About

Garrett M. Brodeur, M.D., is a Professor of Pediatrics (Oncology) at the Perelman School of Medicine at the University of Pennsylvania and a pediatrician at The Children's Hospital of Philadelphia. His research expertise centers on the molecular biology and genetics of childhood cancer, with a particular focus on neuroblastoma, pediatric cancer predisposition, and surveillance. Dr. Brodeur has made significant contributions to understanding the prognostic markers and genetic underpinnings of neuroblastoma, including the identification of MYCN amplification as a prognostic marker and the cloning of the tumor suppressor gene CHD5. He has also elucidated the roles of TrkA and TrkB receptors in neuroblastoma behavior and has been involved in developing targeted therapies such as TRK inhibitors. As the Director of the Cancer Predisposition Program at CHOP, he is engaged in identifying novel cancer predisposition genes, developing surveillance protocols, and expanding research in pediatric cancer predisposition. His work integrates molecular profiling, targeted drug delivery via nanoparticles, and clinical research to improve outcomes for children with cancer.

Research topics

• Medicine
• Biology
• Cancer research
• Internal medicine
• Genetics

Selected publications

• Pediatric Cancer Predisposition and Surveillance Update: Summary Perspective and Future Directions

  Clinical Cancer Research · 2025-05-06 · 10 citations

  reviewOpen access1st authorCorresponding

  An increasing number of studies suggest that a significant proportion of children with cancer harbor an underlying predisposition to malignancy, and it is likely that this proportion will only increase. Targeted surveillance for these individuals would likely improve outcomes. Historically, however, for most predisposition syndromes, there were no standardized surveillance protocols for early detection of cancer in predisposed individuals. Therefore, the Pediatric Cancer Working Group of the American Association for Cancer Research convened a workshop in 2016 to develop consensus surveillance recommendations (published in 2017) for children and adolescents with the most common cancer predisposition syndromes. These recommendations provided a consistent approach for pediatric oncologists and other care providers to use as a plan for cancer surveillance in pediatric patients with these syndromes. We held a second workshop in 2023 to update recommendations based upon new data, as well as to add syndromes that were newly described or not addressed in the prior workshop. The resulting articles represent updated surveillance recommendations for currently recognized predisposition syndromes, organized along similar themes. We also address novel approaches to surveillance that are under investigation, as well as prospects for prevention trials for these high-risk populations.

  PublisherOA PDFDOI

• Supplementary Figure S2 from Radiation Concepts and Considerations for Pediatric Cancer Predisposition Syndrome Surveillance: A Report from the 2023 AACR Childhood Cancer Predisposition Workshop

  2025-09-02

  articleOpen access

  <p>Main concerns or challenges practitioners report when assessing the relative risk of ionizing radiation-based imaging for pediatric cancer predisposition syndromes</p>

  PublisherDOI

• Update on Surveillance in Von Hippel–Lindau Disease

  Clinical Cancer Research · 2025-04-15 · 8 citations

  reviewOpen access

  Von Hippel-Lindau disease (VHL) is a genetic condition characterized by a high lifetime risk for tumors and cysts throughout the body, including the central nervous system, visual-auditory systems, and intra-abdominal organs. This neoplasia leads to significant morbidity and potential mortality in affected individuals. Tumor surveillance enables early intervention and leads to improved clinical outcomes. Since the 2017 publication of VHL tumor surveillance recommendations from the inaugural American Association for Cancer Research Childhood Cancer Predisposition Workshop, several other groups have proposed alternative consensus surveillance recommendations. Although these screening paradigms share some common elements, they also deviate from each other in some substantial ways. Clinical data continue to accrue in VHL, allowing the condition to be better characterized. Furthermore, surgical techniques have improved over time, and the option of targeted medical therapy has emerged for individuals with VHL. It is critical that surveillance strategies continue to be refined. In this perspective, we provide an up-to-date clinical overview of VHL, describe recently proposed tumor screening regimens, and finally present our updated consensus tumor surveillance recommendations during childhood and adolescence from the 2023 American Association for Cancer Research Childhood Cancer Predisposition Workshop.

