Bone marrow failure diseases directly affect the body’s ability to produce new blood cells, weakening the main line of defence and leaving it susceptible to a hoard of other threatening diseases. These rare disorders are often acquired at birth and most commonly seen in young children, but can also be acquired at a later stage in life.
Understanding the landscape
Every important mission begins with a need for understanding. Bone marrow plays a critical role in the body’s health and defence function. The spongy red tissue found within our bones works to produce red blood cells that carry vital oxygen around our body, along with white blood cells that fight infections and kill germs, and platelets that act to stop bleeding.
What is bone marrow failure syndrome?
What are the causes?
Currently the causes of bone marrow failure are unknown. Discovering them is part of our mission. We do know that inherited bone marrow failure is caused by a genetic defect. Genes are the little pieces of information that tell the body what to do or what to make, from the colour of our eyes to how tall we are. Children with inherited bone marrow failure still face the challenge of locating their defective gene before they can begin to search for a cure.
What are the treatments?
Depending on the cause and severity of the disease, treatment for bone marrow failure differs from case to case. The ability to diagnose and treat bone marrow failure as early and rapidly as possible may decrease and relieve symptoms, and help prevent further medical problems.
Without a cure, the next line of defence is ongoing treatment. Blood transfusions involve intravenous drips feeding healthy blood into the body. Medicines such as hematopoietic growth factors help bone marrow produce more blood cells. Immunosuppressive drug therapy prevents the immune system from attacking bone marrow, allowing bone marrow stem cells to grow and, in turn, raise blood counts. Steroids are used to support bone growth and stimulate bone marrow, increasing red blood cell production. Stem cell transplants replace unhealthy bone marrow with healthy cells from a suitable donor.
While these treatments can help, they each come with their own risks – increasingly so in cases of bone marrow transplants. With your support, we can continue to work with research groups to study the causes of rare bone marrow failures and explore new therapies.
Our mission won’t end until we find a cure. Join the cause today: every blood donation or monetary contribution will help save lives.
