In a breakthrough, researchers from Rice University and the University of Texas MD Anderson Cancer Center in Houston discovered a potential drug with leukemia-killing compounds.

Published in the journal Leukemia, the study stated that while the drug was still years away from being tested in patients with cancer, the breakthrough showed plenty of promise, both in the goal and the innovative methods used to get there.

Conducting the research during her postdoctoral studies at Rice, University of Texas researcher and study lead author Svetlana Panina said that one of the biggest challenges was to “establish optimal conditions and doses for testing on both cancer cells and healthy cells.”

“The results from our previously published cytotoxicity assay were helpful, but very little is known about these small-molecule compounds. None of them had been thoroughly described in other studies, and we had to essentially start from scratch to determine how much to use, what they do in cells, everything. All the doses and treatment conditions had to be adjusted by multiple preliminary experiments,” Panina continued.

Per prior research, leukemia cells are not only more damaged than healthy cells, they are also more sensitive to mitochondrial damage. Rice biochemist Natasha Kirienko and MD Anderson physician-scientist Marina Konopleva surmised that mitophagy-inducing drugs might make the cells more susceptible to chemotherapy.

The team tested the toxicity of mitophagy-inducing compounds like PS127B and PS127E against acute myeloid leukemia (AML) cells, the most common form of the disease. In control studies, all mitophagy-inducing drugs caused less harm to healthy cells but were effective at killing AML cells when tested on mice.

Despite the findings, the researchers believed that a new treatment was still far in the future.

“AML has a lot of variations, and we need to know which patients are most likely to benefit from this treatment and which are not. Only after we’ve done that work, which may take a few years, would we be able to start testing in humans,” Kirienko said via Eureka Alert.

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