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1. NBAtlas: A harmonized single-cell transcriptomic reference atlas of human neuroblastoma tumors

Author

Bonine, Noah, Zanzani, Vittorio, Van Hemelryk, Annelies, Vanneste, Bavo, Zwicker, Christian, Thoné, Tinne, Roelandt, Sofie, Bekaert, Sarah-Lee, Koster, Jan, Janoueix-Lerosey, Isabelle, Thirant, Cécile, Van Haver, Stéphane, Roberts, Stephen S., Mus, Liselot M., De Wilde, Bram, Van Roy, Nadine, Everaert, Celine, Speleman, Frank, Vermeirssen, Vanessa, Scott, Charlotte L., and De Preter, Katleen

Published
2024
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2. SOX11 regulates SWI/SNF complex components as member of the adrenergic neuroblastoma core regulatory circuitry

Author

Decaesteker, Bieke, Louwagie, Amber, Loontiens, Siebe, De Vloed, Fanny, Bekaert, Sarah-Lee, Roels, Juliette, Vanhauwaert, Suzanne, De Brouwer, Sara, Sanders, Ellen, Berezovskaya, Alla, Denecker, Geertrui, D’haene, Eva, Van Haver, Stéphane, Van Loocke, Wouter, Van Dorpe, Jo, Creytens, David, Van Roy, Nadine, Pieters, Tim, Van Neste, Christophe, Fischer, Matthias, Van Vlierberghe, Pieter, Roberts, Stephen S., Schulte, Johannes, Ek, Sara, Versteeg, Rogier, Koster, Jan, van Nes, Johan, Zimmerman, Mark, De Preter, Katleen, and Speleman, Frank

Published
2023
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3. Clonal evolution during metastatic spread in high-risk neuroblastoma

Author

Gundem, Gunes, Levine, Max F., Roberts, Stephen S., Cheung, Irene Y., Medina-Martínez, Juan S., Feng, Yi, Arango-Ossa, Juan E., Chadoutaud, Loic, Rita, Mathieu, Asimomitis, Georgios, Zhou, Joe, You, Daoqi, Bouvier, Nancy, Spitzer, Barbara, Solit, David B., Dela Cruz, Filemon, LaQuaglia, Michael P., Kushner, Brian H., Modak, Shakeel, Shukla, Neerav, Iacobuzio-Donahue, Christine A., Kung, Andrew L., Cheung, Nai-Kong V., and Papaemmanuil, Elli

Published
2023
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8. Stage 4N neuroblastoma before and during the era of anti‐GD2 immunotherapy.

Author

Kushner, Brian H., LaQuaglia, Michael P., Cardenas, Fiorella Iglesias, Basu, Ellen M., Gerstle, Justin T., Kramer, Kim, Roberts, Stephen S., Wolden, Suzanne L., Cheung, Nai‐Kong V., and Modak, Shakeel

Subjects
NEUROBLASTOMA, IMMUNOTHERAPY, NATURAL history, MONOCLONAL antibodies, LYMPHATIC metastasis, BONE marrow
Abstract

Patients with stage 4N neuroblastoma (distant metastases limited to lymph nodes) stand out as virtually the only survivors of high‐risk neuroblastoma (HR‐NB) before myeloablative therapy (MAT) and immunotherapy with anti‐GD2 monoclonal antibodies (mAbs) became standard. Because no report presents more recent results with 4N, we analyzed our large 4N experience. All 51 pediatric 4N patients (<18 years old) diagnosed 1985 to 2021 were reviewed. HR‐NB included MYCN‐nonamplified 4N diagnosed at age ≥18 months and MYCN‐amplified 4N. Among 34 MYCN‐nonamplified high‐risk patients, 20 are relapse‐free 1.5+ to 37.5+ (median 12.5+) years post‐diagnosis, including 13 without prior MAT and 5 treated with little (1 cycle; n = 2) or no mAb (n = 3), while 14 patients (7 post‐MAT, 8 post‐mAbs) relapsed (all soft tissue). Of 15 MYCN‐amplified 4N patients, 7 are relapse‐free 2.1+ to 26.4+ (median 11.6+) years from the start of chemotherapy (all received mAbs; 3 underwent MAT) and 4 are in second remission 4.2+ to 21.8+ years postrelapse (all soft tissue). Statistical analyses showed no significant association of survival with either MAT or mAbs for MYCN‐nonamplified HR‐NB; small numbers prevented these analyses for MYCN‐amplified patients. The two patients with intermediate‐risk 4N (14‐months‐old) are relapse‐free 7+ years postresection of primary tumors; distant disease spontaneously regressed. The natural history of 4N is marked by NB confined to soft tissue without early relapse in bones or bone marrow, where mAbs have proven efficacy. These findings plus curability without MAT, as seen elsewhere and at our center, support consideration of treatment reduction for MYCN‐nonamplified 4N. [ABSTRACT FROM AUTHOR]

Published
2023
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9. Supplementary Table 1 from Patient-Driven Discovery, Therapeutic Targeting, and Post-Clinical Validation of a Novel AKT1 Fusion–Driven Cancer

