Clinical Trials for Rare Blood Diseases (Neal Young, M.D.)

Clinical trials are research that's performed in patients, with the goal of developing good evidence for what causes a disease, what constitutes a disease and most important of all, what can be done to either prevent or to treat a disease. There are many advantages to participating in a clinical trial here at NIH. If you're a patient and especially a patient with a rare disease, there's probably the simple advantage of being seen by doctors who have a large experience with your disease, with your syndrome, with your condition, in contrast to even a great doctor in the community or at an academic center who may only see one case in a year or one case in a lifetime of the disease that affects you. So that's worth a lot. There's always the potential benefit of participating in a trial that's wildly successful in which something new and dramatic has been developed and needs to be tested for the first time in humans. So a patient can benefit from that. So in the hematology branch, because we're interested in severe blood diseases, the people who participate in our clinical trials are suffering from a form of bone marrow failure, sometimes a leukemia, a malignant disease of the bone marrow, and other diseases that have a major impact on their life, in fact, can be life-threatening or fatal. Aplastic anemia is a disease of the bone marrow. The bone marrow's the factory for the blood. And in aplastic anemia, you can think about the factory just shutting down. We've conducted probably hundreds of studies of this disease, mainly with the goal of developing effective therapy. In fact, a very recent study that we did compared two types of treatment that had been used interchangeably for aplastic anemia and we showed that the one of the therapies was much superior to the others. And that had not been suspected at all by the experts in the field of aplastic anemia. It's a very important study that influenced the way patients are taken care of all over the world. I think there's also a satisfaction that comes from being a participant in a clinical trial, even if you're not in the better of the two arms or you suffer a side effect, the knowledge that's gained really is going to help everyone else with that disease. The work that's been done here at NIH has had a major impact on how well patients with this disease do. When I was much younger and in training, the occasional patient that I saw with aplastic anemia was doomed to die within a few months. They were usually sent home to for a few transfusions and we didn't see them again. Now most patients -- the great majority of patients with aplastic anemia can be effectively treated and that's an enormous satisfaction to not only see patients -- main satisfaction is to see a patient you treated 20 years ago come back and have a family and do well, but it's also, from the career point of view, wonderful to be a participant in something that's so substantially helped people, so tangibly helped. Not a promise for the future but something that's really happened, to people that you know.