Journal of Bioequivalence & Bioavailability

New drug screening strategy for infectious diseases: The power of common sense

2^nd World Congress on Bioavailability & Bioequivalence: Pharmaceutical R & D Summit-2011 and International Conference on Pharmaceutics & Novel Drug Delivery Systems

06-08 June 2011, Las Vegas, USA

Maryse Picher

Scientific Tracks Abstracts: JBB

Abstract:

C linical trials are generally interrupted by poor drug effi cacy tested late in the pipeline. By that time, vast amounts of money and resources have been wasted on a product that will not reach the market, which deters investors away from Biotech/ Pharma. Th e current reaction is to reduce R&D teams to focus on pushing potential sources of income out of the pipeline. Alternatively, we propose to improve clinical trial success by using better in vitro disease models to ?kill? ineffi cient candidates before they enter complex animal protocols and ?metabolites in safety testing? (MIST). Th e development of drug inhalation therapies for cystic fi brosis (CF) is an example of how common sense will revolutionize drug discovery. Th e capacity of a compound to improve airway clearance is routinely tested on cultures of human airway epithelial cells. Th is high-throughput technique has yielded drugs well tolerated in subjects, but which failed to improve airway clearance in CF patients. We demonstrate the critical importance of a screening strategy accounting for the chronic airway infection experienced by most CF patients. Cultures of epithelial cells from CF patients, chronically exposed to their secretions, constitute a more accurate model of the disease, as demonstrated by in vivo immunolocalization of protein markers. Surprisingly, this model revealed that the airway walls functionally express a diff erent set of surface proteins, which may aff ect drug target availability. Th is study illustrates the tremendous potential of this approach to improve the stringency of drug screening, and the success of clinical trials for infectious diseases.

Biography :

Dr. Picher is a medical research scientist specialized on respiratory diseases, with 12 years of experience in drug discovery. As principal investigator at the Cystic Fibrosis/ Pulmonary Research and Treatment Center (NC), she discovered a new signaling pathway regulating airway clearance, currently targeted by Biotech/Pharma. As independent consultant expert for CFRx and Flatley Venture Capital (FVC), she currently provides advices on in vitro models and drug screening protocols, and evaluates the scienti fi c soundness of drugs selected for clinical trials by companies soliciting partnership with FVC. Dr. Picher published 75 scienti fi c documents and is editor-in-chief of a book on drug discovery (Springer, 2011)