Author

Slotkin, Emily K., primary, Diolaiti, Daniel, primary, Shukla, Neerav N., primary, Dela Cruz, Filemon S., primary, Clark, Jennifer J., primary, Gundem, Gunes, primary, Yellapantula, Venkata D., primary, Levine, Max F., primary, You, Daoqi, primary, Ma, Peilin, primary, Pachhal, Sagarika, primary, Ibanez Sanchez, Glorymar, primary, Benayed, Ryma, primary, Jungbluth, Achim A., primary, Smyth, Lillian M., primary, Mauguen, Audrey, primary, Gushterova, Irena, primary, Ding, Hongxu, primary, Spraggon, Lee, primary, Darnell, Robert, primary, Califano, Andrea, primary, Ladanyi, Marc, primary, Papaemmanuil, Elli, primary, Kung, Andrew L., primary, Hyman, David M., primary, and Roberts, Stephen S., primary

Published
2023
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10. Supplementary Data from Patient-Driven Discovery, Therapeutic Targeting, and Post-Clinical Validation of a Novel AKT1 Fusion–Driven Cancer

Author

Slotkin, Emily K., primary, Diolaiti, Daniel, primary, Shukla, Neerav N., primary, Dela Cruz, Filemon S., primary, Clark, Jennifer J., primary, Gundem, Gunes, primary, Yellapantula, Venkata D., primary, Levine, Max F., primary, You, Daoqi, primary, Ma, Peilin, primary, Pachhal, Sagarika, primary, Ibanez Sanchez, Glorymar, primary, Benayed, Ryma, primary, Jungbluth, Achim A., primary, Smyth, Lillian M., primary, Mauguen, Audrey, primary, Gushterova, Irena, primary, Ding, Hongxu, primary, Spraggon, Lee, primary, Darnell, Robert, primary, Califano, Andrea, primary, Ladanyi, Marc, primary, Papaemmanuil, Elli, primary, Kung, Andrew L., primary, Hyman, David M., primary, and Roberts, Stephen S., primary

Published
2023
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11. Data from Translational Strategies for Repotrectinib in Neuroblastoma

Author

O'Donohue, Tara J., primary, Ibáñez, Glorymar, primary, Coutinho, Diego Ferreira, primary, Mauguen, Audrey, primary, Siddiquee, Armaan, primary, Rosales, Nestor, primary, Calder, Paul, primary, Ndengu, Andoyo, primary, You, Daoqi, primary, Long, Matthew, primary, Roberts, Stephen S., primary, Kung, Andrew L., primary, and Dela Cruz, Filemon S., primary

Published
2023
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12. Supplementary Table 2 from Patient-Driven Discovery, Therapeutic Targeting, and Post-Clinical Validation of a Novel AKT1 Fusion–Driven Cancer

Author

Slotkin, Emily K., primary, Diolaiti, Daniel, primary, Shukla, Neerav N., primary, Dela Cruz, Filemon S., primary, Clark, Jennifer J., primary, Gundem, Gunes, primary, Yellapantula, Venkata D., primary, Levine, Max F., primary, You, Daoqi, primary, Ma, Peilin, primary, Pachhal, Sagarika, primary, Ibanez Sanchez, Glorymar, primary, Benayed, Ryma, primary, Jungbluth, Achim A., primary, Smyth, Lillian M., primary, Mauguen, Audrey, primary, Gushterova, Irena, primary, Ding, Hongxu, primary, Spraggon, Lee, primary, Darnell, Robert, primary, Califano, Andrea, primary, Ladanyi, Marc, primary, Papaemmanuil, Elli, primary, Kung, Andrew L., primary, Hyman, David M., primary, and Roberts, Stephen S., primary

Published
2023
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13. Data from Patient-Driven Discovery, Therapeutic Targeting, and Post-Clinical Validation of a Novel AKT1 Fusion–Driven Cancer

Author

Slotkin, Emily K., primary, Diolaiti, Daniel, primary, Shukla, Neerav N., primary, Dela Cruz, Filemon S., primary, Clark, Jennifer J., primary, Gundem, Gunes, primary, Yellapantula, Venkata D., primary, Levine, Max F., primary, You, Daoqi, primary, Ma, Peilin, primary, Pachhal, Sagarika, primary, Ibanez Sanchez, Glorymar, primary, Benayed, Ryma, primary, Jungbluth, Achim A., primary, Smyth, Lillian M., primary, Mauguen, Audrey, primary, Gushterova, Irena, primary, Ding, Hongxu, primary, Spraggon, Lee, primary, Darnell, Robert, primary, Califano, Andrea, primary, Ladanyi, Marc, primary, Papaemmanuil, Elli, primary, Kung, Andrew L., primary, Hyman, David M., primary, and Roberts, Stephen S., primary

Published
2023
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14. Supplementary Methods from Patient-Driven Discovery, Therapeutic Targeting, and Post-Clinical Validation of a Novel AKT1 Fusion–Driven Cancer

Author

Slotkin, Emily K., primary, Diolaiti, Daniel, primary, Shukla, Neerav N., primary, Dela Cruz, Filemon S., primary, Clark, Jennifer J., primary, Gundem, Gunes, primary, Yellapantula, Venkata D., primary, Levine, Max F., primary, You, Daoqi, primary, Ma, Peilin, primary, Pachhal, Sagarika, primary, Ibanez Sanchez, Glorymar, primary, Benayed, Ryma, primary, Jungbluth, Achim A., primary, Smyth, Lillian M., primary, Mauguen, Audrey, primary, Gushterova, Irena, primary, Ding, Hongxu, primary, Spraggon, Lee, primary, Darnell, Robert, primary, Califano, Andrea, primary, Ladanyi, Marc, primary, Papaemmanuil, Elli, primary, Kung, Andrew L., primary, Hyman, David M., primary, and Roberts, Stephen S., primary