  PublisherOA PDFDOI

• Updated Recommendations for Pediatric Surveillance in Hereditary Endocrine Neoplasia Syndromes: Multiple Endocrine Neoplasias, Hyperparathyroidism–Jaw Tumor Syndrome, and Carney Complex

  Clinical Cancer Research · 2025-06-25 · 8 citations

  articleOpen access

  Hereditary endocrine neoplasia syndromes comprise multiple entities associated with an increased risk for the development of endocrine and nonendocrine neoplasms and other systemic manifestations. These syndromes typically demonstrate autosomal dominant inheritance, and each syndrome is associated with a unique genetic predisposition to a distinct spectrum of tumor susceptibility. Moreover, genotype-phenotype associations within each syndrome may affect the spectrum, penetrance, and age of onset of associated tumors. As many endocrine tumors are benign and/or indolent, a careful approach to monitoring is necessary, wherein the nature, timing of initiation, and frequency of presymptomatic surveillance balance the goal of detecting tumors at a point in which intervention would limit tumor-associated morbidity against the physical, emotional, and financial burdens of surveillance. In this study, we summarize changes in knowledge and practice recommendations related to children with multiple endocrine neoplasia syndromes (types 1, 2A, 2B, 4, and 5), hyperparathyroidism-jaw tumor syndrome, and Carney complex since an initial summary in 2017. These updates reflect the evolving understanding of these complex genetic disorders and aim to improve patient care and outcomes.

  PublisherOA PDFDOI

• Update on Tumor Surveillance for Children with Hereditary Pheochromocytoma/Paraganglioma Syndromes

  Clinical Cancer Research · 2025-06-23 · 4 citations

  reviewOpen access

  Hereditary pheochromocytoma/paraganglioma syndromes (HPPS) are a collection of conditions caused by variants in genes producing subunits of the succinate dehydrogenase (SDH) complex or related proteins. These conditions are characterized by substantial lifetime risks for developing pheochromocytomas, paragangliomas, and other tumors. Affected individuals who develop these tumors may experience severe, acute, and chronic problems. Indeed, aggressive, malignant, and/or disseminated tumors may result in death. Tumor surveillance enables early intervention, which, in turn, should lead to improved clinical outcomes. However, the desire for intensive surveillance strategies must be balanced against medical and psychosocial risks. In 2017, consensus HPPS surveillance recommendations addressing germline predisposition to SDHA-, SDHAF2-, SDHB-, SDHC-, SDHD-, MAX-, and TMEM127 (collectively, SDHx+)-related tumors were published after the inaugural American Association for Cancer Research Childhood Cancer Predisposition Workshop. Based on the limited available clinical data at that time, these recommendations advocated a uniform approach to tumor surveillance in HPPS. Since then, several other groups have proposed alternative consensus surveillance guidelines. Although these surveillance approaches share some common elements, including recommendations tailored to emerging differences in tumor phenotype based on underlying specific SDHx+ genes, these approaches also vary significantly among each other. As clinical data continue to accrue, it is critical that surveillance strategies continue to be refined to address emerging genotype-phenotype differences. In this review, we provide a brief up-to-date clinical overview of HPPS and describe recently proposed tumor surveillance regimens. We then detail our updated consensus pediatric-focused tumor surveillance recommendations from the 2023 American Association for Cancer Research Childhood Cancer Predisposition Workshop.

  PublisherOA PDFDOI

• International neuroblastoma risk group consortium: a model of networking for rare cancers

  JNCI Journal of the National Cancer Institute · 2025-08-23 · 4 citations

  articleOpen access

  It is critical to share knowledge and harmonize approaches to optimize progress in rare cancers. The International Neuroblastoma Risk Group (INRG) Task Force was formed by the 4 major neuroblastoma cooperative groups in 2004 to achieve this goal. Strategies developed for neuroblastoma are an exemplar for other rare malignancies. Data from an initial cohort of 8800 patients were transferred to the INRG Data Commons, and a data-sharing model was developed. Currently, information on more than 25 000 patients is available to the research community. The INRG staging and risk classification systems have led to harmonized approaches for therapeutic groupings. INRG consensus manuscripts have led to uniform criteria for classifying biological data, evaluating the extent of disease, and defining treatment response. More than 40 INRG research studies have been performed by investigators from around the world, including analyses of rare patients, which would not otherwise be possible. The success of this approach for neuroblastoma has been leveraged to create the Pediatric Cancer Data Commons and the Data for the Common Good. Efforts to enrich the INRG Commons with additional genomic and biomarker data, extracted electronic health records, and digital medical images are ongoing. The international networking model developed by the INRG Task Force has led to new research discoveries and progress in neuroblastoma. The approach has now been applied to 16 other cancers and conditions, including rhabdomyosarcoma, germ cell tumor, Lynch syndrome, and cancer predisposition. This framework of international collaboration and data sharing serves as a model for advancing rare adult malignancies.

