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Pediatric Sarcoma

Sarcoma cancers account for an estimated 10 to 15 percent of all newly diagnosed cancers in children and young adults under the age of 20 in the United States. For this reason, there is a significant need for better therapies and new trials for pediatric sarcoma patients.

Eugenie S. Kleinerman, M.D. is Professor and Head of the Division of Pediatrics, the first women Division Head at M. D. Anderson. She is board certified in pediatrics, holds the Mosbacher Pediatrics Chair and is also a Professor of Cancer Biology. Dr. Kleinerman is internationally recognized for her scientific and clinical expertise in sarcomas, particularly osteosarcoma. Dr. Kleinerman answers some common questions about pediatric sarcoma below.

What is pediatric sarcoma and who does it affect?

Sarcomas are cancers that can arise anywhere within the body where there is bone, muscle, or connective tissue. They are rare tumors and account for approximately 10 to 15 percent of all newly diagnosed cancers in children and young adults under the age of 20 in the United States. Rhabdomyosarcoma (a tumor arising from muscle), Ewing sarcoma (a tumor that typically arises from the bone), and osteosarcoma (a tumor that arises from bone) comprise the majority of sarcomas seen in children and young adults, although other forms of sarcomas can also develop.

Why are new trials so important?

While preclinical data can indicate the therapeutic potential of new drugs, it is imperative to determine whether such new drugs have efficacy against sarcoma in the context of randomized clinical trials. In order to scientifically evaluate the effectiveness of new drugs, patients must receive identical chemotherapy, surgery and radiation regimens. The only variable can be whether the patients receive or do not receive the new therapy. Such a clinical trial design allows us to compare groups and determine whether the addition of a new therapy improves the disease-free and long term survival of this group of patients. There have been very few advances in the treatment of childhood cancers over the last few decades, as a result, the need for new trials to develop new therapies for pediatric cancer patients including those affected by sarcoma is tremendous.

What would you recommend to the families of those with pediatric sarcoma?

Because sarcoma is a rare disease, families should consult a specialist in oncology at a specialized center that treats many patients with this disease. The treatment of pediatric sarcoma requires a multidisciplinary team approach involving an orthopedic surgeon, a radiation oncologist as well as a medical or pediatric oncologist that is familiar with the use of dose intensive chemotherapy in children.

Who should the families of pediatric sarcoma patients contact?

They can call (713) 792-5410 to schedule an appointment with Dr. Cynthia E. Herzog, Dr. Peter M. Anderson or Dr. Winston Huh. These physicians are part of the pediatric sarcoma team at the Children's Cancer Hospital at M. D. Anderson. These individuals oversee the chemotherapy, surgery and radiation therapy. More information can be found with regard to the treatment of patients with sarcoma as well as other types of childhood cancer by logging onto our website www.mdanderson.org/children. This website lists all of our clinical trials in a disease specific manner as well as listing all of our faculty information.

What can the cancer community and the public do to help those that have been and will be diagnosed with sarcoma cancers?

It is extremely important that we raise awareness about the rarer forms of cancer such as sarcoma. Medical research is the hope and the future for improving long term outcome of patients with these diseases. We are urgently in need of additional research funds so that these types of cancers can be studied. We need to understand the molecular mechanisms that are responsible for tumorigenesis, tumor growth, metastatic spread and those pathways that contribute to the growth of tumor vessels which bring oxygen and needed nutrients to the growing tumor to allow the cells to multiply, divide and metastasize. We need research dollars to begin to understand how the organ microenvironment contributes to tumor cell growth at the metastatic site. The National Cancer Institute's budget has been severely cut in recent stifling the number of research grants. This is particularly detrimental to research in the sarcoma field. There are numerous opportunities for funding research in lung cancer, breast cancer, prostate cancer, colon cancer from not only the National Cancer Institute and the Department of Defense, but also philanthropic organizations such as CAP Cure, the Komen Foundation, etc. There are not comparable opportunities for sarcoma research. In addition, because these are rare cancers the pharmaceutical industry is loathe to spend the millions of dollars that are necessary for research and development for new drugs to treat rare types of cancer. It is imperative that our government agencies including the National Cancer Institute and the Federal Drug Administration begin to pay more attention to these rare types of cancer and make a concerted effort to support the needed research. Just as the Breast Cancer Coalition raised awareness and influenced the set aside of millions of dollars for research in breast cancer, those of us in the sarcoma community must band together with one voice to demand equal attention and equal opportunity.


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