Published
2023
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15. Supplementary Table 4 from Patient-Driven Discovery, Therapeutic Targeting, and Post-Clinical Validation of a Novel AKT1 Fusion–Driven Cancer

Author

Slotkin, Emily K., primary, Diolaiti, Daniel, primary, Shukla, Neerav N., primary, Dela Cruz, Filemon S., primary, Clark, Jennifer J., primary, Gundem, Gunes, primary, Yellapantula, Venkata D., primary, Levine, Max F., primary, You, Daoqi, primary, Ma, Peilin, primary, Pachhal, Sagarika, primary, Ibanez Sanchez, Glorymar, primary, Benayed, Ryma, primary, Jungbluth, Achim A., primary, Smyth, Lillian M., primary, Mauguen, Audrey, primary, Gushterova, Irena, primary, Ding, Hongxu, primary, Spraggon, Lee, primary, Darnell, Robert, primary, Califano, Andrea, primary, Ladanyi, Marc, primary, Papaemmanuil, Elli, primary, Kung, Andrew L., primary, Hyman, David M., primary, and Roberts, Stephen S., primary

Published
2023
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16. Supplementary Table 5 from Patient-Driven Discovery, Therapeutic Targeting, and Post-Clinical Validation of a Novel AKT1 Fusion–Driven Cancer

Author

Slotkin, Emily K., primary, Diolaiti, Daniel, primary, Shukla, Neerav N., primary, Dela Cruz, Filemon S., primary, Clark, Jennifer J., primary, Gundem, Gunes, primary, Yellapantula, Venkata D., primary, Levine, Max F., primary, You, Daoqi, primary, Ma, Peilin, primary, Pachhal, Sagarika, primary, Ibanez Sanchez, Glorymar, primary, Benayed, Ryma, primary, Jungbluth, Achim A., primary, Smyth, Lillian M., primary, Mauguen, Audrey, primary, Gushterova, Irena, primary, Ding, Hongxu, primary, Spraggon, Lee, primary, Darnell, Robert, primary, Califano, Andrea, primary, Ladanyi, Marc, primary, Papaemmanuil, Elli, primary, Kung, Andrew L., primary, Hyman, David M., primary, and Roberts, Stephen S., primary

Published
2023
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18. Supplementary Table 3 from Patient-Driven Discovery, Therapeutic Targeting, and Post-Clinical Validation of a Novel AKT1 Fusion–Driven Cancer

Author

Slotkin, Emily K., primary, Diolaiti, Daniel, primary, Shukla, Neerav N., primary, Dela Cruz, Filemon S., primary, Clark, Jennifer J., primary, Gundem, Gunes, primary, Yellapantula, Venkata D., primary, Levine, Max F., primary, You, Daoqi, primary, Ma, Peilin, primary, Pachhal, Sagarika, primary, Ibanez Sanchez, Glorymar, primary, Benayed, Ryma, primary, Jungbluth, Achim A., primary, Smyth, Lillian M., primary, Mauguen, Audrey, primary, Gushterova, Irena, primary, Ding, Hongxu, primary, Spraggon, Lee, primary, Darnell, Robert, primary, Califano, Andrea, primary, Ladanyi, Marc, primary, Papaemmanuil, Elli, primary, Kung, Andrew L., primary, Hyman, David M., primary, and Roberts, Stephen S., primary

Published
2023
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25. Clonal evolution during metastatic spread in high-risk neuroblastoma

Author

Gundem, Gunes, primary, Levine, Max F., additional, Roberts, Stephen S., additional, Cheung, Irene Y, additional, Medina-Martínez, Juan S., additional, Feng, Yi, additional, Arango-Ossa, Juan E., additional, Chadoutaud, Loic, additional, Rita, Mathieu, additional, Asimomitis, Georgios, additional, Zhou, Joe, additional, You, Daoqi, additional, Bouvier, Nancy, additional, Spitzer, Barbara, additional, Solit, David B., additional, Cruz Dela, Filemon, additional, LaQuaglia, Michael P., additional, Kushner, Brian H., additional, Modak, Shakeel, additional, Shukla, Neerav, additional, Iacobuzio-Donahue, Christine A., additional, Kung, Andrew L., additional, Cheung, Nai-Kong V., additional, and Papaemmanuil, Elli, additional

Published
2022
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26. RRM2 enhances MYCN-driven neuroblastoma formation and acts as a synergistic target with CHK1 inhibition

Author

Nunes, Carolina, primary, Depestel, Lisa, additional, Mus, Liselot, additional, Keller, Kaylee M., additional, Delhaye, Louis, additional, Louwagie, Amber, additional, Rishfi, Muhammad, additional, Whale, Alex, additional, Kara, Neesha, additional, Andrews, Simon R., additional, Dela Cruz, Filemon, additional, You, Daoqi, additional, Siddiquee, Armaan, additional, Cologna, Camila Takeno, additional, De Craemer, Sam, additional, Dolman, Emmy, additional, Bartenhagen, Christoph, additional, De Vloed, Fanny, additional, Sanders, Ellen, additional, Eggermont, Aline, additional, Bekaert, Sarah-Lee, additional, Van Loocke, Wouter, additional, Bek, Jan Willem, additional, Dewyn, Givani, additional, Loontiens, Siebe, additional, Van Isterdael, Gert, additional, Decaesteker, Bieke, additional, Tilleman, Laurentijn, additional, Van Nieuwerburgh, Filip, additional, Vermeirssen, Vanessa, additional, Van Neste, Christophe, additional, Ghesquiere, Bart, additional, Goossens, Steven, additional, Eyckerman, Sven, additional, De Preter, Katleen, additional, Fischer, Matthias, additional, Houseley, Jon, additional, Molenaar, Jan, additional, De Wilde, Bram, additional, Roberts, Stephen S., additional, Durinck, Kaat, additional, and Speleman, Frank, additional

