At Dana-Farber, scientists seek answers to fundamental questions about the causes and development of hematologic diseases, and strive to convert their findings into better treatments for patients.
At the level of cells and their basic processes, researchers are studying the genetic errors that cause blood-making cells to become "stalled" in an immature state — one of the hallmarks of myelodysplasia and other blood-related cancers. The advent of techniques for scanning the activity of thousands of genes at a time has vastly accelerated the pace of this work, yielding a variety of novel approaches to treatment.
Other efforts aim to deepen the scientific understanding of non-cancerous blood diseases such as sickle-cell disease, myelodysplasia (which results from faulty production of certain bone marrow or blood cells), anemia (a drop in the ability of the blood to transport oxygen), and other conditions. Insights into the underlying causes of these disorders are producing clues that may result in new and better therapies.
In the clinic, investigators are leading studies to determine how potential new therapies can be administered in effective and safe ways to patients. The Center is participating in a national study evaluating inhaled nitric oxide gas as an additional measure for treating pain associated with sickle-cell disease. Another study is evaluating whether a cell-free hemoglobin solution can relieve such pain.
Other clinical projects focus on bleeding conditions and abnormal blood clotting. One study is exploring whether hemophilia A patients can benefit from receiving a long-acting blood-clotting compound called Factor VIII. Another is examining ways of reducing thrombocytopenia, a condition in which there are abnormally few platelets in the blood.