Shrinking Brain Tumors and a Whole Lot More

Everolimus adds pediatric brain tumor treatment to its uses

/ Author:  / Reviewed by: Robert Carlson, M.D

(RxWiki News) It started out as a drug to combat organ rejection in transplant patients. Everolimus now has many uses, including treating various types of cancer. And the list of its applications is expanding.

Everolimus dramatically shrinks brain tumors in children with a rare disorder. The tumors are seen in patients with a genetic disease - tuberous sclerosis complex (TSC) – which causes cancers to grow on primary organs.

The study, which confirms earlier trial findings, suggests everolimus may be effective in treating other diseases including Alzheimer’s, Parkinson’s and autism.

Everolimus is sold under the brand name Afinitor as an anticancer medication. Zortress is the brand name for the drug that prevents organ rejection.

"Ask about the latest drugs for your condition."

The phase III trial was led by David N. Franz, MD, co-director of the TSC Clinic at Cincinnati Children's.

"Every patient in this study experienced a decrease in size of their tumors, and no patient required surgery for their tumors after treatment with everolimus," said lead study author, Dr. Franz, co-director of the TSC Clinic at Cincinnati Children’s. "Thirty-five percent of patients in this study on everolimus had at least a 50 percent reduction in tumor volume after an average of 42 weeks on medication."

TSC is diagnosed in about 750 people each year. It’s a genetic disorder that causes tumors to develop in organs throughout the body, buts mostly in the brain, eyes, heart, kidney, skin and lungs.

Almost 50,000 children and adults in the United States and about a million people around the world live with TSC.

This placebo-controlled trial involved 117 patients who were an average of 9 ½ years old.

The results of an earlier phase II study of everolimus were impressive enough that the US Food and Drug Administration granted it Priority Review and accelerated approval in August of this year. This most recent trial confirmed those findings.

Everolimus is being used to treat tumors called subependymal giant cell astrocytomas, or SEGAs. These tumors appear in about one of every 10 people who has tuberous sclerosis complex (TSC).

Before Afinitor was approved, surgery was usually the only option to treat these tumors. This drug may be an alternative to surgery, according to Dr. Franz.

“The ability to reduce tumor volume with drug therapy and avoid surgery is a clear advantage for patients with this disease,” said Keith L. Black, MD, chair and professor of Cedars-Sinai’s Department of Neurosurgery. Dr. Black, who was not involved in this trial, is also the director of the Cochran Brain Tumor Center, director of the Maxine Dunitz Neurosurgical Institute and is the Ruth and Lawrence Harvey Chair in Neuroscience.

Dr. Franz said in a news release that the drug may not only help youngsters avoid surgery, but could also reduce fluid build-up in the brain which causes pressure inside the skull.

The so-called mTOR pathway that’s associated with excessive cell growth in TSC is also associated with other diseases, including Alzheimer's disease, autism, type II diabetes, Parkinson's and Huntington's disease.

Since Afinitor is a mTOR inhibitor, Dr. Franz says, it could potentially be used to treat these and other disorders involving the pathway.

“Whether insights gained from the response of this subset of brain tumors to everolimus will lead to improved treatments of other types of brain tumors will be of high interest,” Dr. Black said.

Earlier this year, the FDA approved the drug to shrink non-cancerous kidney tumors, which are seen in 80 percent of folks with TSC.

Afinitor is also approved to treat a certain type of pancreatic cancer and advanced kidney cancer. It is also used in combination with Aromasin (exemestane) to treat women with HER2-postive breast cancer.

Findings from this study were published November 14 in The Lancet. The study was funded by Novartis Pharmaceuticals, which manufactures everolimus. A number of the study authors are employees or consultants of Novartis or have other financial relationships with the company.

Reviewed by: 
Review Date: 
November 11, 2012
Last Updated:
November 14, 2012