Published
2022
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29. RRM2 enhances MYCN-driven neuroblastoma formation and acts as a synergistic target with CHK1 inhibition

Author

Nunes, Carolina, Depestel, Lisa, Mus, Liselot, Keller, Kaylee M., Delhaye, Louis, Louwagie, Amber, Rishfi, Muhammad, Whale, Alex, Kara, Neesha, Andrews, Simon R., Cruz, Filemon Dela, You, Daoqi, Siddiquee, Armaan, Cologna, Camila Takeno, De Craemer, Sam, Dolman, Emmy, Bartenhagen, Christoph, De Vloed, Fanny, Sanders, Ellen, Eggermont, Aline, Bekaert, Sarah Lee, Van Loocke, Wouter, Bek, Jan Willem, Dewyn, Givani, Loontiens, Siebe, Van Isterdael, Gert, Decaesteker, Bieke, Tilleman, Laurentijn, Van Nieuwerburgh, Filip, Vermeirssen, Vanessa, Van Neste, Christophe, Ghesquiere, Bart, Goossens, Steven, Eyckerman, Sven, De Preter, Katleen, Fischer, Matthias, Houseley, Jon, Molenaar, Jan, De Wilde, Bram, Roberts, Stephen S., Durinck, Kaat, Speleman, Frank, Nunes, Carolina, Depestel, Lisa, Mus, Liselot, Keller, Kaylee M., Delhaye, Louis, Louwagie, Amber, Rishfi, Muhammad, Whale, Alex, Kara, Neesha, Andrews, Simon R., Cruz, Filemon Dela, You, Daoqi, Siddiquee, Armaan, Cologna, Camila Takeno, De Craemer, Sam, Dolman, Emmy, Bartenhagen, Christoph, De Vloed, Fanny, Sanders, Ellen, Eggermont, Aline, Bekaert, Sarah Lee, Van Loocke, Wouter, Bek, Jan Willem, Dewyn, Givani, Loontiens, Siebe, Van Isterdael, Gert, Decaesteker, Bieke, Tilleman, Laurentijn, Van Nieuwerburgh, Filip, Vermeirssen, Vanessa, Van Neste, Christophe, Ghesquiere, Bart, Goossens, Steven, Eyckerman, Sven, De Preter, Katleen, Fischer, Matthias, Houseley, Jon, Molenaar, Jan, De Wilde, Bram, Roberts, Stephen S., Durinck, Kaat, and Speleman, Frank

Abstract

High-risk neuroblastoma, a pediatric tumor originating from the sympathetic nervous system, has a low mutation load but highly recurrent somatic DNA copy number variants. Previously, segmental gains and/or amplifications allowed identification of drivers for neuroblastoma development. Using this approach, combined with gene dosage impact on expression and survival, we identified ribonucleotide reductase subunit M2 (RRM2) as a candidate dependency factor further supported by growth inhibition upon in vitro knockdown and accelerated tumor formation in a neuroblastoma zebrafish model coexpressing human RRM2 with MYCN. Forced RRM2 induction alleviates excessive replicative stress induced by CHK1 inhibition, while high RRM2 expression in human neuroblastomas correlates with high CHK1 activity. MYCN-driven zebrafish tumors with RRM2 co-overexpression exhibit differentially expressed DNA repair genes in keeping with enhanced ATR-CHK1 signaling activity. In vitro, RRM2 inhibition enhances intrinsic replication stress checkpoint addiction. Last, combinatorial RRM2-CHK1 inhibition acts synergistic in high-risk neuroblastoma cell lines and patient-derived xenograft models, illustrating the therapeutic potential.

Published
2022

30. RRM2 enhances MYCN-driven neuroblastoma formation and acts as a synergistic target with CHK1 inhibition

Author

Afd Pharmaceutics, Pharmaceutics, Nunes, Carolina, Depestel, Lisa, Mus, Liselot, Keller, Kaylee M., Delhaye, Louis, Louwagie, Amber, Rishfi, Muhammad, Whale, Alex, Kara, Neesha, Andrews, Simon R., Cruz, Filemon Dela, You, Daoqi, Siddiquee, Armaan, Cologna, Camila Takeno, De Craemer, Sam, Dolman, Emmy, Bartenhagen, Christoph, De Vloed, Fanny, Sanders, Ellen, Eggermont, Aline, Bekaert, Sarah Lee, Van Loocke, Wouter, Bek, Jan Willem, Dewyn, Givani, Loontiens, Siebe, Van Isterdael, Gert, Decaesteker, Bieke, Tilleman, Laurentijn, Van Nieuwerburgh, Filip, Vermeirssen, Vanessa, Van Neste, Christophe, Ghesquiere, Bart, Goossens, Steven, Eyckerman, Sven, De Preter, Katleen, Fischer, Matthias, Houseley, Jon, Molenaar, Jan, De Wilde, Bram, Roberts, Stephen S., Durinck, Kaat, Speleman, Frank, Afd Pharmaceutics, Pharmaceutics, Nunes, Carolina, Depestel, Lisa, Mus, Liselot, Keller, Kaylee M., Delhaye, Louis, Louwagie, Amber, Rishfi, Muhammad, Whale, Alex, Kara, Neesha, Andrews, Simon R., Cruz, Filemon Dela, You, Daoqi, Siddiquee, Armaan, Cologna, Camila Takeno, De Craemer, Sam, Dolman, Emmy, Bartenhagen, Christoph, De Vloed, Fanny, Sanders, Ellen, Eggermont, Aline, Bekaert, Sarah Lee, Van Loocke, Wouter, Bek, Jan Willem, Dewyn, Givani, Loontiens, Siebe, Van Isterdael, Gert, Decaesteker, Bieke, Tilleman, Laurentijn, Van Nieuwerburgh, Filip, Vermeirssen, Vanessa, Van Neste, Christophe, Ghesquiere, Bart, Goossens, Steven, Eyckerman, Sven, De Preter, Katleen, Fischer, Matthias, Houseley, Jon, Molenaar, Jan, De Wilde, Bram, Roberts, Stephen S., Durinck, Kaat, and Speleman, Frank

