The EMEA's negative opinion is disappointing because previous announcements relating to Ark's Cerepro clinical data suggested that it could be a useful adjunct to surgical treatment of glioma. Ark is one of the firms at the forefront of commercial gene therapy (Nat. Biotechnol. 26, 1057–1059, 2008) and has several other gene therapy products well advanced in its pipeline—some of them, according to financial analysts, even more promising than Cerepro, which had anticipated worldwide annual sales of $200 million.
Cerepro consists of a herpes simplex virus gene for thymidine kinase encased in an adenoviral vector from which the E1 and part of the E3 regions have been deleted to prevent viral replication. The thymidine kinase is capable of converting Basel, Switzerland–based Roche's prodrug ganciclovir into its highly toxic form, deoxyguanosine triphosphate. Indicated for people with operable, high-grade malignant glioma, Cerepro treatment involves injection of the gene therapy vector into the cavity that remains after tumor resection followed by administration of ganciclovir. After taking up the thymidine kinase adenovirus package, any residual tumor cells left after surgery are killed when they convert ganciclovir into its toxic form.
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