A road map for the treatment of pediatric diffuse midline glioma

Carl Koschmann¹, Wajd N Al-Holou², Marta M Alonso³, Jamie Anastas⁴, Pratiti Bandopadhayay⁵, Tara Barron⁶, Oren Becher⁷, Rodrigo Cartaxo², Maria G Castro², Chan Chung⁸, Madison Clausen², Derek Dang², Robert Doherty², Ryan Duchatel⁹, Matthew Dun⁹, Mariella Filbin⁵, Andrea Franson², Stefanie Galban², Marc Garcia Moure³, Hugh Garton², Pruthvi Gowda⁵, Joana Graca Marques³, Cynthia Hawkins¹⁰, Allison Heath¹¹, Esther Hulleman¹², Sunjong Ji², Chris Jones¹³, Lindsay Kilburn¹⁴, Cassie Kline¹¹, Michael A Koldobskiy¹⁵, Daniel Lim¹⁶, Pedro R Lowenstein², Q Richard Lu¹⁷, Joanna Lum², Stephen Mack¹⁸, Suresh Magge², Bernard Marini², Donna Martin², Neena Marupudi², Dana Messinger², Rajen Mody², Meredith Morgan², Mateus Mota², Karin Muraszko², Sabine Mueller¹⁹, Siva Kumar Natarajan², Javad Nazarian²⁰, Michael Niculcea², Nicholas Nuechterlein²¹, Hideho Okada¹⁶, Valerie Opipari², Manjunath P Pai², Sharmistha Pal⁵, Erik Peterson², Timothy Phoenix¹⁷, John R Prensner², Matthew Pun², G Praveen Raju⁷, Zachary J Reitman²², Adam Resnick¹¹, David Rogawski⁶, Amanda Saratsis²³, Stefanie G Sbergio¹⁰, Mark Souweidane²⁴, James M Stafford²⁴, Theophilos Tzaridis²⁵, Sujatha Venkataraman²⁶, Orazio Vittorio²⁷, Jack Wadden², Daniel Wahl², Robert J Wechsler-Reya²⁸, Viveka Nand Yadav²⁹, Xu Zhang²⁸, Qiang Zhang², Sriram Venneti³⁰

Affiliations

¹ University of Michigan, Ann Arbor, MI 48109, USA. Electronic address: ckoschma@med.umich.edu.
² University of Michigan, Ann Arbor, MI 48109, USA.
³ Clinica Universidad de Navarra, Navarra, Spain.
⁴ Baylor College of Medicine, Houston, TX 77030, USA.
⁵ Dana-Faber Cancer Institute, Boston, MA 02215, USA.
⁶ Stanford University, Stanford, CA 94305, USA.
⁷ Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
⁸ Daegu Gyeongbuk Institute of Science & Technology, Daegu, South Korea.
⁹ University of Newcastle, Callaghan, NSW 2308, Australia.
¹⁰ Hospital for Sick Children, Toronto, ON M5G 1E8, Canada.
¹¹ Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
¹² Princess Maxima Center, 3584 CS Utrecht, the Netherlands.
¹³ Division of Molecular Pathology, Institute for Cancer Research, London SM2 5NG, UK.
¹⁴ Children's National, Washington, DC 20010, USA.
¹⁵ John Hopkins University, Baltimore, MD 21205, USA.
¹⁶ University of California, San Francisco, San Francisco, CA 94143, USA.
¹⁷ Cincinnati Children's Hospital Medical Center, and University of Cincinnati, Cincinnati, OH 45229, USA.
¹⁸ St. Jude, Memphis, TN 38105, USA.
¹⁹ University of California, San Francisco, San Francisco, CA 94143, USA; Parker Institute for Cancer Immunotherapy, University of Zurich, Zurich, Switzerland.
²⁰ Children's National, Washington, DC 20010, USA; University of Zurich, Zurich, Switzerland.
²¹ University of Michigan, Ann Arbor, MI 48109, USA; National Institutes of Health, Bethesda, MD, USA.
²² Duke Univesity, Durham, NC 27708, USA.
²³ Advocate Children's Hospital, Park Ridge, IL 60068, USA.
²⁴ Weill Cornell Medicine, New York Presbyterian and Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA.
²⁵ Herbert Irving Comprehensive Cancer Center and Department of Neurology, Columbia University Medical Center, New York, NY 10032, USA.
²⁶ University of Colorado, Denver, CO 80204, USA.
²⁷ School of Biomedical Sciences, Faculty of Medicine and Health, University of New South Wales, Sydney, NSW, Australia.
²⁸ Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA 92037, USA.
²⁹ Children's Mercy, Kansas City, MO 64108, USA.
³⁰ University of Michigan, Ann Arbor, MI 48109, USA. Electronic address: svenneti@med.umich.edu.

