Are great breakthroughs in medicine just around the corner?
In medicine we ve done our best therapeutically when we have understood in great detail the underlying pathogenesis of disease. So I am optimistic that this greater understanding of human disease will ultimately translate into improved therapies. The timing and the pathway to achieving new treatments, however, are much harder to predict. In some cases we may readily identify new targets that are amenable to development of small-molecule agonists or antagonists. In other cases we may find new proteins that can very quickly lead to the development of new therapies. An example of that would be some of the growth factors for the hematopoietic lineage that are already in clinical use. That said, it will not always be the case that understanding the biology of a disease can be translated quickly into a treatment. A good example would be sickle cell anemia, where we ve understood the molecular basis of the disease since 1953 but have yet to have a cure for the large majority of affected patie
