Important note: Information in this article was accurate in 2000. The state of the art may have changed since the publication date.
PRNewswire - December 8, 2000
"Artificial genes have been developed which block HIV-1, the AIDS virus, from growing in human cells," said Kohn, director of the John Connell Gene Therapy Program at The Childrens Hospital, Los Angeles (CHLA). "In this study, we will be trying to put one of these anti-HIV genes into the bone marrow stem cells of children with AIDS." Stem cells are the 'mother' cells in bone marrow, which make all of the blood cells, including T cells, which are the target for HIV-1 infection. Loss of T cells by HIV-1 mediated destruction leads to the progressive immune deficiency, which underlies AIDS.
"Advances in anti-HIV drug therapy have dramatically altered the nature of the epidemic for children in the U.S.," said Church, director of the Pediatric AIDS Program at CHLA, and the division head of Allergy and Immunology.
"Transmission of HIV from infected mothers to their newborn infants has been dramatically reduced, and HIV-infected individuals are living longer, more normal lives. Unfortunately, current HIV treatment regimens have serious limitations: the virus is not eradicated so patients are not 'cured', drug resistance frequently develops, treatment cocktails come with complex dosing instructions, and are often unpalatable, and toxicities are common," said Church, who also is a professor of Pediatrics at USC's Keck School of Medicine.
The study will examine children between the ages of 3 and 13 with HIV-1 infection who are currently being treated at CHLA. Samples of their bone marrow will be collected while they are under general anesthesia. The bone marrow will be processed in the laboratory to isolate the stem cells. Then, a disabled mouse virus will be used to carry the anti-HIV-1 gene into the stem cells while they are grown in sterile cell culture. Five days after the stem cells are collected from the children; they will be given back to them through an intravenous line. Follow-up blood tests will be performed over two years to look for evidence of successful insertion of the anti-HIV-1 gene into stem cells, which form blood T cells, and for side effects from the procedure.
"It must be strongly emphasized that this is an experimental research study, not a known treatment," said Kohn, who also is a professor of Pediatrics and Microbiology at the Keck School of Medicine at USC. "As a Phase 1 study, the primary objective is to evaluate the safety of performing the gene transfer procedure in children with HIV-1 infection." Secondary goals of the study will be to determine whether the anti-HIV-1 gene can be successfully transferred into the patient's stem cells and whether that leads to protection of T lymphocytes from being eliminated by HIV-1.
According to Kohn, development of effective gene therapy has been making gradual progress as greater understanding of genes and stem cells has been attained from laboratory studies. "If safe and effective methods for transferring genes into stem cells are produced, it may be possible to use gene therapy to treat sickle cell anemia, and other genetic blood diseases such as 'bubble baby disease' where children are born without protective immune systems," Kohn said.
Co-investigators on the study include Drs. Steven King and Keith Bishop at the University of Michigan, where the anti-HIV gene vector was developed and produced.
Visit our website: http://www.ChildrensHospitalLA.org
SOURCE The Childrens Hospital, Los Angeles Web Site: http://www.ChildrensHospitalLA.org
001208
PR001211
Copyright © 2000 - PRNewswire. All rights reserved. Reproduced with permission. Reproduction of this article (other than one copy for personal reference) must be cleared through PRNewswire, Permissions, 810 Seventh Ave., 32nd Floor, New York, NY 10019 http://www.prnewswire.com.
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, National Library of Medicine, and donations from users like you.
Always watch for outdated information. This article first appeared in 2000. 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, 2000. 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. .