New drug combo could fight aggressive blood cancer

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Researchers from Karl Landsteiner University of Health Sciences (KL Krems) have discovered that combining two existing drugs can kill leukemia cells and limit their spread. The study, published in Cancer Letters, highlights a new treatment strategy for acute myeloid leukemia (AML), an aggressive blood cancer.

How the Drug Combination Works

The study focused on two well-known drugs:

  • Atovaquone, an antimalarial drug that has shown anti-cancer properties.
  • Selinexor, an approved anticancer drug.

Together, these drugs influence a key protein called STAT3, which plays a role in cancer cell growth. AML cells often have too much of a specific form of STAT3 (STAT3α), which promotes cancer spread. However, another form, STAT3β, can help suppress cancer.

The key discovery: This drug combination shifted the balance toward the cancer-suppressing STAT3β form. As a result:

  • Leukemia cells were killed more effectively.
  • The presence of cancer cells in the bone marrow was significantly reduced.
  • The drug combination also increased levels of CD62L, a molecule that may help slow cancer spread.

Why This Discovery Matters

AML is a fast-growing and hard-to-treat cancer that disrupts normal blood cell production. Scientists have tried to block STAT3 as a treatment strategy, but these efforts have largely failed because STAT3 exists in two forms with opposing effects. This new approach focuses on adjusting the balance between the two forms rather than completely blocking STAT3.

Lead researcher Prof. Dr. Dagmar Stoiber-Sakaguchi explains:
“We were looking for ways to shift this ratio in a way that would provide a therapeutic benefit—and we succeeded.”

A New Direction for Cancer Treatment

This study opens up a new treatment avenue by using already-approved drugs to target STAT3 in a more precise way. While the research is still in the experimental stage, it suggests a promising strategy for improving AML treatment without needing entirely new drugs.

Future studies will be needed to test how effective this combination is in human clinical trials. If successful, this approach could offer a new, more effective treatment option for AML patients, potentially improving survival rates.

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The research findings can be found in Cancer Letters.

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