Exit from the bubble. As gene therapy fights immunodeficiency

The first patient underwent Strimvelis therapy after clinical trials. What does it mean

David Vetter, suffering from severe combined immunodeficiency, spent 12 years under a sterile cap. Photo: Bettmann / Getty images

On May second, the pharmaceutical giant GLAXOSMITHKLINE announced that in March 2017, the first patient passed the genetic therapy of Strimvelis. Until now, it has been used only in the framework of clinical trials. The first patient was a child with severe combined immunodeficiency (TKKU). The company keeps its nationality and source of treatment financing. Only a year ago, in May 2016, Strimvelis was approved in Europe. Now therapy can be obtained in a single medical center in Milan, and it costs about 600 thousand euros. What does this news mean for medicine and what does Glaxosmithkline criticize?

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