One of the chief culprits behind infant leukemia, and a significant number of adult cases, is a fusion protein involving MLL which triggers genes that drive the development of cancer. Proteins from the BET family, investigators say, target those suspect genetic drivers. And they've come up with a new compound that stop BET and MLL from binding with chromatin, proving in mouse studies that if they could interfere with that process they could prevent leukemia from developing in rodents.
The investigation drew together researchers from GlaxoSmithKline ($GSK), Cellzome as well as scientists from the Wellcome Trust-Cancer Research UK Gurdon Institute and the Cambridge Institute for Medical Research who have made an important step in the field of epigenetics, the very early-stage but growing field that promises to produce a new generation of cancer therapies.
"Our work shows that this type of leukemia is reliant on MLL being able to bind to chromatin via BET proteins," says study co-leader Professor Tony Kouzarides. "This 'epigenetic' approach to therapy--which targets chromatin rather than DNA--is an exciting new avenue for drug discovery which we hope will be useful for other types of cancer in addition to mixed-lineage leukemia."
Obviously this program has a long way to go before it can claim success in fighting cancer. But by providing some animal data to back up the medical theory, the investigators have taken a big step toward first-in-human drug studies. And that will encourage all the biotechs exploring epigenetics.
"Even though this is still lab-based...it validates the idea of developing small molecules against epigenetic switches," Kevin Lee, who runs epigenetics discovery research at GSK, notes to Reuters.