krabbe information
On September 10th 2004, Jackson was diagnosed with Krabbe Leukodystrophy (pronounced "cra-BAY") or Krabbe Disease. This is a rare, genetic disease that affects the myelin coating of the nervous system. Children with Krabbe are unable to produce an enzyme called galactocerebrocidase or GALC. The lack of this enzyme leads to a buildup of toxic proteins in the brain, which causes the myelin coating of their nerves to break down. This process eventually leads to permanent nerve damage in the central and peripheral nervous system.
Untreated, children with Krabbe Disease progressively lose their motor skills - this includes their ability to crawl, sit up, eat, speak, smile, and some lose their eyesight. Research is showing that their cognitive skills remain intact, which means that they are fully aware of the loss they are experiencing. It also means that as long as the child is alive, they are aware of their surroundings. Most children diagnosed with Krabbe Disease die within a few years of diagnosis, although some children have lived longer.
Jackson has a rare, slow-progressing form of Krabbe Disease. Because of this, he was found to be a candidate for a stem cell transplant to halt the progression of the disease. Jackson received his stem cell transplant at Duke University Medical Center. The source of his new cells was a unit of donated umbilical cord blood from a full-term healthy baby.
Stem cells get their name from their ability to develop into three types of blood cells: red blood cells, white blood cells and platelets. In a cord blood transplant, the stem cells from the cord blood grow inside bone marrow, creating a factory for new cells that will provide the critical enzyme that is lacking in Krabbe children. Prior to the transplant, Jackson received near-lethal doses of chemotherapy for eight days to completely wipe out his existing bone marrow. After the transplant, which is given similar to a blood transfusion, his immune system was basically non-existent. During this time he was extremely susceptible to infection and numerous other complications. As he gets further out from the transplant, his immune system recovers over time. He has been followed by his medical team very closely to watch for possible problems related to the transplant.
Though used like a bone marrow transplant as a source of stem cells, cord blood doesn't need to be as closely matched to the patient's tissue type, and it is available quickly, frozen and stored in blood banks. Cord blood units are collected from consenting mothers after delivery of their healthy, full-term baby and poses no risk to either mother or child. Donated umbilical cord blood is not tied up in the controversy surrounding stem cell research.
Please see the following links for more information:
Krabbe Leukodystrophy
General Information:
http://www.ninds.nih.gov/health_and_medical/disorders/krabbe_doc.htmMore complicated but very thorough information:
http://www.emedicine.com/ped/topic2892.htm
Stem Cell Transplants using Umbilical Cord Blood
General Information:
http://www.marrow.org/MEDICAL/cord_blood_transplantation_basic.html
Hunter's Hope Foundation
www.huntershope.org
This organization was founded by former NFL quarterback Jim Kelly. His son Hunter was diagnosed with Krabbe's Disease in 1997. Hunter's Hope Foundation's main focus in recent years is the issue of universal newborn screening, which would test all newborn babies for a panel of inherited diseases including Krabbe. Hunter's Hope also provides funding for research and supports families affected by Krabbe.
Duke University Medical Center
Pediatric Blood & Marrow Transplant Program
http://cancer.duke.edu/PBMT
This is where Jackson is received his stem cell transplant. Dr. Joanne Kurtzberg is the director of the program, which is world-renowned for its success in saving children's lives with stem cell and bone marrow transplants.
The Myelin Project
www.myelin.org
This foundation was started by the Odone family, on whom the movie "Lorenzo's Oil" starring Susan Sarandon and Nick Nolte was based (an excellent movie, by the way). Lorenzo Odone had adrenoleukodystrophy, similar in many ways to Krabbe's. This organization supports research on remyelination and other therapies that may be used to treat Krabbe's in the future. Their purpose is to accelerate the process of research and testing to get the treatments to the patients that need them faster.
National Marrow Donor Program
www.marrow.org
This website gives information on how to donate bone marrow & umbilical cord blood. Please consider registering with the National Bone Marrow Registry to give a true gift of life to someone in need.