Treatment:

Presently there is no way to stop Krabbe disease or cure it once it is in full swing. So for a child who was diagnosed to late, like my son. When signifant symptoms are evident, treatment has been limited to supportive care and drug therapy to control the irritability and spacsticity, this treatment, however, will not prevent loss of nervous system function. They are confined to a wheelchair and limited signs of communication. Research is showing that their cognitive skills remain intact, which means they are fully aware of the loss they are experiencing. With today’s modern technology in the medical system, children are living a lot longer with the assistance of feeding tubes, suction machines and oxygen installed in the home. Death usually occurs from a respiratory infection due to a weak palliative state.

Cord Blood Stem Transplant

The first unrelated cord blood transplant in the United States was performed at Duke University Medical Center in 1993. Since then cord blood transplants have become increasingly common. This procedure has saved lives of more than 1000 patients worldwide. Dr. Johanne Kurtsburg the director of the Pediatric Bone Marrow and Stem Cell Transplant program at Duke is the pioneer in cord blood transplants and helped to revlutionize the procedure. Which she has been working on for over 20 years.

Children with Krabbe disease can be saved and their brain development reserved if they receive stem cells from umbilical cord before symptoms of the disease develop. Now that effective therapy is available for Krabbe infants, Newborn Screening is essential to identify babies at a time they can maximally benefit from treatment. Early diagnosis is imperative. Infants are receiving cord stem transplants as early as 2 – 4 weeks of age have a very high success rate. Time is of the essence in treating these infants before symptoms progress and become irreversible. Delaying a transplant even a week or two can make a noticeable difference in a child’s motor skills.

The transplant procedure is difficult for the child. Children receiving cord stem transplants go through high doses of chemotherapy to destroy the diseased cells. Chemotherapy will also destroy the immune system, so it will not attack the donated cells. After this process new blood forming cells donated from umbilical cord blood, are given, through a central line that goes into a vein in the chest. The transplanted cells move into spaces, inside the bones where they create new marrow. They grow and make healthy new red blood cells. After transplant the immune system are wiped out and the child is extremely susceptible to infection. The immune system will recover after time. The disease can be halted and in some cases repair damage of the disease, and most children live a normal life.

Children transplanted after the onset of symptoms, have a lower success rate. The progression that occurred before transplant can never be corrected. Many factors affect the success of the transplant. Most of asymptotic children still experience a degree of altered gross and fine motor skills.