Media note: Embargoed by the Pediatric Academic Societies of America until 3:15 p.m. EDT Saturday, May 2.
U OF MINNESOTA TREATMENT SHOWS PROMISE AGAINST A DEADLY CHILDHOOD BRAIN DISEASE
Treatments developed at the University of Minnesota have shown promise in halting or even reversing progress of a rare brain disease, according to the researchers who devised the treatments. Dr. Charles Peters, associate professor of pediatrics and lead researcher on the project, will present the results of this study at the annual meeting of the Pediatric Academic Societies of America May 2 in New Orleans. Childhood Onset Cerebral Adrenoleukodystrophy (COCALD) is a deadly neurological disease affecting boys between 7 and 12. Passed genetically from mother to son, it destroys the physical and mental functioning of its victims and kills them within a few months or years. One of every 75,000 boys is diagnosed with the disease. A boy might first show signs of COCALD by experiencing difficulty in listening or paying attention in school. His grades may slip, and he might find it hard to walk or raise his hand. Gradually, he will lose his sight, hearing and ability to speak. If untreated, he will probably die before he reaches high school. Since 1981, physicians at the University of Minnesota and around the world have treated more than 100 boys for COCALD using bone marrow transplants. While the procedure is not a guarantee of survival, a transplant successfully halts the progression of the disease in boys with relatively minor impairment in their cognitive and physical abilities. Out of 46 survivors who could be evaluated, 34 are now stable or improved in their functioning. Although transplant results are good for boys without severe impairments, researchers have found that a transplant alone is useless in treating those with severely diminished functioning. Following their transplants, these boys typically progressed into a vegetative state and died. These days, the outlook is brighter for patients with advanced COCALD. A new transplant preparative regimen developed by Peters and fellow University of Minnesota pediatric researchers Drs. Kathryn Dusenbury, Elsa Shapiro, Richard Ziegler, John Wagner and William Krivit has shown promise in halting progression of the disease in those most severely affected. Through a special sequence of radiation and chemotherapy prior to the transplant, the patient's own blood cells are killed and the immune system suppressed to allow acceptance of the new blood cells. A new procedure developed by Dusenbery protects the brain against radiation while at the same time allowing the rest of the patient's body to be treated. "In the severe form of ALD, we consider it a success if we can stop the progression of the disease process and thus save the boy's life," said Peters. "With this treatment, boys with severe impairment are doing things that are unprecedented in the history of the disease." Up to 17 months after receiving the preparative regimen and a transplant, six boys with moderate to advanced COCALD have stabilized in their functioning. This is in marked contrast to boys with moderate to advanced COCALD who did not have this preparative regimen before their transplant, who deteriorated rapidly. If the boys who received the regimen follow their less severely impaired counterparts, the outlook is promising. Boys less severely affected by the disease have often returned to regular school, regained some of their lost intellectual and physical abilities and gone on to enjoy an excellent quality of life.
"Long-term follow-up is needed for clinical researchers to document the benefits into adulthood," Peters said. Other academic health centers have replicated the treatment with similar positive results.
Contacts:
Dr. Charles Peters, associate professor of pediatrics,
(612) 624-5407 in Minneapolis (April 27-29)
(504) 670-8502 in New Orleans (April 30 to May 5)
Teri Charest, Academic Health Center Communications, (612) 624-4604 4/27/98
News releases also on WWW at http://www.umn.edu/urelate/news.html
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