Gene therapy – a risky approach aimed at fixing the malfunctioning genes at the root of some diseases – is finally emerging from its own darkness after weathering high-profile tragedies, including the death of a teenage patient. And, according to the story in the Washington Post, as it evolves from experimental to applied medicine, gene therapy might soon find itself steeped in a new controversy: soaring drug prices. No therapy is approved yet in the United States. But industry leaders are already talking about massive one-time price tags that could make insurers and patients balk. A gene therapy approved in Europe in 2012 costs close to $1 million, and prices are expected to follow suit in the United States. And the industry anticipates the potential backlash against a seven-figure price tag.
As basic research moved forward, excitement about gene therapy soared. But, that initial exuberance would die down as the powerful idea of replacing broken genes collided with the inherent complexity of human biology. In 1999, an Arizona teenager named Jesse Gelsinger died after he experienced an unexpected, severe immune reaction while participating in a clinical trial of gene therapy led by researcher James M. Wilson at the University of Pennsylvania.
Wilson became the subject of legal action and scathing media coverage. The government restricted his work on human subjects. Lawmakers on Capitol Hill held hearings to probe the lack of oversight and the ethical lapses that had marked some gene-therapy trials. Also in the early 2000s, a few patients in a French gene-therapy trial developed leukemia. Along with the Gelsinger case, it proved a tipping point. Private investment in the field rapidly dried up, and a “nuclear winter” followed. Regulators halted dozens of trials.
Gene therapy’s comeback started with a trickle. In 2008, researchers reported that a small number of patients with an inherited form of blindness gained modest improvements in vision with gene therapy. Not long after, gene therapy restored immune function in eight of 10 children with typically lethal “bubble boy” disease. Katherine High, a researcher at the Children’s Hospital of Philadelphia who worked on one of the early blindness trials, started getting cold calls from investors and from pharmaceutical companies, asking if they could partner with her. Then the team of experts she had assembled in Philadelphia began to get job offers.
Today, many companies have treatments in the pipeline. UniQure’s drug, Glybera, in 2012 became the first gene therapy approved in Europe, for a rare metabolic disease. Bluebird Bio, a biotechnology company that went public in 2013, is developing a variety of gene therapies, including a treatment for a genetic blood disease. Regenxbio, where Wilson serves as chief scientific adviser, went public in September.
But the potential sticker shock from a million-dollar drug – even if it’s for a previously incurable disease – is sure to raise some of the same questions from politicians and the public. Spark Therapeutics’ gene therapy is estimated to cost $500,000 per eye. Spending on hemophilia B a gene therapy could conceivably be priced as a one-time payment of $4 million to $6 million. Spark Therapeutics chief executive Jeffrey Marrazzo is reluctant to talk about a dollar figure.