What is the Treatment of Krabbe Disease?

By  ,  Onlymyhealth editorial team
Oct 08, 2012

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Quick Bites

  • There is no cure for an already existing Krabbe disease.
  • Stem cell transplantation can help if provided before disease's onset.
  • Infants usually die by the time they turn 2 years old.
  • Drugs may be administered for muscle spasticity.

Krabbe disease refers to an inherited disorder that causes destruction to myelin of the nerve cells. Myelin is a protective layer on the cells inside the brain as well as throughout the nervous system. In most cases of these diseases, the signs and symptoms develop in the babies before they turn 6 months old. In these babies, the disease usually results in death by the age of two. When krabbe disease develops in adolescents and adults, its course varies greatly.
Statistic shows that krabbe disease affects 1 in 100,000 people in the United States and it is also called globoid cell leukodystrophy.

Krabbe disease

Treatment Options

For those infants who have already been diagnosed with the disease, there really is no treatment that can effectively change the course of the disease. In such a case, all the treatment that is provided is in the form of supportive care and managing symptoms. Some of the interventions in an already existing disease may include:

•    anticonvulsant medications for managing the seizures
•    physical therapy to minimise the deterioration of muscle tone
•    drugs for easing muscle spasticity as well as irritability
•    nutritional support in the form of a tube that delivers fluids and nutrients directly to the gastric tube or stomach.

Some interventions that are used for older children and adults who have less severe form of the disease may include:

•    Occupational therapy for achieving as much independence as possible while performing daily activities
•    Physical therapy for minimizing muscle tone deterioration.

Stem cell transplantation

Stem Cell Transplantation

Hematopoietic stem cells refer to specialised cells that develop into different types of blood cells inside the body. These stem cells are a good source of microglia i.e. specialised debris-eating cells that reside in the nervous system. When an infant, adolescent or adult is suffering from krabbe disease, the microglia get converted or transformed into toxic globoid cells.

When stem cell transplantation is being done, the donor’s stem cells get delivered into the bloodstream of the recipient through a tube that is called a central venous catheter. The stem cells of the donor help the body to produce healthy microglia, which can populate the nervous system and help in delivering healthy, functioning GALC enzymes. Stem cell transplantation can help in restoring a certain level of normal myelin production as well as maintenance.

This treatment can bring fruitful outcome in infants if it is administered before the onset of the symptoms of krabbe disease.


Read more articles on Krabbe Disease.

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