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Genetic therapies? The heck, you say!
September 2012
by Amy Swinderman  |  Email the author

When we put our monthly newspaper together, we let the news take the lead. We use certain standards to select what's appropriate for our publication and audience, and the headlines speak for themselves. Sometimes, when all of the stories have been selected and assigned, I notice patterns. Trends. Commonalities in stories that seemingly have no connection.  
That's no great revelation for a publication that covers an industry that is prone to cyclical changes and susceptible to market forces. But once in a while, the trend is unexpected.  
First came the news that a committee of the European Medicines Agency (EMA) recommended that European regulators authorize the marketing of what would be the first gene-therapy medicine approved for use by patients in the Western world (see Cover story, "Curing disease at the genetic level"). The therapy, uniQure biopharma BV's Glybera, is intended to treat adult patients diagnosed with familial lipoprotein lipase deficiency, an ultra- rare genetic disorder affecting the pancreas. Because the European Commission usually follows the recommendations of the EMA's committee, final approval for this groundbreaking therapy is expected to be granted this fall.  
Gene therapies have been somewhat of an urban legend in the pharma/biotech industries for almost as long as I have been alive. That hasn't escaped the notice of some outspoken industry analysts who regarded the Glybera news with great interest.  
"The whole biotechnology industry has overpromised and underdelivered," Kevin E. Noonan, a partner with McDonnell Boehnen Hulbert & Berghoff LLP, tells me. "Gene therapy was an idea that people were talking about in the late 1970s or early 1980s, but 30 years later, it still hasn't happened. I think things have gotten better in the last 10 years because there are hundreds of biologic drugs in the pipeline. But technology is difficult, and regulators are reticent about letting it happen without oversight. Despite this approval, I think progress in this area is still going to move at a glacial pace."
Despite how far we've come and how far we've yet to go, there is another story this month that suggests genetic medicine may not be as science fiction-y as it seems. In our 'Omics & Systems Biology section, you can read about another quest to marry genetic research with effective patient treatments. In "Study unveils genetic underpinnings of PTSD," I report on an article recently authored by researchers at the Boston University School of Medicine and the Veterans Affairs Boston Healthcare System that identifies a gene linked to post-traumatic stress disorder (PTSD). The study replicates previous research linking the retinoid-related orphan receptor alpha gene to behavioral and psychological disorders, and points to a new avenue for research on the brain's response to trauma.
And these reports follow a pretty groundbreaking story I shared in our July issue, "Probing the bowels of disease," about efforts by the Crohn's and Colitis Foundation of Canada and Vertex Pharmaceuticals Inc. to further research into the genetic causes of Crohn's disease—a condition that my older sister has and that's impacted our whole family. The research intends to find the root cause of Crohn's and a way to attack them in the patients who for the most part currently have no effective treatments.  
A rare pancreatic disorder, Crohn's disease and PTSD—three conditions that seemingly had nothing in common, until recently. Reflecting on the differences and commonalities of these stories, I see that we're inching ever closer to inserting genetic research into medicine—and the advent of this crazy thing called "personalized medicine" that we keep hearing about. Here's hoping it doesn't take another 30 years to get there. In the meantime, I invite you to continue exploring this trend this fall, as we bring you a two-part series on trends in personalized medicine. Stay tuned for that in our October issue.          
            Vertex Pharmaceuticals Inc. 



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