Leukaemia and bone marrow failure are blood disorders common in children and are caused by the overproduction or underproduction of blood cells.

The blood cancer leukaemia is the most common cancer diagnosed in children. It occurs when the child’s bone marrow becomes overrun by abnormal white blood cells.

Healthy white blood cells normally help to fight infections. In leukaemia, these cells are abnormal because of genetic mutations and don’t fight infection well. Instead, leukaemic cells build up in the bone marrow and crowd out healthy cells. Symptoms of leukaemia are mostly related to the loss of normal blood cells. So, 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: 5-year survival for acute myeloid leukaemia (ALL) in children is 90%. The five-year survival for acute myeloid leukaemia (AML) in children is 77%
  • 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

To determine how to better treat blood disorders developing in children, our blood diseases research team use pluripotent stem cells to study how normal human blood is formed, and how the rare blood stem cells of human bone marrow develop. 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 in the laboratory that have these same mutations we will better understand how these diseases develop, evaluate existing therapies and develop new combinations of drugs to improve treatment options. We also hope to make new, healthy blood stem cells to use as treatments for children with leukaemia and bone marrow failure.

Cord blood stem cell research

Blood from the umbilical cord at birth contains small numbers of blood stem cells. Cord blood can be used as a source of blood stem cell transplantation for some children with leukaemia and other life-threatening blood illnesses.

The BMDI (Bone Marrow Donor Institute) Cord Blood Bank stores cord blood and we run a Cord Blood Stem Cell Research Laboratory within the bank. 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 to 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 cell replacement therapies by generating blood stem cells made in the laboratory that are a perfect match to the child's own tissues.