William A. Eaton, M.D., Ph.D., NIH Distinguished Investigator

Research Goal

The purpose of our research is to discover new drugs for treating sickle cell disease.

Current Research

Current research is focused entirely on pathophysiology and drug discovery for sickle cell disease, as well as monitoring ex-vivo sickling in sickle cell patients on drug trials, gene therapy trials, and natural history studies in collaboration with NHLBI and NIAID hematologists. A highly-sensitive, pathophysiologically-relevant and high throughput assays have been developed to screen compounds for ant-sickling activity. The assays use laser photolysis or nitrogen deoxygenation to induce sickling and automated image analysis to detect the formation of sickle fibers in individual red cells. As a strategy for the most rapid path to bringing a drug to market, the first phase of the screen is to test all U.S. Food and Drug Administration-approved drugs.

Applying our Research

Hydroxyurea is the only drug that is currently used to treat sickle cell disease, and helps, but does not cure, only about 50 percent of patients. Additional drugs are critically needed.

Need for Further Study

Additional studies are screening large libraries of compounds that have already been given to humans, in order to reduce or eliminate pre-clinical studies.

Research in Plain Language

My research focuses on understanding the steps involved in protein folding — a process that transforms a string of amino acid molecules (protein building blocks) — into a complex, 3-dimensional structure that performs a biological function. This research uses rapidly pulsed laser light and fluorescent labeling of molecules to observe the steps in the protein folding process. My lab also uses laser techniques to search for a drug for the treatment of sickle cell disease.