A new kind of chemo: Researchers identify a new class of drugs that offer a safer, more targeted treatment for leukemia patients

Chemotherapy sucks. The treatments generally have awful side effects, and it’s no secret that the drugs involved are often toxic to the patient as well as their cancer. The idea is that, since cancers grow so quickly, chemotherapy will kill off the disease before its side effects kill the patient. That’s why scientists and doctors are constantly searching for more effective therapies.

A team led by researchers at UC Santa Barbara, and including collaborators from UC San Francisco and Baylor College of Medicine, has identified two compounds that are more potent and less toxic than current leukemia therapies. The molecules work in a different way than standard cancer treatments and could form the basis of an entirely new class of drugs. What’s more, the compounds are already used for treating other diseases, which drastically cuts the amount of red tape involved in tailoring them toward leukemia or even prescribing them off-label. The findings appear in the Journal of Medicinal Chemistry.

“Our work on an enzyme that is mutated in leukemia patients has led to the discovery of an entirely new way of regulating this enzyme, as well as new molecules that are more effective and less toxic to human cells,” said UC Santa Barbara Distinguished Professor Norbert Reich, the study’s corresponding author.

The epigenome

All cells in your body contain the same DNA, or genome, but each one uses a different part of this blueprint based on what type of cell it is. This enables different cells to carry out their specialized functions while still using the same instruction manual; essentially, they just use different parts of the manual. The epigenome tells cells how to use these instructions. For instance, chemical markers determine which parts get read, dictating a cell’s actual fate.

A cell’s epigenome is copied and preserved by an enzyme (a type of protein) called DNMT1. This enzyme ensures, for example, that a dividing liver cell turns into two liver cells and not a brain cell.

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