Meet Researchers Supported by AAMDSIF
AAMDSIF’s named research funds make it possible for young investigators to delve into the biology of bone marrow failure disease and contribute their findings to the body of scientific work that, one day, will add up to a cure. This is the first of a 3-part series on our 2016 grant recipients, and today we’re introducing you to two of them who are both investigating aplastic anemia.
Kate MacNamara, studying at Albany Medical College in New York, proposed a study on the relationship between excessive inflammation and the destruction of stem cells that are required to maintain daily production of all blood cells. Her study, “Macrophages in the Pathogenesis of Aplastic Anemia”, was made possible by generous support from The Julia Malsin Research Fund.
In brief, MacNamara is researching exactly how stem cell function is compromised by inflammatory molecules, something that is not yet fully understood. To answer this open question, she utilized a mouse model of bone marrow failure to investigate the mechanisms involved. “We made the unexpected observation that IFN-gamma signaling in stem cells themselves was not required for the loss of blood stem cells. We identified macrophages, key phagocytic cells of the immune system, as the direct targets of IFN-gamma in driving the decline of stem cells during bone marrow failure.” If you are a patient with aplastic anemia, inherited or acquired, you may want to ask your doctor to explain these principles.
Thanks to a grant from The Arthur Kunofsky Research Fund, Simona Pagliuca at the University of Naples Federico II in Italy is able to undertake her study, “The Impact of Eltrombopag treatment in combination with immunosuppression on immune derangements of aplastic anemia.”
In 2014, the experimental study named RACE was approved in an effort to improve the results of standard therapy by using it in conjunction with another drug called eltrombopag. Researchers hope to answer a simple question: Which is the best treatment for patients with a diagnosis of aplastic anemia? Cyclosporine and anti-thymoglobuline or cyclosporine, anti-thymoglobuline plus eltrombopag? Patients joining this study will be randomly assigned to one of the two groups. Pagliuca’s work also has a laboratory component to research the immune defect existing in patients with aplastic anemia and to define the biological response after each treatment.
Stay tuned for the next two parts of this series, which will address MDS and PNH research.