title: Potential Reuse of Oncology Drugs in the Treatment of Rare Diseases.
abstract: Cancer research has made remarkable progress with the help of advancing genomics techniques, resulting in more precise clinical application and many new anticancer drugs on the market. By contrast, very few treatment options are available for rare diseases that are often progressive, severe, and life-threatening. In this opinion we elaborate on the possible association between cancers and rare diseases across three different levels including clinical observation, crosstalk between germline mutation and somatic mutation, and shared biological pathways. Consequently, by utilizing systematic drug-repositioning approaches, and taking safety issues into consideration, we suggest that oncology drugs have great potential for reuse in the treatment of rare diseases.
authors.affiliation: National Center for Toxicological Research, US Food and Drug Administration, Jefferson, AR 72079, USA. Electronic address: Zhichao.liu@fda.hhs.gov.
authors.forename: Zhichao
authors.initials: Z
authors.lastname: Liu
authors.affiliation: National Center for Toxicological Research, US Food and Drug Administration, Jefferson, AR 72079, USA.
authors.forename: Hong
authors.initials: H
authors.lastname: Fang
authors.affiliation: National Center for Toxicological Research, US Food and Drug Administration, Jefferson, AR 72079, USA.
authors.forename: William
authors.initials: W
authors.lastname: Slikker
authors.affiliation: National Center for Toxicological Research, US Food and Drug Administration, Jefferson, AR 72079, USA. Electronic address: weida.tong@fda.hhs.gov.
authors.forename: Weida
authors.initials: W
authors.lastname: Tong
mesh_terms: D000818:Animals; D000970:Antineoplastic Agents / Q000494:pharmacology / Q000627:therapeutic use*; D015195:Drug Design; D058492:Drug Repositioning; D023281:Genomics / Q000379:methods; D018095:Germ-Line Mutation; D006801:Humans; D009154:Mutation; D009369:Neoplasms / Q000188:drug therapy / Q000235:genetics; D035583:Rare Diseases / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology
chemical_list: D000970:Antineoplastic Agents
keywords: cancer; drug repositioning; genetic mutations; oncology drugs; rare disease