Ewing Sarcoma - Childhood and Adolescence: Latest Research

Approved by the Cancer.Net Editorial Board, 11/2022

ON THIS PAGE: You will read about the scientific research being done to learn more about Ewing sarcoma and how to treat it. Use the menu to see other pages.

Doctors are working to learn more about Ewing sarcoma, 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 the doctor about the best diagnostic and treatment options for your child.

  • Allogeneic (ALLO) stem cell transplantation. In an ALLO bone marrow/stem cell transplant (see Types of Treatment for a description), the patient is treated with high doses of chemotherapy, radiation therapy, or both to destroy as many tumor cells as possible and to prevent the patient’s immune system from rejecting the donated stem cells. After the high-dose therapy is given, the stem cells obtained from a healthy donor, usually a sibling, are infused into the patient's bloodstream. ALLO stem cell transplants combined with sirolimus (Rapamune) and other drugs have been shown to prevent the growth of Ewing cells in the laboratory.

  • Bilateral lung radiation. For all patients with disease that has spread to the lung, whole-lung radiation therapy should be considered, even if chemotherapy has removed most signs of metastatic disease. During this treatment, radiation therapy is given to both lungs after the patient has completed chemotherapy. For some patients, autologous (AUTO) bone marrow/stem cell transplant (see Types of Treatment for a description) is recommended but should only be done in a clinical trial.

  • Treatment for recurrent disease. Some studies focused on recurrent Ewing sarcoma are evaluating tumor DNA to look for molecular changes that can be targeted during treatment. This is done using a technology called next-generation sequencing.

  • Circulating tumor DNA. Circulating tumor DNA (ctDNA) is cancer DNA found in the blood. Researchers are looking at the benefits of assessing ctDNA at the time of diagnosis and after 2 cycles of chemotherapy.

  • Palliative and supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current Ewing sarcoma treatments to improve comfort and quality of life for patients.

  • Research to better understand Ewing sarcoma. In addition to the consistent aberrations involving the EWSR1 gene at 22q12, additional numerical and structural aberrations have been observed in Ewing sarcoma, including gains of chromosomes 2, 5, 8, 9, 12, and 15; the nonreciprocal translocation t(1;16)(q12;q11.2); and deletions on the short arm of chromosome 6. Trisomy 20 may also be associated with a more aggressive subset of Ewing sarcoma. Other recurring genomic alterations identified in several genes in Ewing sarcoma are STAG2 mutations (seen in 15% to 20% of cases), which is associated with advanced stage disease; CDKN2A deletions (seen in 12% to 22% of cases); and TP53 mutations (seen in 6% to 7% of cases), which, when present with STAG2 mutations, are associated with a poor outcome. Trisomy 8 is the most frequent chromosomal alteration in Ewing sarcoma and occurs in about 50% of tumors. Gain of 1q and loss of chromosome 16q occurs in about 20% of patients and often occur together.

  • Research in fusion oncoproteins. Fusion oncoproteins are abnormal molecules made of the fused parts of 2 proteins. The National Cancer Institute launched the Fusion Oncoproteins in Childhood Cancers (FusOnC2) Consortium to learn more about the role of fusion oncoproteins in childhood cancers.

Looking for More About the Latest Research?

If you would like more information about the latest areas of research in Ewing sarcoma, explore these related items that will take you outside of this guide:

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