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Prioritization of Novel Agents for Patients with Rhabdomyosarcoma: A Report from the Children's Oncology Group (COG)

Holly L Pacenta1,2, Wendy Allen-Rhoades3, David Langenau4

  • 1Cook Children's Medical Center, Division of Hematology and Oncology, Fort Worth, TX 76014, USA.

Journal of Clinical Medicine
|April 30, 2021
PubMed

Insights

Children

Area of Science:

  • Pediatric Oncology
  • Medical Oncology
  • Drug Discovery

Background:

  • Rhabdomyosarcoma is a prevalent soft tissue sarcoma in children and adolescents.
  • Advanced or relapsed rhabdomyosarcoma presents significant treatment challenges with poor patient outcomes.

Purpose of the Study:

  • To systematically evaluate novel therapeutic agents for rhabdomyosarcoma.
  • To prioritize agents for inclusion in future clinical trials (Phase 2/3) for pediatric rhabdomyosarcoma patients.

Main Methods:

  • Convened a multidisciplinary task force (clinicians and scientists) under the Children's Oncology Group (COG).
  • Developed a framework for evaluating and prioritizing novel agents.
  • Reviewed key classes of investigational drugs for rhabdomyosarcoma treatment.

Main Results:

  • Identified and reviewed promising novel agents for rhabdomyosarcoma.
  • Key agent classes discussed include PARP inhibitors, MEK inhibitors, HDAC inhibitors, and novel cytotoxic agents.
  • Prioritized agents for further clinical investigation based on the established framework.

Conclusions:

  • The COG rhabdomyosarcoma new agent task force established a systematic approach for novel drug evaluation.
  • Several classes of novel agents show promise for improving outcomes in pediatric rhabdomyosarcoma.
  • This work will guide the development of future clinical trials for rhabdomyosarcoma.
Keywords:
clinical trialsmetastasisnew agentsrelapserhabdomyosarcoma

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