NEW YORK (Reuters Health) - The glycogen storage disorder Pompe’s disease responds to treatment with recombinant human alpha-glucosidase, investigators report.
Dr. Ans T. Van der Ploeg and associates previously showed that patients with infantile Pompe’s disease, which causes myopathy and reduced pulmonary function, responded to treatment from recombinant human alpha-glucosidase from rabbit milk (rhAGLU). For their current research, they treated patients with late-onset disease, noting that they represent the largest group of patients affected by Pompe’s disease.
Dr. Van der Ploeg at Erasmus MC-Sophia in Rotterdam, The Netherlands, and associates treated three patients, ages 11, 16 and 32, who had been diagnosed up to 10 years earlier. Patients started IV treatment at 10 mg/kg, which was increased to 20 mg/kg at 12 weeks because of a lack of benefit.
The youngest patient improved the most, the authors report in the April issue of the Annals of Neurology. After 72 weeks of treatment, he no longer required a wheelchair, was playing sports, and muscle strength had normalized.
The 16-year old, although still requiring a wheelchair, improved significantly and was able to decrease daily ventilator treatment from 18 to 10 hours per day. She resumed her education and participates in social life.
The oldest patient, who was tetraplegic at baseline, gained muscle strength so that he was better able to dress himself. He regained the ability to talk on the telephone and was able to stay out of bed for 13 hours a day, up from 3 hours before treatment.
Histological examination revealed more normal muscle morphology in all three individuals, with correction of glycogen storage in peripheral nerves.
The only adverse effect was a mild, transient skin reaction in one patient.
“Our studies advocate more focus on transgenic technology because we have demonstrated a product purified from milk of transgenic animals can be safe and effective for the treatment of human diseases,” Dr. Van der Ploeg and associates conclude.
Source: Ann Neurol 2004;55;495-502. [ Google search on this article ]
MeSH Headings:Behavioral Sciences: Behavioral Disciplines and Activities: Humanities: Philosophy: Quality of Life: Recombinant Proteins: Social Sciences: Anthropology, Education, Sociology and Social Phenomena: Humanities: Psychiatry and PsychologyCopyright © 2002 Reuters Limited. All rights reserved. Republication or redistribution of Reuters content, including by framing or similar means, is expressly prohibited without the prior written consent of Reuters. Reuters shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon. Reuters and the Reuters sphere logo are registered trademarks and trademarks of the Reuters group of companies around the world.
