Doctors sometimes refer to particular drugs as an “active treatment” or “active drug therapy.” Active treatments treat the underlying cause of the bone marrow failure. These drugs are typically approved by the U.S. Food and Drug Administration (FDA) to treat a specific disease. It is important to note that active treatments do not cure the disease. The only cure for a bone marrow failure disease is a bone marrow/stem cell transplant. Unfortunately, a bone marrow transplant is not an option for many people. In addition, it carries many risks and potential long-term side effects.