Osteosarcoma - Childhood and Adolescence: Latest Research

Approved by the Cancer.Net Editorial Board, 06/2023

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

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

Improved treatment. In several studies, researchers are looking at adding different drugs to standard treatment that may improve the treatment’s success without increasing the side effects, such as:

  • One drug that was tested is the immunotherapy drug called mifamurtide (Mepact). Immunotherapy, also called biologic therapy, is designed to boost the body's natural defenses to fight the cancer. Mifamurtide is currently licensed in Europe by the European Medicines Association for the treatment of localized osteosarcoma that can be removed with surgery. However, it has not been approved by the U.S. Food and Drug Administration (FDA) in the United States. Other approaches on how best to stimulate the patient's own immune system are in various stages of research.

  • As part of the Children’s Oncology Group AOST 0331 study, pegylated interferon alpha (multiple brand names) was added to treatment after 8 months of standard chemotherapy. This study was done with people who have localized osteosarcoma or metastases to the lungs or bones that can be surgically removed and whose tumor was almost completely eliminated by the first 10 weeks of chemotherapy treatment. The results of this study showed that adding pegylated interferon alpha did not benefit these people.

  • In the same study (AOST 0331), etoposide (Toposar, VePesid) and ifosfamide (Ifex) were added to standard chemotherapy treatment for a total of 10 months of treatment instead of the standard 8 months. This study was done with people when the osteosarcoma was not completely eliminated by the first 10 weeks of chemotherapy. According to the results of this study, adding these drugs after surgery caused more side effects and did not improve the outcome of treatment. Therefore, the more intensive chemotherapy approach is not recommended.

    The Children’s Oncology Group and most of the world's experts consider this combination to be standard treatment: cisplatin (Platinol), doxorubicin (Adriamycin), and high-dose methotrexate (multiple brand names). Selected other combination therapies are similarly effective, but none has been proven better.

  • A study of people with metastatic osteosarcoma has also been completed. The study added a bone-stabilizing drug called zoledronic acid (Zometa) to standard chemotherapy. It showed that this combination of treatments did not increase general side effects, which was the goal of the study. A French group studied whether the addition of zoledronic acid to chemotherapy for newly diagnosed people with osteosarcoma would improve the outcome of treatment. Half of the people received a standard chemotherapy treatment and surgery. The other half received zoledronic acid in addition to the standard treatment. The group that received zoledronic acid did not have additional improvements.

  • The Children's Oncology Group has a number of active trials for people with newly diagnosed osteosarcoma, including a clinical trial testing whether adding a drug called cabozantinib, a typed of targeted therapy called a tyrosine kinase inhibitor, to standard chemotherapy will improve survival outcomes.

  • A large number of clinical trials involve people with recurrent osteosarcoma. These studies include people who have had cancer come back once or more than once; people who have had a local or distant recurrence; and people whose cancer recurrence is located in the lungs, other bones, or both. A few of these clinical trials have found drugs that may work for osteosarcoma and are likely to be studied further. One of the trials being conducted by the Children's Oncology Group is a study investigating if the technique used to surgically remove lung nodules influences survival outcomes. If your child's cancer has come back, talk about the results of these studies with your child's health care team.

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

For information about these and other studies, visit the Children’s Oncology Group website or the U.S. National Institutes of Health website.

Talk with your child’s doctor for more information about clinical trials. They can provide additional details about the availability of the diagnostic tests, treatments, or other aspects of care that are being studied. Also, your doctor can provide details on whether they are appropriate for your child.

Looking for More About the Latest Research?

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

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