Epigenetics: A New Tool for Precision Medicine

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Christopher Vakoc, M.D., Ph.D., leads a team of researchers being funded through LLS’s Career Development Program. At Cold Spring Harbor Laboratory in Long Island, N.Y., Vakoc works in an area of science called epigenetics – the study of chemical changes that switch genes on and off to control cell behavior.

What is epigenetics?

Epigenetics is arguably one of the fastest-moving fields of cancer research.

In the cancer field, it refers to the mechanisms of gene control. Essentially, the machinery in cells that determines whether some genes should be “turned on” or “shut off.”

Why is epigenetics relevant to blood cancer?

As we have studied the DNA (aka the genome) of leukemia cells, and compared it with normal tissues, we have learned that the machines that control epigenetics are commonly altered in this disease.

This has surprised quite a few people, and made many scientists in the field recognize the epigenetics is a major cause of diseases like leukemia. Based on this, many drug-makers are pursuing a hypothesis that targeting epigenetic pathways with drugs might selectively harm leukemia cells while sparing normal tissues.

What is your team investigating?

When changes take place in the proteins that control epigenetic processes, this can affect how genes behave. Our research team is working to discover certain proteins in cells that are responsible for faulty “on” and “off” states of genes.

In blood cancer, cells will often turn “on” cancer-promoting genes that are normally silent and turn “off” genes that can suppress cancer.

Many scientists believe that our investigation of epigenetics in blood cancer will lead to improved targeted cancer therapies, which may restore normality to the epigenetic programing in leukemia cells.

What is innovative about your approach?

Our team is using CRSPR, a new genome editing tool, to reveal ‘hotspots’ in leukemia cells, which might be suitable for drug intervention to treat this disease. We are on the hunt for cancer-causing proteins – or targets – that are below the radar screen of everyone else in the field.

Our hope is that we might “open up” the target landscape in leukemia. This will provide justification for drug developers to pursue next-generation targeted therapies, which will lead to better outcomes for patients.

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