Bone morphogenetic protein (BMP) signaling determines neuroblastoma cell fate and sensitivity to retinoic acid

Abstract Retinoic acid (RA) is a standard-of-care neuroblastoma drug thought to be effective by inducing differentiation. Curiously, RA has little effect on primary human tumors during upfront treatment but can eliminate neuroblastoma cells from the bone marrow during post-chemo maintenance therapy—...

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Main Authors: Min Pan, Yinwen Zhang, William C. Wright, Xueying Liu, Barbara Passaia, Duane Currier, Jonathan Low, Richard H. Chapple, Jacob A. Steele, Jon P. Connelly, Bensheng Ju, Emily Plyler, Meifen Lu, Allister J. Loughran, Lei Yang, Brian J. Abraham, Shondra M. Pruett-Miller, Burgess Freeman, George E. Campbell, Michael A. Dyer, Taosheng Chen, Elizabeth Stewart, Selene Koo, Heather Sheppard, John Easton, Paul Geeleher
Format: Article
Language:English
Published: Nature Portfolio 2025-02-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-57185-y
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author Min Pan
Yinwen Zhang
William C. Wright
Xueying Liu
Barbara Passaia
Duane Currier
Jonathan Low
Richard H. Chapple
Jacob A. Steele
Jon P. Connelly
Bensheng Ju
Emily Plyler
Meifen Lu
Allister J. Loughran
Lei Yang
Brian J. Abraham
Shondra M. Pruett-Miller
Burgess Freeman
George E. Campbell
Michael A. Dyer
Taosheng Chen
Elizabeth Stewart
Selene Koo
Heather Sheppard
John Easton
Paul Geeleher
author_facet Min Pan
Yinwen Zhang
William C. Wright
Xueying Liu
Barbara Passaia
Duane Currier
Jonathan Low
Richard H. Chapple
Jacob A. Steele
Jon P. Connelly
Bensheng Ju
Emily Plyler
Meifen Lu
Allister J. Loughran
Lei Yang
Brian J. Abraham
Shondra M. Pruett-Miller
Burgess Freeman
George E. Campbell
Michael A. Dyer
Taosheng Chen
Elizabeth Stewart
Selene Koo
Heather Sheppard
John Easton
Paul Geeleher
author_sort Min Pan
collection DOAJ
description Abstract Retinoic acid (RA) is a standard-of-care neuroblastoma drug thought to be effective by inducing differentiation. Curiously, RA has little effect on primary human tumors during upfront treatment but can eliminate neuroblastoma cells from the bone marrow during post-chemo maintenance therapy—a discrepancy that has never been explained. To investigate this, we treat a large cohort of neuroblastoma cell lines with RA and observe that the most RA-sensitive cells predominantly undergo apoptosis or senescence, rather than differentiation. We conduct genome-wide CRISPR knockout screens under RA treatment, which identify bone morphogenic protein (BMP) signaling as controlling the apoptosis/senescence vs differentiation cell fate decision and determining RA’s overall potency. We then discover that BMP signaling activity is markedly higher in neuroblastoma patient samples at bone marrow metastatic sites, providing a plausible explanation for RA’s ability to clear neuroblastoma cells specifically from the bone marrow, by seemingly mimicking interactions between BMP and RA during normal development.