Published
2022

33. Contributors

Author

Abrams, Ross A., primary, Adelstein, David J., additional, Alektiar, Kaled M., additional, Alexander, Brian, additional, Alsner, Jan, additional, Altun, Ersan, additional, Anderson, Bethany, additional, Ang, K. Kian, additional, Arthur, Douglas W., additional, Ashman, Jonathan B., additional, Ballo, Matthew T., additional, Barker, Christopher Andrew, additional, Beadle, Beth M., additional, Bedard, Phillipe, additional, Beitler, Jonathan J., additional, Bishop, Michael W., additional, Blackstock, A. William, additional, Bogart, Jeffrey A., additional, Bonner, James A., additional, Bourland, J. Daniel, additional, Bovi, Joseph, additional, Breneman, John, additional, Brito, Juan P., additional, Brown, Paul D., additional, Brundage, Michael D., additional, Buchholz, Thomas A., additional, Burmeister, Bryan Henry, additional, Calderwood, Stuart K., additional, Callister, Matthew D., additional, Calvo, Felipe A., additional, Cannon, George M., additional, Chabner, Bruce A., additional, Chan, Michael D., additional, Chao, Sam T., additional, Charpentier, Anne-Marie, additional, Chung, Christine H., additional, Chung, Peter W.M., additional, Constine, Louis S., additional, Corn, Benjamin W., additional, Covens, Allan, additional, Craciunescu, Oana I., additional, Crane, Christopher H., additional, Creutzberg, Carien L., additional, Crook, Juanita M., additional, Curran, Walter J., additional, Czito, Brian G., additional, Dabaja, Bouthaina S., additional, Das, Shiva, additional, David, Marc, additional, Dawson, Laura A., additional, DeLaney, Thomas F., additional, Devlin, Phillip M., additional, Dewhirst, Mark, additional, Dizon, Don S., additional, Dome, Jeffrey S., additional, Donohue, John H., additional, Duprez, Thierry P., additional, Efstathiou, Jason A., additional, Eisbruch, Avraham, additional, Eisele, David W., additional, Feng, Mary, additional, Fernandes, Rui P., additional, Fielding, Julia R., additional, Fleming, Gini F., additional, Foote, Robert L., additional, Fraass, Benedick A., additional, Freeman, Carolyn R., additional, Garden, Adam S., additional, Gentry, Lindell R., additional, Gien, Lilian T., additional, Gospodarowicz, Mary K., additional, Grau, Cai, additional, Grégoire, Vincent, additional, Greven, Craig M., additional, Greven, Kathryn McConnell, additional, Gunderson, Leonard L., additional, Haddock, Michael G., additional, Halyard, Michele, additional, Hamoir, Marc, additional, Hanna, Timothy Paul, additional, Harari, Paul M., additional, Hay, Ian D., additional, Herman, Joseph M., additional, Holloway, Caroline L., additional, Hong, Theodore Sunki, additional, Horowitz, Neil S., additional, Horseman, Michael R., additional, Howle, Julie, additional, Hrycushko, Brian A., additional, Hsu, David, additional, Hudgins, Patricia A., additional, Hutchinson, Ryan C., additional, Iacobuzio-Donahue, Christine, additional, Izar, Benjamin, additional, Jewells, Valerie L., additional, Jurcic, Joseph Gerard, additional, Kalapurakal, John A., additional, Kavanagh, Brian D., additional, Kelly, Kara M., additional, Khandani, Amir H., additional, Khuntia, Deepak, additional, Kiess, Ana Ponce, additional, Knox, Susan J., additional, Koh, Wui-Jin, additional, Krasin, Matthew J., additional, Kun, Larry E., additional, Laack, Nadia Issa, additional, LaCasce, Ann S., additional, Langer, Corey Jay, additional, Laramore, George E., additional, Lassman, Andrew B., additional, Lawton, Colleen A.F., additional, Lee, Nancy, additional, Lengelé, Benoît, additional, Levin, William P., additional, Lindegaard, Jacob C., additional, Lucas, John T., additional, MacDonald, Shannon M., additional, Mackillop, William J., additional, Mahindra, Anuj, additional, Mancuso, Anthony A., additional, Marcus, Karen Jean, additional, Marks, Lawrence B., additional, Matceyevsky, Diana, additional, Mazeron, Jean-Jacques, additional, McDonald, Mark W., additional, Medin, Paul M., additional, Mehta, Minesh P., additional, Mendenhall, William M., additional, Meredith, Ruby F., additional, Michalski, Jeff M., additional, Milano, Michael T., additional, Minsky, Bruce D., additional, Morrison, William H., additional, Murphy, Erin S., additional, Murthy, Rashmi K., additional, Ng, Andrea K., additional, Nordsmark, Marianne, additional, O'Farrell, Desmond A., additional, Okunieff, Paul, additional, Ove, Roger, additional, Overgaard, Jens, additional, Palta, Manisha, additional, Parker, Alexander S., additional, Pater, Luke E., additional, Peterson, Jennifer L., additional, Pisansky, Thomas M., additional, Potters, Louis, additional, Quon, Harry, additional, Raben, David, additional, Recht, Abram, additional, Rengan, Ramesh, additional, Reyngold, Marsha, Laufer, additional, Riaz, Nadeem, additional, Roberts, Stephen S., additional, Roberts, Kenneth B., additional, Rockhill, Jason K., additional, Rödel, Claus M., additional, Rodriguez-Galindo, Carlos, additional, Rogers, C. Leland, additional, Rosenblat, Todd L., additional, Rule, William G., additional, Russell, Anthony Henryk, additional, Russo, Suzanne, additional, Ryan, David P., additional, Sandlund, John Torsten, additional, Sandow, Pamela L., additional, Sargent, Daniel J., additional, Schild, Steven E., additional, Seegenschmiedt, Michael Heinrich, additional, Shah, Chirag, additional, Shaw, Edward G., additional, Sheehan, Jason P., additional, Sheikh, Arif, additional, Shi, Qian, additional, Siker, Malika L., additional, Small, William, additional, Smith, Benjamin D., additional, Smith, Grace L., additional, Solberg, Timothy D., additional, Stauffer, Paul R., additional, Stevenson, Mary Ann, additional, Stewart, Alexandra J., additional, Suh, John H., additional, Tan, Winston W., additional, Tepper, Joel E., additional, Thomas, Charles R., additional, Thomas, Gillian M., additional, Timmerman, Robert D., additional, Tsang, Richard W., additional, Usuki, Kenneth Y., additional, Valentini, Vincenzo, additional, Valero, Vicente, additional, van den Bent, Martin J., additional, Veness, Michael J., additional, Vicini, Frank A., additional, Vicus, Danielle, additional, Viswanathan, Akila N., additional, Vujaskovic, Zeljko, additional, Wadsworth, J. Trad, additional, Wagner, Henry, additional, Wahl, Daniel R., additional, Warde, Padraig R., additional, Waxweiler, Timothy V., additional, Wehle, Michael J., additional, Weil, Robert J., additional, Weiss, Lawrence M., additional, Werning, John W., additional, Willett, Christopher G., additional, Willey, Christopher Douglas, additional, Wilson, Lynn D., additional, Winkfield, Karen M., additional, Yon-Li, Jennifer, additional, Wolden, Suzanne L., additional, Wong, Terence Z., additional, Wong, Jeffrey Y.C., additional, Wong, William W., additional, Wu, Wenting, additional, Yahalom, Joachim, additional, Yang, Eddy Shih-Hsin, additional, You, Y. Nancy, additional, Zeman, Elaine M., additional, Zeng, Jing, additional, and Zietman, Anthony L., additional

