Scientists erase brain tumors in mice

A cure for brain tumors may be one step closer. Scientists from the Johns Hopkins University School of Medicine have discovered that repurposing a drug used for pre-leukemia was able to erase brain tumor cells in mice.

According to a study published in Oncotarget, the drug 5-azacytidine may now have another purpose: erasing brain tumor cells. The researchers used this drug to target a gene mutation that the team first discovered in 2008. This mutation exists in 70 to 80 percent of low-grade, progressive brain cancers.

The mutation hijacks proteins that convert glucose into energy and forces them to make a new foreign molecule called 2-hydroxyglutarate. This molecule then allows groups of atoms called “methyl groups” to attach to DNA strains. The problem is too many of these methyl groups are allowed to latch on to DNA, changing its cell biology and producing cancerous growths.

To attack the tumors, researchers targeted the methyl groups with the drug 5-azacytidine in the hopes of reversing cancer development. Tumor cells from human patients who were likely to have the IDH1 mutation were injected into the skin of mice. These cells grew into tumors a few months later. Then, researchers injected 5-azacytidine into the mice over the next 14 weeks.

The tumors rapidly decreased and seemingly had a complete relapse. Seven weeks after stopping the drug, the tumors still had not re-grown. The mice are still under close observation, but researchers believe the tumors will not grow back.

The researchers are planning a clinical trial for humans with gliomas. They noted, however, that many cancer treatments have worked in mice but failed in humans.

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