Blood disorders
Leukaemia and bone marrow failure are blood disorders common in children and are caused by the overproduction or underproduction of blood cells.
Leukaemia is the most common cancer diagnosed in children. It is a blood cancer that occurs when the child’s bone marrow becomes overrun by abnormal white blood cells.
Normally, healthy white blood cells help to fight infections. In leukaemia, abnormal white blood cells no longer fight infection well and leukaemic cells build up in the bone marrow and crowd out healthy cells. For children with leukaemia, this happens because genetic mutations have made the blood cells abnormal.
Symptoms of leukaemia are mostly related to the loss of normal blood cells; insufficient platelets cause bleeding, lack of white cells leads to infections, and too few red cells result in anaemia.
A child with bone marrow failure has an underproduction of normal blood cells and suffers similar symptoms depending on the blood lineages affected. Bone marrow failure is caused by mutations in blood genes or by abnormal immune cell function.
Children with either type of blood disorder can experience fever, weakness, tiredness and bleed or bruise easily. We need new treatment options to ensure more children can survive blood disorders.
Who does it affect?
Who does it affect?
- In Australia about 250 cases of childhood leukaemia are diagnosed each year.
- Five years after diagnosis, only six in 10 patients survive. Note: The five-year survival for acute lymphoblastic leukaemia (ALL) in children is 90 per cent. The five-year survival for acute myeloid leukaemia (AML) in children is 77 per cent.
- Each year about 160 people in Australia (mostly children and adolescents) are diagnosed with bone marrow failure syndromes. Only half survive.
Our Blood disorder research
Our Blood disorder research
Human pluripotent stem cell-based research
Our Blood Development research group is looking for ways to better treat blood disorders in children through the use of human pluripotent stem cells. These stem cells are used to study how normal human blood is formed and how the rare blood stem cells of human bone marrow develop (the ‘factory’ of blood cells in the body). These blood stem cells produce all blood cells, both red and white.
Many blood diseases in children are caused by mutations in blood genes. By making blood cells that have these same mutations in the lab, we will better understand how these diseases develop, evaluate existing therapies, and develop new combinations of drugs to improve treatment options. We also aim to generate a new, healthy blood system from lab-made blood cells to use as treatments for children with leukaemia and bone marrow failure.
Cord blood stem cell research
At birth, blood from the umbilical cord is rich in stem cells that can generate all blood cell types. Cord blood can be used as a source of blood stem cell transplantation for some children with leukaemia, immune disorders and other life-threatening blood illnesses.
Cord blood bank
The BMDI (Bone Marrow Donor Institute) Cord Blood Bank is a partnership between Murdoch Children's Research Institute (MCRI), The Royal Children’s Hospital and the Fight Cancer Foundation. It is one of three public cord blood banks in Australia.
Since 1996, the BMDI Cord Blood Bank has given more than 600 children with blood diseases and other life-threatening illnesses a second chance at life by providing blood stem cells for transplantation. The bank collects donated cord blood from its dedicated collection centre located in the Royal Women’s Hospital and Frances Perry House and stores high-quality cord blood that can be later used for treatment.
Impacts of our research
Impacts of our research
Our laboratories have developed (and continue to improve ways of producing) large numbers of the 'right' blood cells to study blood diseases.
In partnership with the Novo Nordisk Foundation for Stem Cell Medicine, reNEW, we are driving a project to take the blood cells we generate in the laboratory and translate them into a new stem cell-based cellular therapy. This therapy aims to treat children suffering from blood diseases who require a bone marrow transplant.
The BMDI Cord Blood Bank runs a Cord Blood Stem Cell Research Laboratory. Our laboratory is exploring cells in cord blood, other than blood stem cells, that could be used for regenerative and cellular therapies to regrow healthy human cells. These healthy human cells could replace damaged ones, improving outcomes for children with a range of illnesses.
Our vision
Our vision
In the future, for children who lack suitably matched cord blood or bone marrow transplants from donors, we see the possibility of using cell replacement therapies. These therapies would involve generating blood stem cells in the laboratory that are a perfect match to the child's own tissues.
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