The Children’s Tumor Foundation’s strategic plan calls for us to double our investment in research, continuing to drive groundbreaking discoveries that can lead to treatments and cures. No matter what, CTF will ensure that NF science has the necessary resources to flourish.
CTF Discovery Fund
Active Research Investigations as of March 2025
Click each category below to expand and view all of the active investigations.
Neurofibromatosis Type 1 (NF1) Projects
NF1 - MPNST (Malignant Peripheral Nerve Sheath Tumor) Investigations
In vivo assessment of BET blockade combined with PARP inhibition to sensitize MPNST to radiation therapy
Keila Torres, The University of Texas MD Anderson Cancer Center
Drug Discovery Initiative, $199,999
CENPF as a biomarker and therapeutic target for NF1-associated MPNST
Aditya Sheth, Indiana University
Young Investigator Award, $144,778
Utilization of Cell Free DNA Liquid Biopsy for Diagnosis and Monitoring of Tumor Evolution in MPNST
John Shern, National Cancer Institute, NIH NCI Pediatric Oncology Branch – Contract Award, $587,164
Angela Hirbe, Washington University in St.Louis – Contract Award, $1,284,610
Nanopore-based genomic analysis of minimally invasive biopsies of ANNUBP and MPNST for better diagnostics and management
Bernat Gel, Germans Trias i Pujol Research Institute
Clinical Research Award, $149,938
NF1 - Plexiform Neurofibromas
Identification of Biomarkers in Children and Adults with NF1 associated Plexiform Neurofibromas Treated with Binimetinib; an NF 108 Exploratory Aim
Stephen Gilene, Cincinnati Children’s Hospital Medical Center
Clinical Research Award, $101,533
Repurposing Montelukast to treat plexiform neurofibromas in combination with Selumetinib
Daochun Sun, The Medical College of Wisconsin, Inc.
Drug Discovery Initiative, $200,000
NF1 - Cutaneous Neurofibromas
Identification and Functional Validations of Actionable Targets for Prevention or Treatment of Cutaneous Neurofibromas and Characterization of NF1 Healthy-Looking Skin
Pernelle Pulh, Institut National de la Santé et de la Recherche Médical – DR Paris Centre Est
Young Investigator Award, $147,586
NF1 - Gene Therapy Investigations
Targeted delivery of gene replacement therapy for NF1 plexiform neurofibromas
Jiangbing Zhou, Yale University
Contract Award with LOI, $488,375
Too much of a GAP: full-length NF1 reconstitution in neurofibroma and MPNST
Harish Vasudevan, The Regents of the University of California, San Francisco
Contract Award with LOI, $494,445
NF1 - Pain Research
Role of Schwann cells in NF1-related pain development
Michael Jankowski, Cincinnati Children’s Hospital Medical Center
Special Call on Neurofibromatosis and Schwannomatosis Pain, $200,000
The role of Schwann cells in the onset of pain due to neurofibromatosis-1 (NF1)
Namrata Raut, Cincinnati Children’s Hospital Medical Center
Young Investigator Award, $181,275
NF1 - Skeletal and Muscle Research
Developmental Analyses of Skeletal Manifestations in “Mild” Neurofibromatosis Type I Patient Mutation p.M992del in Knock-In Mouse Model
Alexis Stillwell, Board of Supervisors of Louisiana State University Agricultural and Mechanical College represented by Pennington Biomedical Research Center
Young Investigator Award, $71,660
Assessing efficacy of L-carnitine supplementation to treat muscle fatigue and weakness in children with NF1. A placebo controlled, blinded clinical trial.
Yemima Berman, Northern Sydney Local Health District
Contract Award, $228,590
NF1 - Cognitive/Neurological Research
Allele-specific behavioural pharmacogenetics of novel Nf1 mutations
Efthimios Skoulakis, Alexander Fleming Biomedical Sciences Research Center
Drug Discovery Initiative, $77,806
Cognitive aging in middle-aged and older adults with neurofibromatosis type 1: Patterns and predictors
Yang Hou, Florida State University Research Foundation, Inc.
Clinical Research Award, $149,940
NF1 - Other Research
Identifying Moyamoya: MRI and TCD in Children with Neurofibromatosis Type I (MATCh) Study
Laura Lehman, Boston Children’s Hospital
Clinical Research Award, $150,000
Targeting PRMT5 in MTAP-deleted NF1 High Grade Gliomas
Ljubica Caldovic, Children’s Research Institute (CNMC)
Drug Discovery Initiative, $196,213
Pathways that drive inflammation and EMT in Schwann cells after NF1 loss
Ramya Ravindran, Cincinnati Children’s Hospital Medical Center
Young Investigator Award, $71,660
Study of NF1 in Families
Margaret Wallace, University of Florida
Clinical Research Award, $146,348
Creation of a Disease-Specific Measure of Self-Care for Adults with Neurofibromatosis I – A Mixed-Methods Study
Diane Zelman, Alliant International University
Contract Award, $85,311
Characterization of novel bioengineered vectors targeting Schwann cells in a non-human primate model for human gene therapies
Samantha Ginn, Children’s Medical Research Institute, Sydney, Australia
Contract Award, $196,000
NF2-Related Schwannomatosis (NF2-SWN) and Schwannomatosis (SWN) Projects
SWN Investigations
Multiomic integration to identify schwannomatosis predisposition genes and tumorigenesis
Antonio Iavarone, Sylvester Comprehensive Cancer Center/University of Miami Health Systems
Contract Award, $1,733,565
GsMTtx-4 as a novel inhibitor to block non-NF2 SWN pain
Kimberly Ostrow, Johns Hopkins University School of Medicine
Drug Discovery Initiative, $198,400
Exploring the Interplay between Lipid Metabolism and LZTR1 in Peripheral Nerve Pathologies
GEORGIA DARAKI, Leibniz Institute on Aging – Fritz Lipmann Institute e.V.
