What’s the Latest Development?
The governmental body that oversees European health regulations is set to approve a specific gene therapy to treat a rare genetic disease, perhaps lending the beleaguered medical field some long-awaited support. The therapy, called Glybera, will treat lipoprotein lipase deficiency, a genetic mutation that prevents those affected from “producing an enzyme needed to break down certain fat-carrying particles that circulate in the bloodstream after meals.” The therapy is administered through several shots into the leg muscle and is expected to allow patients to produce the important enzyme for at least a few years.
What’s the Big Idea?
Gene therapy has long been promised as a novel and effective way to treat genetic diseases but setbacks during the 1990s, including the death of a teenager during a clinical trial in Pennsylvania, have delayed its approval. “But researchers have been slowly overcoming the obstacles and in the last few years there have been reports of successes in attempts to treat cancer, hemophilia B, certain immune diseases and a condition that causes blindness.” Dr. Mark A. Kay, a professor of pediatrics and genetics at Stanford, says: “It didn’t occur as rapidly, I think, as people had kind of promised or suggested 15 or 20 years ago, but we are starting to see success.”
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