Important note: Information in this article was accurate in 1995. The state of the art may have changed since the publication date.
Important note: Information in this article was accurate in 1995. The state of the art may have changed since the publication date.
Reuters NewMedia, Inc. - 19 Oct 1995
Deborah Zabarenko
The success of the experimental therapy on the children with ADA deficiency could pave the way for treatment of other immune system ailments, according to Dr. R. Michael Blaese.
"The ADA treatment will teach us something about how we might go after AIDS or sickle cell," Blaese said in a telephone interview, commenting on an article he co-wrote that appears in Friday's edition of the journal Science.
The article detailed four years of research into the cases of two girls born with faulty ADA genes, which set off a chain reaction that ultimately killed off most of the T-cells that mobilize the body's defense against disease.
With their immune systems severely weakened, babies born with ADA deficiency are prone to chronic and repeated infections and generally die during childhood.
To combat ADA deficiency, researchers extracted some of the sick girls' blood, which included weakened T-cells. These cells were infused with a genetically engineered virus carrying a copy of the normal ADA gene, and after 12 days the infused blood was re-injected back into the girls.
The girls, aged 4 and 9 when the research began, both had repeated ADA infusions and showed marked immune system improvement, even two years after the infusions had ended, the study reported.
"These kids are sort of leading the way in gene therapy," said Blaese, of the National Center for Human Genome Research at the National Institutes of Health. But he cautioned that any progress in gene therapy would be slow.
"Most of us believe that this is going to be a revolution in medicine but it's not going to be an overnight coup ... It's a tiny first step," Blaese said.
He added that such gene therapy would likely have applications for disorders where faulty biological material could be extracted from the body, repaired and re-injected. Both AIDS and sickle cell anemia fit this model, he said.
For this reason, gene therapy is unlikely to target other genetically linked disorders such as cystic fibrosis, which attacks the lungs, or multiple sclerosis, which affects the nervous system, Blaese said, because the out-of-body work required would be impossible with current technology.
951019
RE951013
Copyright © 1995 - Reuters, Ltd. All rights reserved. Republication or redistribution of Reuters content 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. Contact Reuters.
AEGiS is a 501(c)3, not-for-profit, tax-exempt, educational corporation. AEGiS is made possible through unrestricted funding from Broadway Cares/Equity Fights AIDS, Elton John AIDS Foundation, the National Library of Medicine, Pacific Life Foundation and donations from users like you.
Always watch for outdated information. This article first appeared in 1995. This material is designed to support, not replace, the relationship that exists between you and your doctor.
AEGiS presents published material, reprinted with permission and neither endorses nor opposes any material. All information contained on this website, including information relating to health conditions, products, and treatments, is for informational purposes only. It is often presented in summary or aggregate form. It is not meant to be a substitute for the advice provided by your own physician or other medical professionals. Always discuss treatment options with a doctor who specializes in treating HIV.
Copyright ©1980, 1995. AEGiS. All materials appearing on AEGiS are protected by copyright as a collective work or compilation under U.S. copyright and other laws and are the property of AEGiS, or the party credited as the provider of the content. .