Phase II trial of efatutazone shows challenge of matching treatment to population

April 8, 2014

Work at the University of Colorado Cancer Center led to phase II trial of efatutazone with erlotinib in patients with refractory non-small cell lung cancer. Results are reported today at the American Association for Cancer Research (AACR) Annual Meeting 2014. While efatutazone did not improve the efficacy of erlotinib in this trial, researchers hope lessons from the trial will allow them to make better future use of the drug or other drugs in its class.

"In oncology and especially in , everything is personalized. We're exploring different molecular markers that identify which drugs are for the right patient After this phase II trial, we're working to find the right biomarker that could help us discover who is most likely to respond to efatutazone. This trial was done in an unselected population. But if we had the right population with the right marker, we hope that we could find a meaningful effect," says Ana Oton, MD, investigator at the CU Cancer Center, Associate Professor of Medical Oncology at the University of Colorado School of Medicine, and the study's first author.

Efatutazone belongs to a class of drugs known as thiazolidinediones, members of which are currently in use for the treatment of type II diabetes. Drugs in this class influence the expression of cell proteins by binding to the nuclear receptor PPARy. In diabetes, the drugs increase the production of proteins that drive metabolism. In cancer, the drug showed anti-cancer activity in preclinical models of non-small cell lung cancer. A phase I trial the alone showed anti-cancer activity in with solid malignancies.

The current, multi-center study compared efatutazone with erlotinib versus alone in 90 patients previously treated for non-small cell lung cancer. Unfortunately, Oton explains, "In vitro it looked really promising, but in vivo it was not so good."

The majority of the difference between success in the lab and lack of success in patients was due to the side effect of .

"Fluid retention in lower extremities is a side effect that can be easily managed," Oton says, "however, if this fluid retention is in the pleural space in patients that have already metastatic lung cancer, it can be very detrimental."

"We're not denying this combination could be useful, but with the knowledge we have we shouldn't do more studies in vivo at this time. We need to determine what patient population will be best served and second, discover how to manage this side-effect" Oton says.

Explore further: Scientists looking for second-line defense for patients with NSCLC

Related Stories

Scientists looking for second-line defense for patients with NSCLC

July 25, 2012
DENVER- In lung cancer, patients who benefit from drugs like erlotinib will inevitably develop drug resistance. This is heralded by cancer growth and increasing tumor-related symptoms. Now scientists are investigating a second ...

Pfizer reports promising results for cancer drug

April 7, 2014
An experimental drug has shown encouraging results in treating advanced breast cancer in an early clinical trial, pharmaceutical giant Pfizer reported Sunday.

New treatment holds promise for resistant lung cancer

April 9, 2013
A new chemotherapy regimen appears to produce minimal side effects in patients with lung cancer that has not responded to previous therapy, paving the way for additional research to determine if the new regimen also helps ...

Study to treat deadly form of thyroid cancer shows promise, researchers say

April 19, 2013
(Medical Xpress)—A combination of therapies may prove to be a promising advance for the treatment of anaplastic thyroid cancer based on results of a phase I clinical trial, say researchers at Mayo Clinic in Florida.

Recommended for you

Vitamin C may encourage blood cancer stem cells to die

August 17, 2017
Vitamin C may "tell" faulty stem cells in the bone marrow to mature and die normally, instead of multiplying to cause blood cancers. This is the finding of a study led by researchers from Perlmutter Cancer Center at NYU Langone ...

Outdoor light at night linked with increased breast cancer risk in women

August 17, 2017
Women who live in areas with higher levels of outdoor light at night may be at higher risk for breast cancer than those living in areas with lower levels, according to a large long-term study from Harvard T.H. Chan School ...

Scientists develop novel immunotherapy technology for prostate cancer

August 17, 2017
A study led by scientists at The Wistar Institute describes a novel immunotherapeutic strategy for the treatment of cancer based on the use of synthetic DNA to directly encode protective antibodies against a cancer specific ...

Toxic formaldehyde is produced inside our own cells, scientists discover

August 16, 2017
New research has revealed that some of the toxin formaldehyde in our bodies does not come from our environment - it is a by-product of an essential reaction inside our own cells. This could provide new targets for developing ...

Cell cycle-blocking drugs can shrink tumors by enlisting immune system in attack on cancer

August 16, 2017
In the brief time that drugs known as CDK4/6 inhibitors have been approved for the treatment of metastatic breast cancer, doctors have made a startling observation: in certain patients, the drugs—designed to halt cancer ...

Researchers find 'switch' that turns on immune cells' tumor-killing ability

August 16, 2017
Molecular biologists led by Leonid Pobezinsky and his wife and research collaborator Elena Pobezinskaya at the University of Massachusetts Amherst have published results that for the first time show how a microRNA molecule ...


Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.