Gene therapy for pituitary tumors

SEILICOVICH; PISERA; SCIASCIA; CANDOLFI; PUNTEL; XIONG; JAITA; CASTRO

CURRENT GENE THERAPY

BENTHAM SCIENCE PUBL LTD

Año: 2005 p. 559 - 572

1566-5232

Abstract: Pituitary tumors are the most common primary intracranial neoplasms. Although most pituitary tumors are consideredtypically benign, others can cause severe and progressive disease. The principal aims of pituitary tumor treatmentare the elimination or reduction of the tumor mass, normalization of hormone secretion and preservation of remaining pituitaryfunction. In spite of major advances in the therapy of pituitary tumors, for some of the most difficult tumors, currenttherapies that include medical, surgical and radiotherapeutic methods are often unsatisfactory and there is a need todevelop new treatment strategies. Gene therapy, which uses nucleic acids as drugs, has emerged as an attractive therapeuticoption for the treatment of pituitary tumors that do not respond to classical treatment strategies if the patients becomeintolerant to the therapy. The development of animal models for pituitary tumors and hormone hypersecretion has provento be critical for the implementation of novel treatment strategies and gene therapy approaches. Preclinical trials usingseveral gene therapy approaches for the treatment of anterior pituitary diseases have been successfully implemented.Several issues need to be addressed before clinical implementation becomes a reality, including the development of moreeffective and safer viral vectors, uncovering novel therapeutic targets and development of targeted expression oftherapeutic transgenes. With the development of efficient gene delivery vectors allowing long-term transgene expressionwith minimal toxicity, gene therapy will become one of the most promising approaches for treating pituitary adenomas