  PublisherOA PDFDOI

• Data from Radiation Concepts and Considerations for Pediatric Cancer Predisposition Syndrome Surveillance: A Report from the 2023 AACR Childhood Cancer Predisposition Workshop

  2025-09-02

  articleOpen access

  <div>Abstract<p>Children with cancer predisposition syndromes have an increased risk of developing certain cancers. Surveillance imaging plays an increasingly important role in the management of these children. The approach to imaging must account for several factors, including age of the child, onset and frequency of imaging, whether sedation is needed, and the types of tumors associated with a particular syndrome. Consideration must also be given to the potential risks and benefits associated with a given imaging technique. Whole-body MRI offers many advantages, including a comprehensive examination without ionizing radiation. Other techniques, such as CT, involve low doses of ionizing radiation but may be appropriate depending on the clinical circumstance. This article offers an overview of available imaging techniques along with potential strategies to guide the discussion with patients and families when deciding on the most appropriate surveillance imaging approach.</p></div>

  PublisherDOI

• Supplementary Text File S1 from Radiation Concepts and Considerations for Pediatric Cancer Predisposition Syndrome Surveillance: A Report from the 2023 AACR Childhood Cancer Predisposition Workshop

  2025-09-02

  articleOpen access

  <p>Perspectives and Understanding of Radiation Risks in CPS: A Practitioner Survey</p>

  PublisherDOI

• Radiation Concepts and Considerations for Pediatric Cancer Predisposition Syndrome Surveillance: A Report from the 2023 AACR Childhood Cancer Predisposition Workshop

  Clinical Cancer Research · 2025-06-25 · 3 citations

  articleOpen access

  Children with cancer predisposition syndromes have an increased risk of developing certain cancers. Surveillance imaging plays an increasingly important role in the management of these children. The approach to imaging must account for several factors, including age of the child, onset and frequency of imaging, whether sedation is needed, and the types of tumors associated with a particular syndrome. Consideration must also be given to the potential risks and benefits associated with a given imaging technique. Whole-body MRI offers many advantages, including a comprehensive examination without ionizing radiation. Other techniques, such as CT, involve low doses of ionizing radiation but may be appropriate depending on the clinical circumstance. This article offers an overview of available imaging techniques along with potential strategies to guide the discussion with patients and families when deciding on the most appropriate surveillance imaging approach.

  PublisherOA PDFDOI

• Update on Retinoblastoma Predisposition and Surveillance Recommendations for Children

  Clinical Cancer Research · 2025-02-25 · 10 citations

  reviewOpen accessSenior author

  Hereditary retinoblastoma is a classic cancer predisposition syndrome with risks beginning in early infancy. About 45% of children with retinoblastoma (RB) have hereditary disease. These children are at risk for both intraocular disease and additional neoplasms throughout their lifetime. Germline pathogenic/likely pathogenic variants in RB1 typically lead to bilateral intraocular disease, elevated risks of trilateral RB, and risks of non-ocular subsequent malignant neoplasms (SMN), especially sarcomas and melanomas. There is further increased risk of SMNs if radiation treatment is used. In this report, with a reconvening of the American Association for Cancer Research (AACR) Childhood Cancer Predisposition Workshop, we expand on strategies for identifying individuals with hereditary RB, with a focus on testing strategies for children with RB. We also provide updates from previous recommendations. Given the high penetrance of retinal tumors, we review the importance of close intraocular surveillance and consider recent data on surveillance for SMNs. Finally, we discuss the importance of counseling for survivors of intraocular disease to address risks of adult-onset tumors as well as to consider reproductive risks.

  PublisherOA PDFDOI

Recent grants

• NIH Grant K04CA001027

  NIH · $243k · 1990

• NIH Grant P01NS034514

  NIH · $10.1M · 2002

• NIH Grant R01CA039771

  NIH · $7.5M · 2015

• NIH Grant R01CA094194

  NIH · $3.7M · 2014

• Prodrugs targeting norepinephrine transporter for dual-selective therapy of refractory neuroblastoma

  NIH · $3.1M · 2020–2025

Frequent coauthors

• Robert P. Castleberry

  University of Alabama at Birmingham

  311 shared

• Susan L. Cohn

  268 shared

• John M. Maris

  256 shared

• A. Thomas Look

  Dana-Farber Cancer Institute

  242 shared

• Wendy B. London

  227 shared

• E. Ide Smith

  Arizona Oncology

  156 shared

• Katherine K. Matthay

  University of California, San Francisco

  143 shared

• Vijay Joshi

  Indian Institute of Space Science and Technology

  137 shared

Education

• MD, Medicine

  Washington University School of Medicine in St. Louis

  1975

• BA, Chemistsry

  St. Louis University

  1971

Awards & honors

• Child Health Research Career Development Award (CHRCDA)
• National Research Service Award (NRSA) Institutional T32 Tra…