Kidney Drug Helps Transplants & Fights Cancer

Angiomyolipoma treatment with everolimus has successful phase III trial

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) Frequently, drugs start life as a treatment for one condition. With increasingly complicated drugs affecting the immune system, it seems the sky's the limit for potential uses of some medications.

Early research using everolimus focused on the key ability to suppress the immune system, allowing kidney transplants to survive. A portion of recent research has also explored additional cancer suppressing properties of the drug.

"Ask your oncologist about everolimus."

Everolimus, marketed under the name Zortress" data-scaytid="7">Zortress and Afinitor" data-scaytid="9">Afinitor in the United States depending on the usage, has been researched for possible use in treating several types of cancer in addition to its extensive use in kidney transplantation. A recent phase III trial showed tumor reductions of over 50 percent for 42 percent of the patients in the trial that had a rare kidney tumor, angiomyolipoma (AML).

The study design included a total of 112 patients, 79 of who were given 10 milligrams of everolimus and 39 who received a  placebo. Confirmed results after more than 40 weeks of treatment included cases where tumors were halved in size.

Nearly 42 percent of the patients in the study had a clear response to everolimus shown by reduction in tumor volume, in comparison to zero response for the placebo.

"Having a drug therapy that actually shrinks the AMLs is huge...but then this drug also appears to [positively] affect other aspects of the disease," saidJohn Bissler, MD, from Cincinnati Children's Hospital Medical Center.

Most of the side effects in the study were minor, and the few serious side effects were considered to be an acceptable risk given the seriousness of the cancer.

Angiomyolipoma is uncommon and few significant treatment options exist. Most cases of angiomyolipoma occur in patients who have a genetic disorder known as tuberous sclerosis.

The study authors concluded their presentation by stating that, "Everolimus represents the first potential pharmacologic treatment option for patients with AML."

Everolimus was first approved by the FDA for the treatment of advanced kidney cancer in 2009. Researchers have announced their intention to use the results from this phase III clinical trial in seeking regulatory approval internationally.

Research presented at conferences is considered preliminary until publication in a peer-reviewed journal.

The authors of this paper disclosed financial relationships with several corporations, including the medical technology corporation Gambro and pharmaceutical company Novartis.

Reviewed by: 
Review Date: 
May 30, 2012
Last Updated:
June 6, 2012