In animal studies, Children’s Center scientists have been able to prevent the cascade of brain pathology that appears both to cause and signal the final and fatal stages of acute and chronic liver disease in children and adults. The findings (“Hyper-ammonemic Encephalopathy”) which appeared in the May 2002 issue of Medicine, could have major implications for extending the lives of individuals awaiting liver transplants.
Pediatric biochemical geneticist Saul Brusilow, M.D., writes that the chemical methionine sulfoximine (MSO) appears to prevent the brain swelling (encephalopathy) in patients with acute liver failure. It is the swelling, which causes pressure on the patient’s brain, which leads to coma and ultimately death. Brusilow hopes to begin clinical trials of MSO in such patients within a year.
Brusilow is widely regarded for his research on and the treatment of patients with a rare biochemical defect known as urea cycle disorder, which has some of the earmarks of the ammonia toxicity and brain pathology that characterize end-stage acute and chronic liver disease.
The study was funded by the National Institute of Child Health and the National Institute of Neurological Diseases.
(Reprinted with permission from Johns Hopkins Children’s Center News, Summer 2002.)
