Texas Children’s Rare Tumors Program

The Orphan Drug Act of 1983 and United States Rare Diseases Act 2002 classifies a rare disease as any disease that affects fewer than 200,000 people in the U.S. Fortunately, by these standards, all childhood cancers are rare. In the past three decades, through cooperative group clinical trials, childhood cancer survival has increased, but some childhood cancers have been left behind. These cancers have such a low incidence that even some pediatric oncologists may not treat a patient with them during their career. For these children, providing treatment in a clinical trial is not possible. Because there is much we do not know about these rare childhood cancers, there can be a delay in diagnosing them and often there is no standard of care for patients.

Though the incidence of each individual rare tumor may be low, as a group, rare tumors contribute to 15 percent of all childhood cancers. The Rare Tumors Program at Texas Children’s Cancer Center was developed to help families who are facing a rare childhood cancer. This multidisciplinary program brings together a team of physicians, nurse practitioners, social workers and researchers. The members of the team will vary depending on the particular tumor type a child has. For example, children with skin cancer (melanoma) will be treated by a pediatric oncologist, dermatologist, radiation oncologist and a surgeon. Children with thyroid cancers will be treated by a pediatric oncologist, endocrinologist, nuclear medicine physician and a surgeon specializing in thyroid surgery.

Rare tumors in children mainly fall into two groups. The first is tumors that occur exclusively in children, the second is adult type tumors (breast cancer, ovarian cancer, colorectal cancer) that occur in children. As the largest children’s cancer center in North America, Texas Children’s Cancer Center is uniquely positioned to help families whose children have rare cancers. Our team treats more children with cancer (including rare cancers) than any other cancer center in North America. In addition, patients come to us from all over the U.S., as well as from other countries, for second opinions. We use the latest cutting edge molecular technology to accurately diagnose rare cancers so they can be appropriately treated. When indicated, we can even study the genetic sequence of these tumors to formulate individualized treatment for patients.

We have ongoing research projects on various rare tumors including thyroid tumors, esthesioneuroblastoma, alveolar soft part sarcoma, myofibroma and neuroendocrine tumor (carcinoid tumor). In addition, we collaborate with national registries to advance the understanding of rare tumors. In fact, we are currently developing a national registry for pediatric neuroendocrine tumors.

Our mission is to help children with rare cancers living anywhere in the world. We have developed a second opinion service which will give recommendations to referring physicians and families in a timely manner. Families and physicians may contact us directly by emailing raretumors@texaschildrens.org.