Mesothelioma Community Keeps Watchful Eye on EU Recommendation for Gene Therapy
by Nancy Meredith
Gene therapy has been gaining momentum for years. Within the last few years, geneticists and physicians have partnered to successfully treat patients with incurable, rare diseases that in the past were left untreated. Now, for the first time in the Western world, a committee has recommended approval of gene therapy. This could be the catalyst needed to move gene therapy out of the lab and to the bedside for sufferers of rare diseases, including mesothelioma.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of a gene therapy medicine to treat lipoprotein lipase (LPL), a rare inherited disorder that involves a defective gene, according to a press release posted Friday on its website. The treatment, known as Glybera, offers a new medical treatment for patients with severe or multiple pancreatitis attacks due to lipoprotein lipase deficiency, according to the EMA.
If the European Medicines Agency follows the committee’s recommendation, as is typical according to the New York Times, the medicine would be the first gene therapy medication approved for authorization in the European Union. To date, the U.S. Food and Drug Administration has not approved any gene therapy in the United States, however, the makers of Glybera intend to apply for approval in the U.S. in the future.
In simplest terms, gene therapy pinpoints a defect in a single gene and replaces that gene with a healthy one. The process of developing a gene therapy for a disease, however, is anything but simple. In fact, the chair of the CHMP noted the complexity of moving forward with gene therapy. “Our established ways of assessing the benefits and risks of Glybera were challenged by the extreme rarity of the condition and also by uncertainties associated with data provided.”
In the Forever Fix, Ricki Lewis writes about the first successful use of gene therapy in the United States in the treatment to restore the vision of a young boy who was nearly blind from a hereditary disorder. Lewis explains that while gene therapy typically targets rare, inherited diseases, now it has progressed to the point where acquired diseases, potentially even mesothelioma, could benefit. Specifically, she discusses in the book the often-targeted vascular endothelial growth factor (VEGF) protein.
VEGF is currently targeted in several experimental treatments for mesothelioma. Studies have shown that VEGF, an autocrine growth factor released by mesothelioma cells, causes new blood vessels to form. VEGF levels are shown to be higher in mesothelioma patients suggesting that VEGF may be a proper target for mesothelioma treatments. Although gene therapy has been successful in targeting VEGF in macular degeneration, according to Lewis, gene therapy in non-inherited diseases like mesothelioma is complex because the disease often involves multiple genetic mutations and other contributing factors.
“Getting at the basis of why one person develops mesothelioma and another person doesn’t, that is going to hold a clue to really fighting it,” Lewis said in an interview with MesotheliomaHelp.net. “Then we will know what to do the gene therapy on.”
Mesothelioma is an incurable, asbestos-caused cancer of the membranes that surround many of the body’s vital organs. The most common form, as many as eighty percent of all diagnoses, is pleural mesothelioma, where the cancer attacks the pleural tissue surrounding the lung. The cancer is highly aggressive and is resistant to many cancer treatments making it a difficult disease to treat effectively.
Mesothelioma patients, like many rare disease patients, are closely watching the field of gene therapy and the pending approval from the EMA. Gene therapy is touted as the new frontier in medicine, and the thought that once untreatable diseases could be cured offers hope to patients that they could only dream about in the past.