Knocking out a pair of genes may help prevent a relapse of leukemia

Working around the notion that cancer-causing mutations on receptors found in bone marrow stem cells could explain why some patients grow resistant to currently used leukemia drugs, researchers at the Indiana University School of Medicine say they've been successful in testing a new approach to fighting the disease.

The big idea here is that these bone marrow stem cells stay hidden from therapies that are used to fight acute myeloid leukemia, allowing the leukemia to make a comeback after chemotherapy is complete.

"The issue in the field for a long time has been that many patients relapse even though chemotherapy and other currently available drugs get rid of mature blast cells quite readily," Indiana University's Reuben Kapur said, referring to the cancerous cells that overrun the blood system in leukemia. "The problem is that the majority of patients relapse because they have remaining residual leukemic stem cells in the bone marrow that are resistant to currently available therapies, including chemotherapy."

But by knocking out genes that produce two proteins, FAK and PAK1, the researchers prevented leukemia from developing in mice--even though they had the cancer-causing receptor mutations in their bone marrow stem cells.

Using mice and human tissue samples, they identified experimental drugs that can knock out the genes. And now they want to refine this approach in a preclinical program and see if they can move into human clinical studies.

- here's the release