Genetic therapies? The heck, you say!

Gene therapies have been somewhat of an urban legend in the pharma/biotech industries for almost as long as I have been alive. Despite how far we’ve come and how far we’ve yet to go, we’re reporting some stories this month that suggest genetic medicine may not be as science fiction-y as it seems.

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When we put our monthly newspaper together, we let the newstake the lead. We use certain standards to select what's appropriate for ourpublication 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 anindustry 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 EuropeanMedicines Agency (EMA) recommended that European regulators authorize themarketing of what would be the first gene-therapy medicine approved for use bypatients in the Western world (see Cover story, "Curing disease at the geneticlevel"). The therapy, uniQure biopharma BV's Glybera, is intended to treatadult patients diagnosed with familial lipoprotein lipase deficiency, anultra-rare genetic disorder affecting the pancreas. Because the EuropeanCommission usually follows the recommendations of the EMA's committee, finalapproval for this groundbreaking therapy is expected to be granted this fall.
Gene therapies have been somewhat of an urban legend in thepharma/biotech industries for almost as long as I have been alive. That hasn'tescaped the notice of some outspoken industry analysts who regarded the Glyberanews with great interest.
"The whole biotechnology industry has overpromised andunderdelivered," Kevin E. Noonan, a partner with McDonnell Boehnen Hulbert& Berghoff LLP, tells me. "Gene therapy was an idea that people weretalking about in the late 1970s or early 1980s, but 30 years later, it stillhasn't happened. I think things have gotten better in the last 10 years becausethere are hundreds of biologic drugs in the pipeline. But technology isdifficult, and regulators are reticent about letting it happen withoutoversight. Despite this approval, I think progress in this area is still goingto 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 asscience fiction-y as it seems. In our 'Omics & Systems Biology section, youcan read about another quest to marry genetic research with effective patienttreatments. In "Study unveils genetic underpinnings of PTSD," I report on anarticle recently authored by researchers at the Boston University School ofMedicine and the Veterans Affairs Boston Healthcare System that identifies agene linked to post-traumatic stress disorder (PTSD). The study replicatesprevious research linking the retinoid-related orphan receptor alpha gene tobehavioral and psychological disorders, and points to a new avenue for researchon the brain's response to trauma.
And these reports follow a pretty groundbreaking story I sharedin our July issue, "Probing the bowels of disease," about efforts by theCrohn's and Colitis Foundation of Canada and Vertex Pharmaceuticals Inc. tofurther research into the genetic causes of Crohn's disease—a condition that myolder sister has and that's impacted our whole family. The research intends to findthe root cause of Crohn's and a way to attack them in the patients who for themost part currently have no effective treatments.
A rare pancreatic disorder, Crohn's disease and PTSD—threeconditions that seemingly had nothing in common, until recently. Reflecting onthe differences and commonalities of these stories, I see that we're inchingever closer to inserting genetic research into medicine—and the advent of thiscrazy thing called "personalized medicine" that we keep hearing about. Here'shoping it doesn't take another 30 years to get there. In the meantime, I inviteyou to continue exploring this trend this fall, as we bring you a two-partseries on trends in personalized medicine. Stay tuned for that in our Octoberissue.
Vertex Pharmaceuticals Inc. 

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