Insights into pediatric rhabdomyosarcoma research: Challenges and goals

Marielle E. Yohe; Christine M. Heske; Elizabeth Stewart; Peter C. Adamson; Nabil Ahmed; Cristina R. Antonescu; Eleanor Chen; Natalie Collins; Alan Ehrlich; Rene L. Galindo; Berkley E. Gryder; Heidi Hahn; Sharon Hammond; Mark E. Hatley; Douglas S. Hawkins; Madeline N. Hayes; Andrea Hayes-Jordan; Lee J. Helman; Simone Hettmer; Myron S. Ignatius; Charles Keller; Javed Khan; David G. Kirsch; Corinne M. Linardic; Philip J. Lupo; Rossella Rota; Jack F. Shern; Janet Shipley; Sivasish Sindiri; Stephen J. Tapscott; Christopher R. Vakoc; Leonard H. Wexler; David M. Langenau

doi:10.1002/pbc.27869

Insights into pediatric rhabdomyosarcoma research: Challenges and goals

Marielle E. Yohe, Christine M. Heske, Elizabeth Stewart, Peter C. Adamson, Nabil Ahmed, Cristina R. Antonescu, Eleanor Chen, Natalie Collins, Alan Ehrlich, Rene L. Galindo, Berkley E. Gryder, Heidi Hahn, Sharon Hammond, Mark E. Hatley, Douglas S. Hawkins, Madeline N. Hayes, Andrea Hayes-Jordan, Lee J. Helman, Simone Hettmer, Myron S. IgnatiusCharles Keller, Javed Khan, David G. Kirsch, Corinne M. Linardic, Philip J. Lupo, Rossella Rota, Jack F. Shern, Janet Shipley, Sivasish Sindiri, Stephen J. Tapscott, Christopher R. Vakoc, Leonard H. Wexler, David M. Langenau

Research output: Contribution to journal › Review article › peer-review

53 Scopus citations

Abstract

Overall survival rates for pediatric patients with high-risk or relapsed rhabdomyosarcoma (RMS) have not improved significantly since the 1980s. Recent studies have identified a number of targetable vulnerabilities in RMS, but these discoveries have infrequently translated into clinical trials. We propose streamlining the process by which agents are selected for clinical evaluation in RMS. We believe that strong consideration should be given to the development of combination therapies that add biologically targeted agents to conventional cytotoxic drugs. One example of this type of combination is the addition of the WEE1 inhibitor AZD1775 to the conventional cytotoxic chemotherapeutics, vincristine and irinotecan.

Original language	English (US)
Article number	e27869
Journal	Pediatric Blood and Cancer
Volume	66
Issue number	10
DOIs	https://doi.org/10.1002/pbc.27869
State	Published - Oct 2019

Keywords

cancer biology
early-phase clinical trials
genomics
rhabdomyosarcoma

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Hematology
Oncology

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Yohe, M. E., Heske, C. M., Stewart, E., Adamson, P. C., Ahmed, N., Antonescu, C. R., Chen, E., Collins, N., Ehrlich, A., Galindo, R. L., Gryder, B. E., Hahn, H., Hammond, S., Hatley, M. E., Hawkins, D. S., Hayes, M. N., Hayes-Jordan, A., Helman, L. J., Hettmer, S., ... Langenau, D. M. (2019). Insights into pediatric rhabdomyosarcoma research: Challenges and goals. Pediatric Blood and Cancer, 66(10), Article e27869. https://doi.org/10.1002/pbc.27869

Yohe, ME, Heske, CM, Stewart, E, Adamson, PC, Ahmed, N, Antonescu, CR, Chen, E, Collins, N, Ehrlich, A, Galindo, RL, Gryder, BE, Hahn, H, Hammond, S, Hatley, ME, Hawkins, DS, Hayes, MN, Hayes-Jordan, A, Helman, LJ, Hettmer, S, Ignatius, MS, Keller, C, Khan, J, Kirsch, DG, Linardic, CM, Lupo, PJ, Rota, R, Shern, JF, Shipley, J, Sindiri, S, Tapscott, SJ, Vakoc, CR, Wexler, LH & Langenau, DM 2019, 'Insights into pediatric rhabdomyosarcoma research: Challenges and goals', Pediatric Blood and Cancer, vol. 66, no. 10, e27869. https://doi.org/10.1002/pbc.27869

