囊性纤维化的个性化药物治疗：梦想照进现实

Title:Personalized Drug Therapy in Cystic Fibrosis: From Fiction to Reality

Volume: 16 Issue: 9

Author(s): Fernando Augusto de Lima Marson, Carmen Silvia Bertuzzo and Jose Dirceu Ribeiro

Affiliation:

关键词: 囊性纤维化跨膜电导调节因子(CFTR)，校正器，囊性纤维化，个性化药物治疗，增效剂，PTC124，VX-770，VX-809

摘要: Personalized drug therapy for cystic fibrosis (CF) is a long-term dream for CF patients, caregivers, physicians and researchers. After years of study, the fiction of personalized treatment has turned to hope. Basic information about CFTR mutations classes and new treatments is needed if we are to deal properly with the new CF era. The problems involved in this issue, however, should be evaluated with greater care and attention. VX-770 is a new drug available to treat CF patients with some class III CFTR mutations and other drugs are being studied regarding other classes. The scientific literature has constantly given information about each therapy, both in vitro and in vivo. The hope is increasing. Nevertheless the “scientific world” still lacks information about patients´ reality and daily health related practical needs. Clinical trials have showed good evaluation of some drugs so far, but clinical response is a wide spectrum yet to be analyzed: CFTR mutations spectrum, costs related to the treatment with new drugs (for VX-770 therapy), variability of CF clinical expression, limitations to test in vitro drugs, absence of good clinical markers to evaluate drug response, absence of long-term studies and with patients below six years old, multidrug treatment used to improve the expression response, and finally, the most important problem, who will benefit from the new drugs therapy, are issues that constitute a barrier that should be overcome. Personalized drug therapy may not be a fiction anymore, but it is not yet a reality for all CF patients.

Cite this article as:

Fernando Augusto de Lima Marson, Carmen Silvia Bertuzzo and Jose Dirceu Ribeiro , Personalized Drug Therapy in Cystic Fibrosis: From Fiction to Reality, Current Drug Targets 2015; 16 (9) . https://dx.doi.org/10.2174/1389450115666141128121118