title: Clinical experiences with systemically administered siRNA-based therapeutics in cancer.
abstract: Small interfering RNA (siRNA)-based therapies are emerging as a promising new anticancer approach, and a small number of Phase I clinical trials involving patients with solid tumours have now been completed. Encouraging results from these pioneering clinical studies show that these new therapeutics can successfully and safely inhibit targeted gene products in patients with cancer, and have taught us important lessons regarding appropriate dosages and schedules. In this Review, we critically assess these Phase I studies and discuss their implications for future clinical trial design. Key challenges and future directions in the development of siRNA-containing anticancer therapeutics are also considered.
authors.affiliation: Department of Pathology and Laboratory Medicine, University of California Los Angeles, Los Angeles, California 90095, USA.
authors.forename: Jonathan E
authors.initials: JE
authors.lastname: Zuckerman
authors.affiliation: Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA.
authors.forename: Mark E
authors.initials: ME
authors.lastname: Davis
mesh_terms: D000970:Antineoplastic Agents / Q000494:pharmacology; D017321:Clinical Trials, Phase I as Topic; D020868:Gene Silencing; D006801:Humans; D009369:Neoplasms / Q000188:drug therapy*; D034741:RNA, Small Interfering / Q000494:pharmacology*; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D034741:RNA, Small Interfering