Europe Gives Green Light to Gene Therapy for Children

The European Medicines Agency has announced its approval for the first life-saving gene therapy for children. This gene therapy, developed by Italian scientists and the British research and development company GlaxoSmithKline, is recommended for a small group of children in Europe suffering from 'severe combined immunodeficiency.' Bone marrow transplants are not effective for these children. The immune systems of children born with this deficiency lack the ability to combat bacterial, fungal, and viral infections. Approximately fifteen children in Europe are born each year with this very rare genetic disease, which incapacitates their bodies from producing a type of white blood cell. These children rarely live beyond two years unless their immune systems are restored through a suitable bone marrow transplant. 'Severe combined immunodeficiency' is also known as 'bubble boy disease' because children with this condition have such weak immune systems that they must live in germ-free environments. According to Reuters, this treatment, named Strimvelis, is expected to receive marketing authorization from the European Commission within a few months. If approved, Strimvelis will be the second gene therapy in Europe after Glybera, developed by UniQure, which is used to treat a rare blood deficiency in adults. The U.S. Food and Drug Administration has yet to approve any gene therapy methods, but several U.S. biotechnology and pharmaceutical companies are developing products in this area. Research in gene therapy dates back twenty-five years, but several incidents, including the death of an American patient in 1999 and some unsuccessful laboratory results in the late 1990s and early 2000s, hindered progress and development in this field. Currently, optimism is increasing with the invention of better methods for replacing genes within cells. Martin Andrews, head of rare diseases at GlaxoSmithKline, believes this technology, although in the early stages of development, will prove effective. He states, 'We are on the first page of the first chapter of a new pharmaceutical book.' However, some issues still need to be addressed. The most significant problem is the pricing of these drugs. Strimvelis and Glybera made history in 2014 as the first drugs priced at one million dollars. Although GlaxoSmithKline has not set a price for its product, a source close to the company says that if Strimvelis is approved, it will be priced significantly lower than one million dollars.