Treatment of Krabbe Disease with Umbilical Cord Stem Cells

krabbe diseaseRecently, some states have officially approved the second week of September as “Krabbe Disease Awareness Week.” In Ohio, the designation was approved effective October 15, 2015 in honor of Madison Layton who passed away after battling the disease from birth to her second birthday.

What is Krabbe Disease?

Krabbe (pronounced KRAH-buh) disease is an inherited disorder, also known as globoid cell leukodystrophy. It is a progressive neurological deterioration that often causes death in early childhood. It is not currently tested for during standard newborn screenings. With Krabbe disease, the protective coating (myelin) of nerve cells in the brain and nervous system is destroyed. According to the Mayo Clinic, it affects about 1 in 100,000 people in the United States. Symptoms, which typically begin to show around six months, include feeding difficulties, unexplained fever, a decline in alertness, muscle spasms, and loss of head control.

Treatment with Cord Blood Stem Cells

A study of treatment using umbilical cord blood stem cells for infants with inherited Krabbe disease was conducted in 2005 and reported in the New England Journal of Medicine. This study showed positive outcomes for 100% of the infants who had umbilical cord stem cells implanted before they began showing symptoms of the disease. According to the study, “Infants who underwent transplantation before the development of symptoms showed progressive central myelination and continued gains in developmental skills, and most had age-appropriate cognitive function and receptive language skills, but a few had mild-to-moderate delays in expressive language and mild-to-severe delays in gross motor function.” For infants who were already symptomatic before they received Krabbe disease treatment, the survival rate was 43%.

Since the initial studies, Cord blood has become a standard treatment option for Krabbe disease if caught pre-symptom. It may also be beneficial in older cases if the symptoms are mild. In either case, the treatment uses allogeneic hematopoietic stem cell transplantation (HSCT), meaning the cord blood would need to come from a healthy sibling or matched unrelated donor.

Early Diagnosis is Important

If you have a family history of Krabbe Disease, and you’re pregnant or planning to be, it is especially important to discuss this with your health care providers as soon as possible. Banking cord blood from each of your children provides the opportunity for them to help a sibling who may later be born with Krabbe Disease or another disorder helped by allogeneic HCST. Also, since cord blood treatment for Krabbe Disease will be more successful before symptoms start to show, early intervention is key.

Are you pregnant and looking for information on cord blood banking or health and wellness? Subscribe to our email newsletter or connect with us on Facebook.

krabbe disease