Craniopharyngioma - Childhood: Latest Research

Approved by the Cancer.Net Editorial Board, 11/2021

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

For craniopharyngioma, due to the general success of surgery and radiation therapy, research focuses on treatments for recurrent tumors or when tumors are unable to be completely removed with surgery. There is also ongoing research to understand what allows craniopharyngioma to grow. Always talk with your child’s doctor about the best diagnostic and treatment options for your child.

  • Treatment of cysts. Sometimes, the tumor itself is not actively growing, but it has a cyst that is growing and causing symptoms. These cysts can be treated with surgery or by injections of other treatments. Such treatments include radioactive phosphorous P-32, interferon (Roferon-A, Infergen, Intron A, Alferon), and bleomycin (available as a generic drug).

  • Immunotherapy. Immunotherapy, also called biologic therapy, is designed to boost the body’s natural defenses to fight the tumor. It uses materials made either by the body or in a laboratory to improve, target, or restore immune function. Interferon is a type of immunotherapy that helps regulate the body’s natural immune system. Interferon may help treat some childhood craniopharyngiomas. It is given either as a weekly injection under the skin or by injection directly into a cyst (see above).

  • Improved methods of giving radiation therapy. For patients with a tumor that cannot be completely removed during surgery, doctors are studying new techniques for giving radiation therapy. The use of 3-dimensional radiation techniques allows high doses of radiation therapy to be delivered to the tumor with lower doses to nearby healthy brain tissue. These methods may help reduce damage to healthy tissue.

  • Proton therapy. Proton therapy is a type of external-beam radiation therapy that uses protons rather than x-rays. At high energy, protons can destroy tumor cells. Doctors are studying proton therapy for people with a tumor that cannot be completely removed with surgery. Proton therapy allows for high doses of radiation therapy to be delivered to the tumor while reducing radiation doses to the healthy brain tissue. Proton therapy is believed to be equally as effective as existing radiation therapy methods but may have fewer side effects.

  • Molecular profiling and subtyping of craniopharyngioma. Recent studies have identified 2 subtypes of craniopharyngiomas: adamantinomatous craniopharyngioma (ACP) and papillary craniopharyngioma (PCP). ACPs are typically diagnosed in children and teens 5 to 15 years old and in people 45 to 60 years old, whereas PCPs are diagnosed mainly in adults in their 50s and 60s. ACPs are most commonly cystic in appearance and are driven by mutations in the CTNNB1 gene (encoding β-catenin) that affect β-catenin stability. PCPs are typically solid tumors and frequently have BRAF V600E mutations. Research is currently underway to better understand the biology of these subtypes and develop molecular-based targeted therapy for craniopharyngiomas.

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

  • Managing long-term side effects. There are several ongoing studies to find more effective ways to treat obesity, which is a common long-term side effect for survivors of childhood craniopharyngioma. Learn more in this guide's Follow-Up Care section.

Looking for More About the Latest Research?

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

The next section in this guide is Coping with Treatment. It offers some guidance on how to cope with the physical, emotional, social, and financial changes that a central nervous system (CNS) tumor and its treatment can bring. Use the menu to choose a different section to read in this guide.