title: Technology insight: gene therapy and its potential role in the treatment of medullary thyroid carcinoma.
abstract: Metastatic medullary thyroid cancer (MTC) responds poorly to conventional treatments with chemotherapy and radiotherapy. Gene therapy--the transfer of genetic material for therapeutic purposes--might have therapeutic potential for patients with progressive metastatic MTC that is incurable by conventional treatments. To date, a number of gene-therapy strategies have been explored, primarily those that use replication-deficient adenovirus vectors to transfer therapeutic genes to tumor cells. Tissue-specific expression of the promoter for calcitonin and calcitonin-related polypeptide alpha has allowed therapeutic genes to be specifically expressed in calcitonin-secreting cells and in the MTC tumors derived from them; such tissue-specific expression contributes to improved safety of gene therapies and has the potential to increase their therapeutic index. In addition, the identification of an MTC-specific peptide ligand raises the possibility of developing an MTC-selective vector. In this article, we have described the exciting area of gene therapy in the management of MTC with a focus on preclinical in vitro and in vivo MTC models.
authors.affiliation: Cancer Genetics Unit of Kolling Institute of Medical Research, Royal North Shore Hospital, St Leonards, New South Wales, Australia. messina@med.usyd.edu.au
authors.forename: Marinella
authors.initials: M
authors.lastname: Messina
authors.forename: Bruce G
authors.initials: BG
authors.lastname: Robinson
mesh_terms: D018276:Carcinoma, Medullary / Q000628:therapy*; D002986:Clinical Trials as Topic; D003131:Combined Modality Therapy; D018390:Gene Targeting; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D007167:Immunotherapy / Q000379:methods; D013964:Thyroid Neoplasms / Q000628:therapy*