I have worked for more than 50 years on understanding the causes, consequences and treatment of neutropenia. I began studying the disease cyclic neutropenia because of an interest in helping a patient under my care at the NIH. This disorder also occurs in collie dogs. I studied the human version and the canine version of the disease in parallel with several collaborators. The work evolved from pathophysiological studies to genetic investigations and clinical trials. We discovered cyclic variation in the substance we then called colony-stimulating factor. Our report in Science predicted that this might be the regulatory substance for the production of neutrophils. This was proven to be correct several years later after cloning the gene for granulocyte colony-stimulating factor (G-CSF) by Lawrence M. Souza and his collaborators. When the company Amgen began producing G-CSF for clinical studies, we conducted phase 2 and phase 3 trials here at the Clinical Research Center (CRC), now known as the Translational Research Unit (TRU), proving its efficacy for long-term treatment of neutropenia. We have since conducted several clinical studies at the TRU.
In recent years my research is focused on understanding the genetic and molecular causes for neutropenia, working with several geneticists, molecular biologists and the staff at the Institute for Stem Cell and Regenerative Medicine.
The end goal for our research is to improve care for patients with neutropenia of all causes knowing that we can best do this through international collaborations and careful genetic molecular, physiological and clinical studies.