PMID: 38039965
PMCID: PMC11067690
DOI: 10.1016/j.ccell.2023.11.002

A road map for the treatment of pediatric diffuse midline glioma

Carl Koschmann et al. Cancer Cell. 2024.

. 2024 Jan 8;42(1):1-5.

doi: 10.1016/j.ccell.2023.11.002. Epub 2023 Nov 30.

Authors

Affiliations

¹ University of Michigan, Ann Arbor, MI 48109, USA. Electronic address: ckoschma@med.umich.edu.
² University of Michigan, Ann Arbor, MI 48109, USA.
³ Clinica Universidad de Navarra, Navarra, Spain.
⁴ Baylor College of Medicine, Houston, TX 77030, USA.
⁵ Dana-Faber Cancer Institute, Boston, MA 02215, USA.
⁶ Stanford University, Stanford, CA 94305, USA.
⁷ Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
⁸ Daegu Gyeongbuk Institute of Science & Technology, Daegu, South Korea.
⁹ University of Newcastle, Callaghan, NSW 2308, Australia.
¹⁰ Hospital for Sick Children, Toronto, ON M5G 1E8, Canada.
¹¹ Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
¹² Princess Maxima Center, 3584 CS Utrecht, the Netherlands.
¹³ Division of Molecular Pathology, Institute for Cancer Research, London SM2 5NG, UK.
¹⁴ Children's National, Washington, DC 20010, USA.
¹⁵ John Hopkins University, Baltimore, MD 21205, USA.
¹⁶ University of California, San Francisco, San Francisco, CA 94143, USA.
¹⁷ Cincinnati Children's Hospital Medical Center, and University of Cincinnati, Cincinnati, OH 45229, USA.
¹⁸ St. Jude, Memphis, TN 38105, USA.
¹⁹ University of California, San Francisco, San Francisco, CA 94143, USA; Parker Institute for Cancer Immunotherapy, University of Zurich, Zurich, Switzerland.
²⁰ Children's National, Washington, DC 20010, USA; University of Zurich, Zurich, Switzerland.
²¹ University of Michigan, Ann Arbor, MI 48109, USA; National Institutes of Health, Bethesda, MD, USA.
²² Duke Univesity, Durham, NC 27708, USA.
²³ Advocate Children's Hospital, Park Ridge, IL 60068, USA.
²⁴ Weill Cornell Medicine, New York Presbyterian and Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA.
²⁵ Herbert Irving Comprehensive Cancer Center and Department of Neurology, Columbia University Medical Center, New York, NY 10032, USA.
²⁶ University of Colorado, Denver, CO 80204, USA.
²⁷ School of Biomedical Sciences, Faculty of Medicine and Health, University of New South Wales, Sydney, NSW, Australia.
²⁸ Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA 92037, USA.
²⁹ Children's Mercy, Kansas City, MO 64108, USA.
³⁰ University of Michigan, Ann Arbor, MI 48109, USA. Electronic address: svenneti@med.umich.edu.

PMID: 38039965
PMCID: PMC11067690
DOI: 10.1016/j.ccell.2023.11.002

Abstract

Recent clinical trials for H3K27-altered diffuse midline gliomas (DMGs) have shown much promise. We present a consensus roadmap and identify three major barriers: (1) refinement of experimental models to include immune and brain-specific components; (2) collaboration among researchers, clinicians, and industry to integrate patient-derived data through sharing, transparency, and regulatory considerations; and (3) streamlining clinical efforts including biopsy, CNS-drug delivery, endpoint determination, and response monitoring. We highlight the importance of comprehensive collaboration to advance the understanding, diagnostics, and therapeutics for DMGs.

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Conflict of interest statement

Declaration of interests The authors declare no competing interests.

Figures

**Figure 1.. Overview of proposed assessment and banking of H3K27-altered diffuse midline gliomas.**
Diffuse Midline Gliomas arise mainly in children and young adults within midline structures including the thalamus, brainstem, cerebellum, and spinal cord. We propose a comprehensive longitudinal assessment/banking of (a) patient clinical parameters including imaging, therapy response, toxicity, and quality of life; (b) tumor tissues from both biopsy and autopsy, and (c) factors such as CSF, plasma, urine, and stools for biomarker development. We suggest an integrated analysis using multiple platforms for biomarker development, hypothesis generation, refinement of tumor response definitions, clinical trials, and generation of preclinical models. Figure constructed using BioRender.

See this image and copyright information in PMC

References

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A road map for the treatment of pediatric diffuse midline glioma

Affiliations

A road map for the treatment of pediatric diffuse midline glioma

Authors

Affiliations

Abstract

Conflict of interest statement

Figures

References

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MeSH terms

Grants and funding

LinkOut - more resources

Full Text Sources

Medical