Novel drug demonstrates early promise against leukemia spurred by oncogene

Researchers at Georgetown Lombardi Comprehensive Cancer Center say that a novel drug that has shown promise against Ewing's sarcoma and prostate cancer also appears to be effective in combating some forms of leukemia. And they want to move toward clinical studies that can help better determine the drug's safety and potential for efficacy in humans.

The researchers, led by Aykut Üren, published their findings online in Oncotarget earlier this week.

Their drug, YK-4-279, was originally developed to hit chromosomal translocations common in patients with the aggressive bone cancer--Ewing's sarcoma--as well as prostate cancers. Since these translocations (two normal genes relocating and fusing together) produce an oncogene protein, EWS-FLI1, that favors the metastasis of cancer cells, the researchers hypothesized this could be effectively prevented by treatment with YK-4-279.

In a mouse model where the prosurvival EWS-FLI1 is overactive, YK-4-279 administration was shown to significantly lower the amount of leukemia cells that was caused by the oncogene protein.

"The fact that treated mice did not get sick from the YK-4-279 gives us an early indication that it might be safe to use in humans, but that is a question that can't be answered until we conduct clinical trials," Üren said. "We are looking for ways that would allow us to administer more of it, or even to formulate a pill."

- here's the release
- read the research article