Published
2016
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35. Delineation of Two Clinically and Molecularly Distinct Subgroups of Posterior Fossa Ependymoma

Author

Witt, Hendrik, Mack, Stephen C., Ryzhova, Marina, Bender, Sebastian, Sill, Martin, Isserlin, Ruth, Benner, Axel, Hielscher, Thomas, Milde, Till, Remke, Marc, Jones, David T.W., Northcott, Paul A., Garzia, Livia, Bertrand, Kelsey C., Wittmann, Andrea, Yao, Yuan, Roberts, Stephen S., Massimi, Luca, Van Meter, Tim, Weiss, William A., Gupta, Nalin, Grajkowska, Wiesia, Lach, Boleslaw, Cho, Yoon-Jae, von Deimling, Andreas, Kulozik, Andreas E., Witt, Olaf, Bader, Gary D., Hawkins, Cynthia E., Tabori, Uri, Guha, Abhijit, Rutka, James T., Lichter, Peter, Korshunov, Andrey, Taylor, Michael D., and Pfister, Stefan M.

Published
2011
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37. Immunotherapy with anti‐GD2 monoclonal antibody in infants with high‐risk neuroblastoma.

Author

Kushner, Brian H., Modak, Shakeel, Kramer, Kim, Basu, Ellen M., Iglesias‐Cardenas, Fiorella, Roberts, Stephen S., and Cheung, Nai‐Kong V.

