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Pediatr Blood Cancer. 2019 Oct;66(10):e27869. doi: 10.1002/pbc.27869. Epub 2019 Jun 21.

Insights into pediatric rhabdomyosarcoma research: Challenges and goals.

Author information

1
National Cancer Institute, Bethesda, Maryland.
2
St. Jude Children's Research Hospital, Memphis, Tennessa.
3
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
4
Texas Children's Hospital, Baylor College of Medicine, Houston, Texas.
5
Memorial Sloan Kettering Cancer Center, New York, New York.
6
University of Washington, Seattle, Washington.
7
Boston Children's Hospital, Boston, Massachusetts.
8
Focus on Rhabdo, New York, New York.
9
University of Texas Southwestern Medical Center, Dallas, Texas.
10
University Medical Center Gӧttingen, Gӧttingen, Germany.
11
Summer's Way Foundation, Centennial, Colorado.
12
Seattle Children's Hospital, University of Washington, Fred Hutchinson Cancer Research Center, Seattle, Washington.
13
Molecular Pathology Unit, Massachusetts General Hospital, Charlestown, Massachusetts.
14
University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.
15
Children's Hospital of Los Angeles, Los Angeles, California.
16
University Hospital Berlin, Berlin, Germany.
17
Greehey Children's Cancer Research Institute, San Antonio, Texas.
18
Children's Cancer Therapy Development Institute, Beaverton, Oregon.
19
Duke University School of Medicine, Durham, North Carolina.
20
Children's Hospital Bambino Gesù, IRCCS, Rome, Italy.
21
The Institute of Cancer Research, Sutton, Surrey, United Kingdom.
22
Fred Hutchinson Cancer Research Center, Seattle, Washington.
23
Cold Spring Harbor Laboratory, Cold Spring Harbor, New York.

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.

KEYWORDS:

cancer biology; early-phase clinical trials; genomics; rhabdomyosarcoma

PMID:
31222885
PMCID:
PMC6707829
[Available on 2020-10-01]
DOI:
10.1002/pbc.27869

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