(RxWiki News) Even when new medications show a lot of promise in the early stages of testing, they don't always prove to work so well in later stages of testing. For certain liver cancer patients, everolimus (Afinitor) may be less effective against tumors than previously thought.
In a recent trial, everolimus did not boost survival compared to placebo (mock medication) in patients with an advanced form of liver cancer.
"Ask your oncologist if there are new treatments for your cancer."
Andrew Zhu, MD, PhD, of the Massachusetts General Hospital Cancer Center and Harvard Medical School in Boston, was this study's lead author.
This investigation enrolled 546 cancer patients living in 17 countries and monitored them from May 2010 through March 2012. The patients were at least 18 years old and had not been diagnosed with HIV or undergone an organ transplant. Except for hepatitis, no infectious diseases existed among the group.
The patients were in the advanced stages of hepatocellular carcinoma and had not been helped by or could not tolerate sorafenib (Nexavar). Thus far, sorafenib is the only treatment prolonging the lives of patients with advanced hepatocellular carcinoma. Sorafenib’s effectiveness is mild and short-lived, with the disease eventually worsening, according to Dr. Zhu and colleagues.
Instead of continuing on sorafenib, 362 of this study’s participants were randomly selected to receive 7.5 milligrams of everolimus per day. Previously, separate, smaller trials of a group of patients in Taiwan and a patient group in the United States showed that everolimus kept tumors from growing. The remaining 184 study participants were assigned to take a placebo instead of sorafenib.
The groups continued to take either everolimus or the placebo until their cancer worsened or they could no longer tolerate everolimus or the placebo. They also continued to receive radiation, chemotherapy or other supportive care.
The worst side effects of everolimus in this study were anemia, loss of appetite and severe physical weakness. Hepatitis B showed in the blood streams of 29 everolimus patients and 10 placebo patients. However, no physical symptoms of hepatitis B flared up in any of those patients.
During this two-year investigation, these researchers found the survival rate to be roughly the same in the two patient groups. Of the 362 patients taking everolimus, 303 (83.7 percent) died. Of the 184 patients taking the placebo, 151 (82.1 percent) died.
This study was the third phase of clinical trials for everolimus, which is one of various medications that researchers hoped would slow the progression of this type of advanced liver cancer.
These results are “highlighting the challenge of developing effective therapies for this cancer,” the researchers wrote.
This study and others also show how hard it can be to determine whether a medication will work solely based on results from first phase and second phase trials, the researchers added.
Among other life-sustaining functions, the liver helps the body absorb nutrients and clear itself of toxins.
Of the known causes of liver cancer in this study's participants, alcohol abuse triggered 20 percent of the cancer cases, the researchers wrote. Hepatitis B triggered 26.2 percent and hepatitis C triggered 25.1 percent of the cancers.
This study was published in the July 2 issue of JAMA.
Novartis, maker of everolimus, funded this study.
Several of the study’s 20 authors have received research grants, speaker, consulting and other fees from a variety of pharmaceutical companies, including Novartis.