Subjects
NEUROBLASTOMA, MONOCLONAL antibodies, POSTERIOR leukoencephalopathy syndrome, INFANTS, OLDER patients, IMMUNOTHERAPY
Abstract

Anti‐GD2 monoclonal antibodies (mAb) improve the prognosis of high‐risk neuroblastoma (HR‐NB). Worldwide experience almost exclusively involves toddlers and older patients treated after multimodality or second‐line therapies, that is, many months postdiagnosis. In contrast, at our center, infants received anti‐GD2 mAbs because this immunotherapy started during or immediately after induction chemotherapy. We now report on the feasibility, safety, and long‐term survival in this vulnerable age group. Thirty‐three HR‐NB patients were <19 months old when started on 3F8 (murine mAb; n = 21) or naxitamab (humanized‐3F8; n = 12), with 30″ to 90″ intravenous infusions. Patients received analgesics and antihistamines. Common toxicities (pain, urticaria, cough) were manageable, allowing outpatient treatment. Capillary leak, posterior reversible encephalopathy syndrome, and mAb‐related long‐term toxicities did not occur. Two 3F8 cycles were aborted due to bradycardia (a preexisting condition) and asthmatic symptoms, respectively. One patient received ½ dose of Day 1 naxitamab because of hypotension; full doses were subsequently administered. Post‐mAb treatments included chemotherapy, radiotherapy, and anti‐NB vaccine. Among 3F8 patients, 17/21 are in complete remission off all treatment at 5.6+ to 24.1+ (median 13.4+) years from diagnosis. Among naxitamab patients, 10/12 remain relapse‐free post‐mAb at 1.7+ to 4.3+ (median 3.1+) years from diagnosis. Toxicity was similar with short outpatient infusions and matched that observed with these and other anti‐GD2 mAbs in older patients. These findings were reassuring given that naxitamab is dosed >2.5× higher (~270 mg/m2/cycle) than 3F8, dinutuximab, and dinutuximab beta (70‐100 mg/m2/cycle). HR‐NB in infants proved to be highly curable. [ABSTRACT FROM AUTHOR]

Published
2023
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38. Translational Strategies for Repotrectinib in Neuroblastoma

Author

O'Donohue, Tara J., primary, Ibáñez, Glorymar, additional, Coutinho, Diego Ferreira, additional, Mauguen, Audrey, additional, Siddiquee, Armaan, additional, Rosales, Nestor, additional, Calder, Paul, additional, Ndengu, Andoyo, additional, You, Daoqi, additional, Long, Matthew, additional, Roberts, Stephen S., additional, Kung, Andrew L., additional, and Dela Cruz, Filemon S., additional

Published
2021
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45. Stepwise Strategic Mitigation Planning in a Pediatric Oncology Center During the COVID-19 Pandemic

Author

Szenes, Victoria, primary, Bright, Rachel, additional, Diotallevi, Deborah, additional, Melendez, Giselle, additional, Martinez, Cassie, additional, Zakak, Nicole, additional, Killinger, James, additional, Gilheeney, Stephen, additional, Roberts, Stephen S., additional, Kamboj, Mini, additional, Bender, Julia Glade, additional, Kung, Andrew L., additional, and Boulad, Farid, additional

Published
2021
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46. Extracellular Vesicle and Particle Biomarkers Define Multiple Human Cancers

