Researchers from the Houston area say they have discovered a potential new drug that will deliver a ‘deadly one-two punch’ against leukemia.
According to a news release, the researchers from Rice University and the University of Texas MD Anderson Cancer Center said that although the potential drugs are still years away from being clinically tested, their recent study highlights the innovative methods that led to the discovery.
“In previous studies, the research groups of Rice biochemist Natasha Kirienko and MD Anderson physician-scientist Marina Konopleva screened some 45,000 small-molecule compounds to find a few that targeted mitochondria,” the release explained. “In the new study, they chose eight of the most promising compounds, identified between five and 30 closely related analogs for each, and conducted tens of thousands of tests to systematically determine how toxic each analog was to leukemia cells, both when administered individually or in combination with existing chemotherapy drugs like doxorubicin.”
As of now, the compound is being experimented with through a cutting-edge technique called a patient-derived xenograft (PDX), also referred to as a “mouse clinical trial,” where the mice will be implanted with leukemia cells before being exposed to the drugs.
“Although this is very promising, we’re still some distance from having a new treatment we can use in the clinic,” Kirienko added. “We still have a lot to discover. For example, we need to better understand how the drugs work in cells. We need to refine the dose we think would be best, and perhaps most importantly, we need to test on a wide variety of AML cancers. 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.”
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According to a news release, the researchers from Rice University and the University of Texas MD Anderson Cancer Center said that although the potential drugs are still years away from being clinically tested, their recent study highlights the innovative methods that led to the discovery.
“In previous studies, the research groups of Rice biochemist Natasha Kirienko and MD Anderson physician-scientist Marina Konopleva screened some 45,000 small-molecule compounds to find a few that targeted mitochondria,” the release explained. “In the new study, they chose eight of the most promising compounds, identified between five and 30 closely related analogs for each, and conducted tens of thousands of tests to systematically determine how toxic each analog was to leukemia cells, both when administered individually or in combination with existing chemotherapy drugs like doxorubicin.”
As of now, the compound is being experimented with through a cutting-edge technique called a patient-derived xenograft (PDX), also referred to as a “mouse clinical trial,” where the mice will be implanted with leukemia cells before being exposed to the drugs.
“Although this is very promising, we’re still some distance from having a new treatment we can use in the clinic,” Kirienko added. “We still have a lot to discover. For example, we need to better understand how the drugs work in cells. We need to refine the dose we think would be best, and perhaps most importantly, we need to test on a wide variety of AML cancers. 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.”
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