Rhabdomyosarcoma - Childhood: Latest Research

Approved by the Cancer.Net Editorial Board, 04/2016

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. To see other pages, use the menu.

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 diagnostic and treatment options best for your child.

  • Palliative 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 disease, research is aimed at finding more treatment options. A study of intermediate-risk patients, started in 2006 and recently completed, is testing whether the combination of irinotecan/vincristine, added to VAC chemotherapy, improves the success of this approach. This study is also testing whether the early use of local radiation therapy will decrease local recurrences. Also, a new European study is testing whether the addition of doxorubicin might improve survival.

    A recently completed COG study for high-risk rhabdomyosarcoma tested whether irinotecan, vincristine, and chemotherapy given every 2 weeks improves outcome. While some improvement for children less than 10 years old with embryonal rhabdomyosarcoma, no significant improvement was seen from this study.

  • Kinase inhibitors. Kinase inhibitors are a form of so-called “targeted therapy,” or 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 normal 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 also 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 (tissue taken from a healthy part of the body to replace unhealthy tissue in another part of the body), are ongoing.

  • Tissue collection. All children with rhabdomyosarcoma are encouraged to enroll on the COG study D9902. This study collects tumor and bone marrow samples that are not needed for diagnosis so that scientists can learn more about the disease.

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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