Thank you most sincerely for your decision to support our efforts in hematology. As we have discussed, my group focuses on problems as presented both by individual patients and by patient populations. The underlying force behind our approach is to study a small number of individuals in great detail in order to better understand a problem. This information is then used to apply to the broader issues we face in day-to-day management of patients with hematologic disease.
Recently we have directed a large part of our effort to the development of a facile test for the circulating level of an enzyme called ADAMTS13. Patients with the rare disease called TTP (Thrombotic Thrombocytopenic Purpura) present one of the true emergencies of our specialty. These patients can die before treatment can be started, and even in the best of settings we are unable to save about one person in seven. We hope and expect that having an assay for ADAMTS13 available will help us recognize those patients in whom an early escalation in therapy would lead to a better outcome. We expect a great deal of our effort to be focused on this problem for the next few years.
A second function of my laboratory is to act as a crucible for the development of critical thinking skills in our fellows in training. This is a most important function since it develops the next generation of practitioners in the field. There is no better way to develop critical thinking skills than to be immersed in a research project. This is the approach taken with our fellows in training, and it has been eminently successful. The Special Hematology Laboratory is the center of a wide range of studies by our Fellows. Recent work has ranged widely. Apolipoprotein-E genotypes in chemotherapy induced cognitive failure, warfarin resistance and mutations in the VKORC1 gene, the genetic basis of alpha-2-plasmin inhibitor deficiency, mutation in the erythropoietin gene and the development of a highly sensitive assay for ADAMTS13 activity in plasma.
Two other very interesting projects await our attention. The first is the implementation of a simple test which discriminates between benign and malignant forms of high platelet count. This distinction is critical since it guides our intervention strategy. The second deals with the risk of developing leukemia after high dose radiation therapy for a malignant tumor. For both of these projects the science is strong, what we need is sufficient seed funding to bring them forward to active research projects.
Many of these projects could, with further development, lead to the submission of competitive applications for NIH funding. So I see a great opportunity for leveraging your fund raising efforts by obtaining major funding from the National Institutes of Health.
Again I thank you for your support and I make myself available to meet with you or any donor at any time to answer any questions.
John Owen MD MBA