Our partners

NTAP brings together patients, clinicians, researchers, industry, and government to accelerate scientific research on neurofibromatosis type 1 (NF1)-associated plexiform neurofibroma (pNF) and cutaneous neurofibroma (cNF). We work with patient organizations to make sure NF1 research is directed to where it will have the greatest impact.

By collaborating across continents and institutions, we challenge assumptions inherent to traditional approaches for this condition and encourage the development of innovative strategies to treat pNFs and cNFs. NTAP fosters relationships among research initiatives that accelerate efforts to generate reagents and resources for development of effective NF1 therapies.

Patient organizations

Ultimately, a therapy is effective only if it treats the clinical issues that matter to patients. NTAP works with patient organizations to enable global clinical studies, including natural history studies and patient reported outcome initiatives. This allows us to identify patient priorities and ensure that therapeutic development programs are focused on addressing their needs.

  • The Children’s Tumor Foundation (CTF)
  • Department of Defense Neurofibromatosis Consortium
  • Cancer Therapeutic Evaluation Program (CTEP)
  • National Center for Advancing Translational Studies (NCATS)
  • Neurofibroma Research Initiative (NF Research Initiative)
  • Neurofibromatosis MidAtlantic
  • Neurofibromatosis MidWest
  • Brain Science Institute (BSI)


Centers and Institutes

Although our offices are housed at Johns Hopkins University, we fund innovative investigators at research centers and institutes around the world. We continuously seek out skilled investigators with the creativity and expertise required to develop therapies for plexiform and cutaneous neurofibromas. Our partners include:

  • BioBasix, Inc.
  • Bispebjerg University Hospital, Denmark
  • Brigham and Women’s Hospital
  • Case Western Reserve University School of Medicine
  • Center for Cancer Research, NCI, NIH
  • Children’s Hospital of Philadelphia (CHOP)
  • Children’s National Health System
  • Children’s National Medical Center
  • Cincinnati Children’s Hospital
  • Department of Defense (DoD)
  • East Carolina University
  • Galen Research Ltd., United Kingdom
  • Harvard University
  • Hôpital Henri-Mondor, France
  • Iconcologia
  • Indiana University
  • Infixion Bioscience, Inc.
  • Innocentive
  • Istituto di chimica Biomolecolare (ICB), Italy
  • Institute of Predictive and Personalized Medicine of Cancer (IMPPC), Spain
  • Massachusetts General Hospital
  • Medical Research Council, Scotland
  • Memorial Sloan Kettering Cancer Center (MSKCC)
  • Michigan State University College of Human Medicine
  • National Cancer Institute (NCI)
  • Northwestern University
  • Padova University
  • Rainbow Babies and Children’s Hospital
  • Riley Hospital for Children
  • Royal North Shore Hospital, Australia
  • Sage Bionetworks
  • Spectrum Health Systems, Inc.
  • The Board of Trustees of the Leland Stanford Junior University (Stanford)
  • The Brain Science Institute (BSi)
  • The Childhood Brain Tumor Foundation
  • The Children’s Tumor Foundation (CTF)
  • The European Medical Administration (EMA)
  • The FDA, Drug Development Tools (DDTs) Qualification Program
  • The Medical College of Wisconsin
  • The NIH National Center for Advancing Translational Sciences (NCATS)
  • The NIH Office of Rare Diseases
  • The Institute for Health Science Research Germans Trias i Pujol (IGTP), Spain
  • The Institut national de la santé et de la recherche médicale (Inserm), France
  • The Pediatric Oncology Branch of the National Cancer Institute (POB-NCI)
  • The US Food and Drug Administration (FDA)
  • TOOsonix, Denmark
  • University College of London, United Kingdom
  • University Health Network (University of Toronto), Canada
  • University of Alabama at Birmingham
  • University of California Irvine
  • University of California San Francisco (UCSF)
  • University of California Los Angeles (UCLA)
  • University of Chicago
  • University of Florida
  • University of Iowa
  • University of Manchester, United Kingdom
  • University of MichiganUniversity of Milan
  • University of Padua, Italy
  • University Paris-Est Créteil (UPEC), France
  • University of Sydney, Australia
  • University of Texas (UT) Southwestern
  • University of Turku, Finland
  • Van Andel Institute
  • Washington University in St. Louis
  • Wayne State University


An international effort is required to address the unique challenges associated with rare disease drug development. Given the relatively low prevalence and high variability seen with plexiform and cutaneous neurofibromas, only a collaborative, multi-center effort can bring together the patients and researchers that will advance new therapies. We work closely with these academic and clinical consortia:



NTAP initiatives are informed by a clear understanding of the challenges faced by drug companies who work in rare disorders. Engagement with industry partners ensures that essential data and resources are in place to de-risk NF1 therapeutic programs and remove obstacles that could otherwise delay therapies reaching the market. NTAP actively supports collaboration between investigators and industry to help refine research strategies and access the best available resources.

Read about our partnerships with biopharmaceutical and biotech companies.



It’s critical to have a clear understanding of patient priorities and an effective means to assess clinical relevance, particularly in rare diseases where advances can be delayed due to a lack of precedent with the groups that must evaluate the efficacy of novel treatments. NTAP consults with regulatory and other governmental agencies to ensure our initiatives will provide the data necessary to meet the rigorous requirements for drug approval and reimbursement support.

NTAP has played a key role in the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) international collaboration since its inception. In 2017, NTAP played a role in helping to establish the “Response Evaluation in Neurofibromatosis & Schwannomatosis International Collaboration cNF working group”, through which NTAP has regular interaction with representatives from the FDA to address the challenges in the “clinical development of cNF therapies”. NTAP also interfaced with the FDA as part of its role on the clinical study evaluating the small molecule inhibitor Selumetinib for pNF. Finally, in collaboration with the Children’s Tumor Foundation, NTAP engages with the FDA for patient engagement opportunities and to participate in scientific symposia.