissues from different tumor types.


How does one access scientific data on NF? CTF initiated the first ever NF Data Portal, housed at Sage Bionetworks and is now co-funded by several major NF funders, including CTF. Visit


How does one access preclinical models? We have extensively collaborated with other NF funders to develop a comprehensive catalog of preclinical models. NTAP, one of the collaborating funders, has developed NF cell lines that are available through For the full list of tools, visit here


Which clinical trial endpoints are available? In close collaboration with the REiNS collaborative, CTF has helped develop agreeable endpoints across the clinical trial community globally. Visit


What should the target product profile be for NF drugs? CTF is available as the conduit to the necessary experts to help pharma develop a strong NF TPP. For more information, contact CTF Chief Scientific Officer, Salvatore La Rosa at


Are the CTF services globally available? The Children’s Tumor Foundation has developed a global presence, including an active European foundation at The current focus of CTF Europe is on the development of platform trials for NF through the IMI grant EU-PEARL. For further information, please contact CTF Europe CSO, Marco Nievo at, or Chair of the CTF Europe Board Annette Bakker at


What’s the market possibility for an NF drug? CTF has collaborated with Lapidus Consulting to develop a basic market model based on manifestation incidence. For further information, please contact Marco Nievo at


If there’s anything we didn’t answer above, please contact us at

To watch the R&D Enabling Platform in action, please watch our ‘Connecting the Unconnected to End NF’ video here.