Author

Hoshino, Ayuko, Kim, Han Sang, Bojmar, Linda, Gyan, Kofi Ennu, Cioffi, Michele, Hernandez, Jonathan, Zambirinis, Constantinos P., Rodrigues, Goncalo, Molina, Henrik, Heissel, Sren, Mark, Milica Tesic, Steiner, Loic, Benito-Martin, Alberto, Lucotti, Serena, Di Giannatale, Angela, Offer, Katharine, Nakajima, Miho, Williams, Caitlin, Nogues, Laura, Vatter, Fanny A. Pelissier, Hashimoto, Ayako, Davies, Alexander E., Freitas, Daniela, Kenific, Candia M., Ararso, Yonathan, Buehring, Weston, Lauritzen, Pernille, Ogitani, Yusuke, Sugiura, Kei, Takahashi, Naoko, Aleckovic, Masa, Bailey, Kayleen A., Jolissant, Joshua S., Wang, Huajuan, Harris, Ashton, Schaeffer, L. Miles, Garcia-Santos, Guillermo, Posner, Zoe, Balachandran, Vinod P., Khakoo, Yasmin, Raju, G. Praveen, Scherz, Avigdor, Sagi, Irit, Scherz-Shouval, Ruth, Yarden, Yosef, Oren, Moshe, Malladi, Mahathi, Petriccione, Mary, De Braganca, Kevin C., Donzelli, Maria, Fischer, Cheryl, Vitolano, Stephanie, Wright, Geraldine P., Ganshaw, Lee, Marrano, Mariel, Ahmed, Amina, DeStefano, Joe, Danzer, Enrico, Roehrl, Michael H. A., Lacayo, Norman J., Vincent, C. Theresa, Weiser, Martin R., Brady, Mary S., Meyers, Paul A., Wexler, Leonard H., Ambati, Srikanth R., Chou, Alexander J., Slotkin, Emily K., Modak, Shakeel, Roberts, Stephen S., Basu, Ellen M., Diolaiti, Daniel, Krantz, Benjamin A., Cardoso, Fatima, Simpson, Amber L., Berger, Michael, Rudin, Charles M., Simeone, Diane M., Jain, Maneesh, Ghajar, Cyrus M., Batra, Surinder K., Stanger, Ben Z., Bui, Jack, Brown, Kristy A., Rajasekhar, Vinagolu K., Healey, John H., de Sousa, Maria, Kramer, Kim, Sheth, Sujit, Baisch, Jeanine, Pascual, Virginia, Heaton, Todd E., La Quaglia, Michael P., Pisapia, David J., Schwartz, Robert, Zhang, Haiying, Liu, Yuan, Shukla, Arti, Blavier, Laurence, DeClerck, Yves A., LaBarge, Mark, Bissell, Mina J., Caffrey, Thomas C., Grandgenett, Paul M., Hollingsworth, Michael A., Bromberg, Jacqueline, Costa-Silva, Bruno, Peinado, Hector, Kang, Yibin, Garcia, Benjamin A., O'Reilly, Eileen M., Kelsen, David, Trippett, Tanya M., Jones, David R., Matei, Irina R., Jarnagin, William R., Lyden, David, Hoshino, Ayuko, Kim, Han Sang, Bojmar, Linda, Gyan, Kofi Ennu, Cioffi, Michele, Hernandez, Jonathan, Zambirinis, Constantinos P., Rodrigues, Goncalo, Molina, Henrik, Heissel, Sren, Mark, Milica Tesic, Steiner, Loic, Benito-Martin, Alberto, Lucotti, Serena, Di Giannatale, Angela, Offer, Katharine, Nakajima, Miho, Williams, Caitlin, Nogues, Laura, Vatter, Fanny A. Pelissier, Hashimoto, Ayako, Davies, Alexander E., Freitas, Daniela, Kenific, Candia M., Ararso, Yonathan, Buehring, Weston, Lauritzen, Pernille, Ogitani, Yusuke, Sugiura, Kei, Takahashi, Naoko, Aleckovic, Masa, Bailey, Kayleen A., Jolissant, Joshua S., Wang, Huajuan, Harris, Ashton, Schaeffer, L. Miles, Garcia-Santos, Guillermo, Posner, Zoe, Balachandran, Vinod P., Khakoo, Yasmin, Raju, G. Praveen, Scherz, Avigdor, Sagi, Irit, Scherz-Shouval, Ruth, Yarden, Yosef, Oren, Moshe, Malladi, Mahathi, Petriccione, Mary, De Braganca, Kevin C., Donzelli, Maria, Fischer, Cheryl, Vitolano, Stephanie, Wright, Geraldine P., Ganshaw, Lee, Marrano, Mariel, Ahmed, Amina, DeStefano, Joe, Danzer, Enrico, Roehrl, Michael H. A., Lacayo, Norman J., Vincent, C. Theresa, Weiser, Martin R., Brady, Mary S., Meyers, Paul A., Wexler, Leonard H., Ambati, Srikanth R., Chou, Alexander J., Slotkin, Emily K., Modak, Shakeel, Roberts, Stephen S., Basu, Ellen M., Diolaiti, Daniel, Krantz, Benjamin A., Cardoso, Fatima, Simpson, Amber L., Berger, Michael, Rudin, Charles M., Simeone, Diane M., Jain, Maneesh, Ghajar, Cyrus M., Batra, Surinder K., Stanger, Ben Z., Bui, Jack, Brown, Kristy A., Rajasekhar, Vinagolu K., Healey, John H., de Sousa, Maria, Kramer, Kim, Sheth, Sujit, Baisch, Jeanine, Pascual, Virginia, Heaton, Todd E., La Quaglia, Michael P., Pisapia, David J., Schwartz, Robert, Zhang, Haiying, Liu, Yuan, Shukla, Arti, Blavier, Laurence, DeClerck, Yves A., LaBarge, Mark, Bissell, Mina J., Caffrey, Thomas C., Grandgenett, Paul M., Hollingsworth, Michael A., Bromberg, Jacqueline, Costa-Silva, Bruno, Peinado, Hector, Kang, Yibin, Garcia, Benjamin A., O'Reilly, Eileen M., Kelsen, David, Trippett, Tanya M., Jones, David R., Matei, Irina R., Jarnagin, William R., and Lyden, David

Abstract

There is an unmet clinical need for improved tissue and liquid biopsy tools for cancer detection. We investigated the proteomic profile of extracellular vesicles and particles (EVPs) in 426 human samples from tissue explants (TEs), plasma, and other bodily fluids. Among traditional exosome markers, CD9, HSPA8, ALIX, and HSP90AB1 represent pan-EVP markers, while ACTB, MSN, and RAP1B are novel pan-EVP markers. To confirm that EVPs are ideal diagnostic tools, we analyzed proteomes of TE- (n =151) and plasma-derived (n =120) EVPs. Comparison of TE EVPs identified proteins (e.g., VCAN, TNC, and THBS2) that distinguish tumors from normal tissues with 90% sensitivity/94% specificity. Machine-learning classification of plasma-derived EVP cargo, including immunoglobulins, revealed 95% sensitivity/90% specificity in detecting cancer Finally, we defined a panel of tumor-type-specific EVP proteins in TEs and plasma, which can classify tumors of unknown primary origin. Thus, EVP proteins can serve as reliable biomarkers for cancer detection and determining cancer type.

Published
2020
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50. SOX11 is a lineage-dependency factor and master epigenetic regulator in neuroblastoma

Author

Decaesteker, Bieke, primary, Louwagie, Amber, additional, Loontiens, Siebe, additional, De Vloed, Fanny, additional, Roels, Juliette, additional, Vanhauwaert, Suzanne, additional, De Brouwer, Sara, additional, Sanders, Ellen, additional, Denecker, Geertrui, additional, D’haene, Eva, additional, Van Haver, Stéphane, additional, Van Loocke, Wouter, additional, Van Dorpe, Jo, additional, Creytens, David, additional, Van Roy, Nadine, additional, Pieters, Tim, additional, Van Neste, Christophe, additional, Fischer, Matthias, additional, Van Vlierberghe, Pieter, additional, Roberts, Stephen S, additional, Schulte, Johannes, additional, Ek, Sara, additional, Versteeg, Rogier, additional, Koster, Jan, additional, van Nes, Johan, additional, De Preter, Katleen, additional, and Speleman, Frank, additional

Published
2020
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