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title = "Insights into pediatric rhabdomyosarcoma research: Challenges and goals",

abstract = "Overall survival rates for pediatric patients with high-risk or relapsed rhabdomyosarcoma (RMS) have not improved significantly since the 1980s. Recent studies have identified a number of targetable vulnerabilities in RMS, but these discoveries have infrequently translated into clinical trials. We propose streamlining the process by which agents are selected for clinical evaluation in RMS. We believe that strong consideration should be given to the development of combination therapies that add biologically targeted agents to conventional cytotoxic drugs. One example of this type of combination is the addition of the WEE1 inhibitor AZD1775 to the conventional cytotoxic chemotherapeutics, vincristine and irinotecan.",

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author = "Yohe, {Marielle E.} and Heske, {Christine M.} and Elizabeth Stewart and Adamson, {Peter C.} and Nabil Ahmed and Antonescu, {Cristina R.} and Eleanor Chen and Natalie Collins and Alan Ehrlich and Galindo, {Rene L.} and Gryder, {Berkley E.} and Heidi Hahn and Sharon Hammond and Hatley, {Mark E.} and Hawkins, {Douglas S.} and Hayes, {Madeline N.} and Andrea Hayes-Jordan and Helman, {Lee J.} and Simone Hettmer and Ignatius, {Myron S.} and Charles Keller and Javed Khan and Kirsch, {David G.} and Linardic, {Corinne M.} and Lupo, {Philip J.} and Rossella Rota and Shern, {Jack F.} and Janet Shipley and Sivasish Sindiri and Tapscott, {Stephen J.} and Vakoc, {Christopher R.} and Wexler, {Leonard H.} and Langenau, {David M.}",

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year = "2019",

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doi = "10.1002/pbc.27869",

language = "English (US)",

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AU - Yohe, Marielle E.

AU - Heske, Christine M.

AU - Stewart, Elizabeth

AU - Adamson, Peter C.

AU - Ahmed, Nabil

AU - Antonescu, Cristina R.

AU - Chen, Eleanor

AU - Collins, Natalie

AU - Ehrlich, Alan

AU - Galindo, Rene L.

AU - Gryder, Berkley E.

AU - Hahn, Heidi

AU - Hammond, Sharon

AU - Hatley, Mark E.

AU - Hawkins, Douglas S.

AU - Hayes, Madeline N.

AU - Hayes-Jordan, Andrea

AU - Helman, Lee J.

AU - Hettmer, Simone

AU - Ignatius, Myron S.

AU - Keller, Charles

AU - Khan, Javed

AU - Kirsch, David G.

AU - Linardic, Corinne M.

AU - Lupo, Philip J.

AU - Rota, Rossella

AU - Shern, Jack F.

AU - Shipley, Janet

AU - Sindiri, Sivasish

AU - Tapscott, Stephen J.

AU - Vakoc, Christopher R.

AU - Wexler, Leonard H.

AU - Langenau, David M.

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N2 - Overall survival rates for pediatric patients with high-risk or relapsed rhabdomyosarcoma (RMS) have not improved significantly since the 1980s. Recent studies have identified a number of targetable vulnerabilities in RMS, but these discoveries have infrequently translated into clinical trials. We propose streamlining the process by which agents are selected for clinical evaluation in RMS. We believe that strong consideration should be given to the development of combination therapies that add biologically targeted agents to conventional cytotoxic drugs. One example of this type of combination is the addition of the WEE1 inhibitor AZD1775 to the conventional cytotoxic chemotherapeutics, vincristine and irinotecan.

AB - Overall survival rates for pediatric patients with high-risk or relapsed rhabdomyosarcoma (RMS) have not improved significantly since the 1980s. Recent studies have identified a number of targetable vulnerabilities in RMS, but these discoveries have infrequently translated into clinical trials. We propose streamlining the process by which agents are selected for clinical evaluation in RMS. We believe that strong consideration should be given to the development of combination therapies that add biologically targeted agents to conventional cytotoxic drugs. One example of this type of combination is the addition of the WEE1 inhibitor AZD1775 to the conventional cytotoxic chemotherapeutics, vincristine and irinotecan.

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KW - early-phase clinical trials

KW - genomics

KW - rhabdomyosarcoma

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Insights into pediatric rhabdomyosarcoma research: Challenges and goals

Abstract

Keywords

ASJC Scopus subject areas

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