Young Investigator Award, $71,660
Mechanisms of LZTR1-mediated schwannomatosis pain
Andrei Sdrulla, Oregon Health & Science University – OHSU
Special Call on Neurofibromatosis and Schwannomatosis Pain, $200,000
Partnerships
NF2-SWN - Vestibular Schwannoma and Hearing Loss Investigations
Targeting HIF-2 for the treatment of NF2 Vestibular Schwannoma
Lei Xu, Massachusetts General Hospital (Mass General)
Drug Discovery Initiative, $199,966
Temporal Trends in Plasma Biomarkers: Implications for Hearing and Tumor Progression in People with Vestibular Schwannoma
Konstantina Stankovic, Stanford University School of Medicine
Clinical Research Award, $150,000
Developing a thrombopoietin inhibitor to treat NF2 hearing loss and schwannoma growth
Lawrence Sherman, Oregon Health & Science University – OHSU
Drug Discovery Initiative, $84,744
NF2-SWN - Clinical Trials and Quality of Life
Phase 1/2 clinical trial of PRG-N-01 and data collection by MRI in NF2 patients
Bum-Joon Park, PRG S& TECH INC
Contract Award, $150,000
Demonstrating Clinical Benefit of New Treatments for NF2-related schwannomatosis: Developing A More Comprehensive and Sensitive Measure of Quality of Life for NF2 Clinical Trials
Vanessa Merker, Massachusetts General Hospital (Mass General)
Clinical Research Award, $149,972
Repurposing anti-retroviral drugs to treat NF2 related tumours
Clemens Hanemann, University of Plymouth
Contract Award, $445,141
NF2-SWN - Preclinical Investigations
To understand the role of Apelin mediated angiogenesis in NF2-associated tumors
Srirupa Bhattacharyya, Massachusetts General Hospital (Mass General)
Young Investigator Award, $146,862
Tumor: Macrophage Interactions in Schwannoma
Sara Veiga, Massachusetts General Hospital (Mass General)
Young Investigator Award, $145,791
Deciphering crucial cell death pathways in NF2-related schwannomatosis
Anna Nagel, The University of Central Florida Board of Trustees
Young Investigator Award, $151,272
Development of cell penetrating peptides targeting the Yap/Tead complex in the context of Neurofibromatosis type 2
Dominique Lallemand, Inserm Délégation régionale Paris-IDF Centre Nord
Drug Discovery Initiative, $85,000
Cross-Condition NF Projects
Cross Condition (NF1, NF2-SWN, SWN) Investigations
Supplementing The Children’s Tumor Foundation-Neurofibromatosis Registry with Electronic Health Record Dataset from Mayo Clinic Rochester
Mayowa Osundiji, Mayo Clinic Arizona
Contract Award, $114,348
Clinical Genome Resource (ClinGen) Variant Curation Expert Panel (VCEP) for NF1, NF2 and schwannomatosis
Elisabeth Castellanos-Perez, Germans Trias i Pujol Research Institute
Contract Award, $149,886
Federated deep-learning platform for volumetric quantification of tumor burden in patients with neurofibromatosis on whole-body MRI
Gordon Harris, Massachusetts General Hospital (Mass General)
Clinical Research Award, $149,866
CTF By the Numbers
47
Years of Funding NF Research
200
Young Investigators (YI) Drawn to the NF Field via the YI Award
800
NF Research Projects Funded
2
FDA-approved Drugs for NF, and more on the horizon
CTF-funded research paved the way for two FDA-approved drugs for NF1, Koselugo (selumetinib) and Gomekli (mirdametinib)
3X
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!
47
years of funding NF research
CTF was founded 47 years ago to drive research and support patients with all types of NF.
11,000
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.
20,000
patients each year visit the NF Clinic Network
The NF Clinic Network (NFCN) is dedicated to improving NF clinical care and establishing best practices.
Investments and Partnerships
At the Children’s Tumor Foundation, research funding is a vital part of a broader, ambitious strategy to drive progress for everyone affected by NF. Our efforts go beyond awarding grants: we are committed to expanding the research field, accelerating drug development, fast-tracking clinical trials, and empowering patients, families, and healthcare providers. By investing in innovative science and building the pathways that turn discoveries into real-world treatments, we are transforming the future of NF care—together.
