“Genomic Data in Clinical Trials: It is Inevitable,” was the title of a presentation by Boris Umylny, Ph.D., from Japan Bioinformatics.
Although utilization of genomic information in drug development and clinical trials has been slow to take hold, Dr. Umylny believes that in the near future it will play a greater role in predicting adverse reactions, rescuing failed products, and contributing to the design of adaptive clinical trials.
Driving increased utilization of genomic data are continuing improvements in data-generation techniques that are leading to the $1,000 personal genome, healthcare budget pressures and a move toward outcome-dependent pricing, and FDA support for the collection of genomic data during clinical trials. The evolution toward personalized medicine will require access to personalized genomic information, noted Dr. Umylny.
He predicted that one of the main applications of genomic data in clinical trials will for modeling and simulation of pharmacokinetic and pharmacodynamic studies. A key technology challenge is developing the software capability to apply genomic information and the millions of biomarkers involved. Enhancing software capabilities can rely on the purchase of commercial products or accessing public domain options.
In his presentation “New Methods of Genetic Manipulation and Their Role in Drug Discovery,” Eric Ostertag, M.D., Ph.D., president and CEO of Transposagen Biopharmaceuticals, proposed a solution to the high failure rate plaguing therapeutic candidates in late-stage development. He described the advantages of working with new rat models that more closely represent human physiology, size, and toxicologic responses, compared to traditional mouse models of disease.
Dr. Ostertag summarized the main approaches for producing genetic alterations in rats—mutagenesis, transgenesis, and the creation of humanized animal models. Among the techniques for mutagenesis is mobile DNA technology, using DNA transposons such as piggyBac or Sleeping Beauty to generate insertional mutations.