In addition to bone marrow transplants (the preferred method of treatment), supportive treatments and immune system suppression, advancements are being made in studies of the immune system to treat aplastic anemia.
Researchers are working to improve the drugs used for immunosuppression and stem cell transplantation. The goal is to find treatments that have fewer side effects than the present treatments with better results.
Studies Into Acquired (or Secondary) Aplastic Anemia
One drug under study is called daclizumab (Zenapax®). This is a monoclonal antibody (a laboratory-made antibody that attaches to specific substances) that blocks the action of interleukin-2 (IL-2). IL-2 is a normal part of the immune system that stimulates the immune response. Blocking IL-2 suppresses the immune system in a way different from ATG. Allergic reactions to this drug are rare. It is currently being used as a part of stem cell transplants, and is under study as a treatment for aplastic anemia.
Stem Cell Research To Treat Aplastic Anemia
Doctors are also trying to make stem cell transplants safer and more available. One approach is to use stem cells that come from the umbilical cord blood of newborns. This is a very rich source of stem cells. Efforts are being made nationwide to develop storage facilities for cord blood. The advantage of cord blood stem cells is that they may lead to less graft-versus-host disease. The disadvantage is that there may be too few cells to successfully "take." In one case, doctors have overcome this by transplanting cord blood stem cells from 2 separate donors. This has proven successful and may have an even lower risk of graft versus host disease. This method is currently under study.
Research for Inherited Aplastic Anemia
Researchers are also studying the possibility of using gene therapy to treat some inherited forms of aplastic anemia. Another approach is to give a drug called amifostine to help protect the bone marrow in children with Fanconi anemia.
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