The Potential of Epigenetics

A better understanding of the microscopic universe that surrounds and controls DNA offers innovative therapeutic possibilities.

It has long been assumed that we’re stuck with the genes we’re born with—for better or worse. But epigenetics, the dialing up and down of genes by environmental factors, is altering that view.

The study of epigenetics is the study of how information coded by genes is translated and expressed by cells, and it looks at how inherited traits are altered by environmental conditions without changing the actual DNA sequence.

Exactly how the environment influences genes is still being unraveled. Lifestyle choices and other factors appear to alter the molecules that surround DNA, affecting how tightly it is packed and thus how active or inactive certain genes are. The packing is controlled by several families of enzymes that add or remove tiny chemical “tags” on the DNA.

The exciting thing about epigenetic changes, unlike genetic mutations, is that they can be reversed. And that makes it attractive to researchers hoping to cure diseases of all kinds. By playing with the tagging enzymes, troublesome genes that give rise to cancers can be reset to a normal state. “It’s easy to make inhibitors of enzymes,” Scott Armstrong, oncologist at Memorial Sloan-Kettering Cancer Center in New York, recently told Nature. “Everyone sees this as a potential opportunity.”

Targeted therapies activate or deactivate parts of the chromatin with the aim of slowing or stopping a disease. Therapies such as this can also be used in combination with more traditional treatments such as chemotherapy.

While fewer than half a dozen epigenetic medications are on the market today, their potential is on the rise. Celgene has two therapies that target epigenetic alterations and is looking to develop more. To that end, Celgene partnered with Cambridge, Mass.-based Epizyme, a biopharmaceutical company focused on epigenetics, in April 2012.

The companies are looking to identify therapies to treat a rare blood cancer called mixed lineage leukemia (MLL). Although the patient population is not large, those with this form of leukemia have few options. And that makes it the perfect arena for Celgene.