Goghy Stokin, M.D.
The Center’s drug development program involves design, synthesis, and characterization of molecules that are potential treatments for a variety of neurological disorders, most of which are presently intractable to treatment. To accomplish this, we have assembled a group of chemists who are able to synthesize molecules in sufficient quantities to allow us to characterize their potential utility as drug candidates. This is the first step in an elaborate process since the delivery of a drug to the brain is more complicated than the delivery of a drug to other organs because of the existence of the blood-brain barrier. Accordingly, at the outset, two key drug properties need to be evaluated: the ability of our molecules to survive breakdown in the liver upon oral administration and the ability of these to cross from the blood to the brain in sufficient quantities to be effective. The vast majority of drug candidates we design do not pass these tests, and when they do, it is essential to determine that they are “druggable.” To us, druggable means that a convenient dose can be determined, and when attained, this proves to be safe and effective. Over the last few years, the Center has evaluated approximately 20 such molecules, a few of which are promising drug candidates. For the money invested, the Center’s track record of developing CNS therapeutics in collaboration with pharmaceutical companies is unprecedented.