As reported by MIT Technology Review website, the latest gene therapy for children suffering from ADA-SCID will be offered with a money-return guarantee. ADA-SCID is a rare immunological disorder resulting in that the organisms of children are not capable of fighting common infections. Typically, young patients affected die within the first few years of their lives.
Strimvelis, a new gene therapy aimed at the treatment of ADA-SCID, was commercialised by GlaxoSmithKline. Throughout the last 15 years, there has been research conducted on its effectiveness - the result of it is that 18 children have been outright cured. The greatest drawback of the method is the cost it incurs - EUR 594,000. According to MIT Technology Review, it makes it one of the most expensive one-time therapies that has ever been sold by a drug company. However, the representatives of the Italian Medicines Agency, which will be responsible for the arrangement of treatment for European children, have negotiated the guarantee of cost reimbursement if the therapy does not work. This is the first case of this kind in the world.
The representatives of GSK admit that they will probably not cash in on Strimvelis, as the treatment will concern 12 children a year. However, on the one hand, they find it valuable that there will be help provided to small patients, while - on the other hand - it will also allow them for gaining experience associated with the therapies of this kind. Currently, they are co-working with Adaptimmune company on genetically-altered cells that are aimed at combating cancer.Sources: www.technologyreview.com, www.economist.comImage credits: PublicDomainPicturesImage source: www.pixabay.com