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CTF Research Portfolio

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.

View Current and Past CTF Funding

49

Active Investigations as of March 2025

CTF Discovery Fund

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

Investigating the role of ZNF423 in NF1-related MPNST 
Sarah Morrow, Indiana University
Young Investigator Award, $68,836

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Mechanisms of MPNST Metastasis
Alexa Sheehan, The University of Iowa
Young Investigator Award, $69,568

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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 - Optic Pathway Glioma (OPG) Consortium

OPG Consortium: Developing Evidence-Based Criteria for Initiating Treatment for NF1-OPG
Drs Robert Avery and Michael Fisher, The Children’s Hospital of Philadelphia
Consortium including 21 sites across the U.S. and Australia, $2,796,008 

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NF1 - Pain Research

Evaluation of a Novel Physiologic Biomarker of Pain in Neurofibromatosis Type 1 – NCI/CNH collaboration
Julia Finkel, Children’s National Medical Center and Children’s Research Institute
Clinical Research Award, $74,613

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Evaluation of a Novel Physiologic Biomarker of Pain in Neurofibromatosis Type 1
Pamela Wolters, National Cancer Institute, Center for Cancer Research
Clinical Research Award, $74,864

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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

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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

Genetic and molecular investigation of the neuronal functions of NF1
Alex Dyson, Massachusetts General Hospital (Mass General)
Young Investigator Award, $150,164

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Investigating the link between sensory and social deficits in a Drosophila model of Neurofibromatosis type I
Jadwiga Bilchak, University of Pennsylvania, School of Veterinary Medicine
Young Investigator Award, $132,468

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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

Risk factors and characteristics of NF1-associated cancer
Roope Kallionpää, University of Turku
 Young Investigator Award, $145,791

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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-SWN and 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

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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

Dissecting DGCR8 syndrome and the molecular mechanisms driving DGCR8-associated schwannomatosis 
Clara Nogué i Ansón, Bellvitge Biomedical Research Institute (Fundació Institut d’Investigació Biomèdica de Bellvitge, IDIBELL)
Young Investigator Award, $57,974

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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

Proteome analysis of inner ear fluids in NF2 mouse models with hearing loss
Isam Naber, The Regents of the University of California, Los Angeles
Young Investigator Award, $155,224

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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

Innovative Trial for Understanding the Impact of Targeted Therapies in NF2 (INTUITT-NF2)
Scott Plotkin, Massachusetts General Hospital (Mass General)
Contract Award, $300,000

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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

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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

Impact of CTF Funding

47

Years of Funding NF Research

200

Young Investigators (YI) Drawn to the NF Field via the YI Award

800

NF Research Projects Funded

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.

CTF ImpactIndustry Partnerships