The Coming Worldwide Epidemic of Neurological Disorders, and What We’re Doing About It

The rapid aging of the population in most developed countries will eventually produce a world in which almost a third of its inhabitants will be over 60 years of age. Coincidentally, 60 is the age at which the incidence of neurodegenerative diseases such as Parkinson’s Disease, Alzheimer’s Disease and stroke begin to rise exponentially. Effective treatments are lacking for these and nearly all of the major neurologic disorders, which, taken together, cost the U.S. over $330 billion per year. We have created a new approach, which we call structural neurology, to bring the techniques of structure-guided drug discovery to bear on a number of these diseases. A highlight of our strategy is the use of what we call pharmacological chaperones to increase the steady-state concentration of neuroprotective proteins – we are not only limited to finding inhibitors of neurotoxic ones. We will give examples of this approach in the treatment of Gaucher Disease and the prevention of Parkinson’s Disease.