AAAS Meeting 2009: Epigenetic Therapy

Posted by Amy Maxmen, PhD Science Writer for the Journal of Experimental Medicine

Shoot at methylated sites, recommends Peter Jones from the University of Southern California on Saturday at the AAAS Meeting. Theoretically at least, the approach packs more bang for your buck. Releasing a promoter from the grips of methylation allows for the transcription of a perfectly fine gene. 

With whole genomes at our disposal, scientists have identified chromosomal areas of epigenetic modification prevalent in cancer. Jones displays a couple of diagrams illustrating these identified chromosomes and points out where the methylation of some lysine groups has silenced genes, leading to cancerous cell proliferation.

Drugs to flip this methylated switch to reactivate silenced genes were at first inadvertently invented decades ago. One such demethylating drug is now in Phase III clinical trials for the treatment of myeloid leukemia and the results look promising. 

Yet without methylation in moderation, we would surely die. For example, women would be overwhelmed by an excess of gene expression coming from their second X chromosome. Another audience member speaks my thoughts as he asks about side effects of epigenetic therapy. After a pause, Jones nods, “This does remain an issue.” For now, he says, these drugs would only be given to people in a life-threatening situation.