Creating Human Tissues to Test Drugs for Rare Diseases

Marshall L. Summar, MD: I am Marshall Summar, Chief of Genetics and Metabolism at Children's National Medical Center in Washington, DC, for Medscape Rare Diseases.

I am at the National Organization for Rare Diseases (NORD) Orphan Products Breakthrough Summit with Jim Powers, the CEO of HemoShear (Charlottesville, Virginia). Jim's company is leading some of the technological revolutions in rare diseases, and we are going to talk about what is going on.

In dealing with rare diseases, we don't have a lot of patients for clinical trials and clinical studies, and one of the holy grails of genetics has been finding tissue models that are relevant to study. Could you tell us about the technology that your company is developing, and how it will help?

James C. Powers: It will probably help for me to describe what our basic technology is, and then talk about the rare diseases applications. Our company creates human tissue systems that accurately replicate human diseases in the laboratory. When you think about some of the technological innovations that have happened over the years—high-throughput screening, combinatorial chemistry, analyzing the human genome—we still lack systems in the laboratory that accurately replicate human disease and that can predict whether a new drug candidate will work in a human.