Umbilical Cord Blood and Krabbe Disease

A study published by the University of Pittsburgh School of Medicine suggests cord blood could extend the lives of children with Krabbe disease.

A study published by the University of Pittsburgh School of Medicine suggests cord blood could extend the lives of children with Krabbe disease.

Krabbe’s disease is a rare and usually lethal disease affecting the nervous system. It is a hereditary genetic disease that is transmitted from one generation to the next. Those affected by Krabbe’s disease are not able to produce enough galactosilceramidase (GALC), an essential precursor to myelin, which the body uses to wrap and protect nerve fibres. Without this protection, brain cells die and the brain and peripheral nerves are unable to function properly.

Krabbe’s disease is mainly found in children, but it can also develop later. Unfortunately, at the moment there is no cure for Krabbe’s disease and most of the children affected by this disease do not reach 2 years of age.

Research, led by Dr Maria Escolar, has discovered two procedures that could affect the progression of Krabbe’s disease and not just cure its symptoms: bone marrow transplantation and cord blood transplantation. The study shows that cord blood transfusions not only improve life expectancy for patients, but also increase quality of life. During these procedures, the person affected by Krabbe’s disease receives cells from a healthy person. The new cells are able to produce GALC, which the patient cannot produce on his own.

https://www.nejm.org/doi/full/10.1056/NEJMoa042604