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spelling doaj-art-ad29072542b34df48abed66ff9de0d492025-08-20T03:03:58ZengNature PortfolioNature Communications2041-17232025-02-0116112010.1038/s41467-025-57185-yBone morphogenetic protein (BMP) signaling determines neuroblastoma cell fate and sensitivity to retinoic acidMin Pan0Yinwen Zhang1William C. Wright2Xueying Liu3Barbara Passaia4Duane Currier5Jonathan Low6Richard H. Chapple7Jacob A. Steele8Jon P. Connelly9Bensheng Ju10Emily Plyler11Meifen Lu12Allister J. Loughran13Lei Yang14Brian J. Abraham15Shondra M. Pruett-Miller16Burgess Freeman17George E. Campbell18Michael A. Dyer19Taosheng Chen20Elizabeth Stewart21Selene Koo22Heather Sheppard23John Easton24Paul Geeleher25Department of Computational Biology, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalDepartment of Chemical Biology and Therapeutics, St. Jude Children’s Research HospitalDepartment of Chemical Biology and Therapeutics, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalDepartment of Cell and Molecular Biology, St. Jude Children’s Research HospitalDepartment of Cell and Molecular Biology, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalDepartment of Pathology, St. Jude Children’s Research HospitalDepartment of Cell and Molecular Biology, St. Jude Children’s Research HospitalDepartment of Chemical Biology and Therapeutics, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalDepartment of Cell and Molecular Biology, St. Jude Children’s Research HospitalPreclinical Pharmacokinetic Shared Resource, St. Jude Children’s Research HospitalCellular Imaging Shared Resource, St. Jude Children’s Research HospitalDepartment of Developmental Neurobiology, St. Jude Children’s Research HospitalDepartment of Chemical Biology and Therapeutics, St. Jude Children’s Research HospitalCellular Imaging Shared Resource, St. Jude Children’s Research HospitalDepartment of Pathology, St. Jude Children’s Research HospitalDepartment of Pathology, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalDepartment of Computational Biology, St. Jude Children’s Research HospitalAbstract Retinoic acid (RA) is a standard-of-care neuroblastoma drug thought to be effective by inducing differentiation. Curiously, RA has little effect on primary human tumors during upfront treatment but can eliminate neuroblastoma cells from the bone marrow during post-chemo maintenance therapy—a discrepancy that has never been explained. To investigate this, we treat a large cohort of neuroblastoma cell lines with RA and observe that the most RA-sensitive cells predominantly undergo apoptosis or senescence, rather than differentiation. We conduct genome-wide CRISPR knockout screens under RA treatment, which identify bone morphogenic protein (BMP) signaling as controlling the apoptosis/senescence vs differentiation cell fate decision and determining RA’s overall potency. We then discover that BMP signaling activity is markedly higher in neuroblastoma patient samples at bone marrow metastatic sites, providing a plausible explanation for RA’s ability to clear neuroblastoma cells specifically from the bone marrow, by seemingly mimicking interactions between BMP and RA during normal development.https://doi.org/10.1038/s41467-025-57185-y
spellingShingle Min Pan
Yinwen Zhang
William C. Wright
Xueying Liu
Barbara Passaia
Duane Currier
Jonathan Low
Richard H. Chapple
Jacob A. Steele
Jon P. Connelly
Bensheng Ju
Emily Plyler
Meifen Lu
Allister J. Loughran
Lei Yang
Brian J. Abraham
Shondra M. Pruett-Miller
Burgess Freeman
George E. Campbell
Michael A. Dyer
Taosheng Chen
Elizabeth Stewart
Selene Koo
Heather Sheppard
John Easton
Paul Geeleher
Bone morphogenetic protein (BMP) signaling determines neuroblastoma cell fate and sensitivity to retinoic acid
Nature Communications
title Bone morphogenetic protein (BMP) signaling determines neuroblastoma cell fate and sensitivity to retinoic acid
title_full Bone morphogenetic protein (BMP) signaling determines neuroblastoma cell fate and sensitivity to retinoic acid
title_fullStr Bone morphogenetic protein (BMP) signaling determines neuroblastoma cell fate and sensitivity to retinoic acid
title_full_unstemmed Bone morphogenetic protein (BMP) signaling determines neuroblastoma cell fate and sensitivity to retinoic acid
title_short Bone morphogenetic protein (BMP) signaling determines neuroblastoma cell fate and sensitivity to retinoic acid
title_sort bone morphogenetic protein bmp signaling determines neuroblastoma cell fate and sensitivity to retinoic acid
url https://doi.org/10.1038/s41467-025-57185-y
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