Rhabdomyosarcoma - Childhood: Latest Research

Approved by the Cancer.Net Editorial Board, 06/2021

ON THIS PAGE: You will read about the scientific research being done now to learn more about this type of cancer and how to treat it. Use the menu to see other pages.

Doctors are working to learn more about rhabdomyosarcoma, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your child’s doctor about the best diagnostic and treatment options for your child.

  • Palliative care/supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current rhabdomyosarcoma treatments to improve patients’ comfort and quality of life. For children with rhabdomyosarcoma at a low risk of recurrence, research is focused on decreasing the side effects of treatment by decreasing the total dose of chemotherapy, limiting radiation therapy, and decreasing the total length of time in treatment.

  • Higher-risk rhabdomyosarcoma. For children with higher-risk rhabdomyosarcoma, research is aimed at finding new treatment options and better ways to deliver therapy. A recent COG study of patients with intermediate-risk rhabdomyosarcoma tested whether adding irinotecan/vincristine to standard VAC chemotherapy might improve outcomes. While survival was the same between the 2 treatment regimens, the addition of irinotecan/vincristine did reduce some side effects at an overall lower dose of cyclophosphamide. Another COG study for high-risk rhabdomyosarcoma tested whether irinotecan, vincristine, and chemotherapy given every 2 weeks might improve outcomes. While there was some improvement for children younger than 10 years old with embryonal rhabdomyosarcoma, no significant improvement was seen from this study.

  • Kinase inhibitors. Kinase inhibitors are a type of cancer medication called “targeted therapy.” Targeted therapies are medications that target specific cancer genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of treatment blocks the growth and spread of cancer cells while limiting damage to healthy cells, usually leading to fewer side effects than other cancer medications. For patients with high-risk disease, current research is evaluating a type of targeted therapy that blocks the nutrients needed for a tumor to grow and spread. Learn more about targeted treatments.

  • New approaches to treatment. Research to improve understanding of rhabdomyosarcoma and how drugs affect this disease are being performed. Investigations into the importance of gene translocations in rhabdomyosarcoma are underway. Identifying these translocations may be better than conventional methods of identification in distinguishing between the 2 major types of rhabdomyosarcoma. Research about new treatments, including the use of human rhabdomyosarcoma growing as a tumor xenograft (transplanting tumor tissue into an animal model), are ongoing.

  • Tissue collection. All children with rhabdomyosarcoma are encouraged to consider enrolling on the COG study APEC14B1, "The Project: EveryChild Protocol: A Registry, Eligibility Screening, Biology and Outcome Study." This study collects tumor and tissue samples that are not needed for diagnosis as well as demographic, treatment, and outcome information so that scientists can learn more about the disease, investigate new therapies, and reduce early- and late-onset toxicities.

Looking for More about Latest Research?

If you would like additional information about the latest areas of research regarding rhabdomyosarcoma, explore these related items that take you outside of this guide:

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