Childhood Cancer May Have New Foe

Ewing sarcoma protein identified as a possible therapeutic target

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

(RxWiki News) The presence of genes, genetic mutations and whether genes are switched on or off can all play a role in cancer. Researchers have discovered a compound that targets a gene involved in childhood cancer.

Ewing sarcoma is a group of cancers that affect the bones in children and young adults.

Scientists have found a compound that targets a molecule which starts the cancer’s journey.

"Never ignore bone pain."

Scientists from Huntsman Cancer Institute (HCI) at the University of Utah worked together to first discover the mechanism behind this cancer and then developed an agent that blocks this activity.

“Ewing sarcoma is a very aggressive bone cancer of children and young adults. Investigators have developed a new understanding of how these tumors develop that suggest an entirely new way to treat these tumors,” Stephen Lessnick, MD, PhD, director of HCI’s Center for Children’s Cancer Research, told dailyRx News.

He explained that a protein called EWS/FLI is nearly always the cause of Ewing sarcoma, which is diagnosed in about 250 youngsters every year in the US.

Dr. Lessick and his team discovered an enzyme called lysine specific demethylase (LSD-1) works with EWS/FLI to turn off certain genes, and these interactions then lead to and result in Ewing sarcoma.

“This makes LSD-1 an important target for the development of new drugs to treat Ewing sarcoma,” said Dr. Lessnick, who is a professor of medicine at UU and an HCI investigator.

While Dr. Lessick’s lab was unraveling the basic biology, Sunil Sharma, MD, director of HCI’s Center for Investigational Therapeutics, professor at the University of Utah, was developing a drug that goes after LSD-1.

Dr. Sharma and colleagues realized LSD-1 is involved in a number of cancers including breast and prostate tumors, as well as acute leukemias.

When this experimental compound – which is years away from being available – was evaluated, “Our tests in Ewing sarcoma tissue cultures show they are extremely potent,” according to Dr. Sharma.

Drs. Lessick and Sharma will continue to test this agent in the lab and in animal models in preparation for human trials.

This study was published November 27 in the journal Oncogene. This research was funded by the National Institutes of Health.

Several of the researchers involved in this study have filed a patent application for this agent.

Reviewed by: 
Review Date: 
November 28, 2012
Last Updated:
November 29, 2012