'Bionanotechnology from Theory to Practice' is a short online, course providing an interdisciplinary and up-to-date overview of the rapidly developing area of bionanotechnology
Page URL: https://www.bionews.org.uk/page_93572

HIV gene therapy is safe and long-lasting, but is it effective?

8 May 2012
Appeared in BioNews 655

HIV (human immunodeficiency virus) patients treated over a decade ago with genetically modified immune cells have suffered no serious side effects from the treatment.

Although the cells may not have been successful in targeting and killing HIV-infected cells, which they were designed to do, the results are promising as they bolster the safety credentials of gene therapy for clinical use.

Patients enrolled in three clinical trials were monitored for up to 16 years. Each patient received injections of their own T cells, a type of immune cell, modified to identify and kill HIV-infected cells in the body.

Over a decade after the treatment, called adoptive T cell transfer, the modified T cells were still circulating in the blood stream.

'We have 43 patients and they are all healthy', confirmed senior author Professor Carl June of the University of Pennsylvania. 'And out of those, 41 patients show long term persistence of the modified T cells in their bodies'.

The study highlights the potential of gene therapy as a long-lasting alternative to the costly lifetime course of anti-retroviral drug therapy currently used in HIV.

'Just think about what an HIV patient has to do: take drugs every day for the rest of their life, and the minute a person stops taking them, the virus starts coming back', said Dr John Rossi of the Beckman Research Institute in California, who was not involved with the study.

Most importantly, there was no evidence that the modified T cells caused cancer. Previous trials of adoptive T cell transfer had resulted in the genes being inserted in the wrong place and causing out-of-control, cancerous cell growth.

However, the study, published in Science Translational Medicine, did not indicate that the therapy was active against HIV. 'It may not have worked at all', summarised co-author Dr Frederic Bushman.

Using a baseball analogy, Dr Pablo Tebas, director of the AIDS Clinical Trials Unit at the University of Pennsylvania, told NPR: 'We're not hitting a home run. This is a single'.

In further encouraging news, a potential stem cell therapy against HIV was safe and effective in animal studies. Researchers at the University of California tested a technique which involves replacing the immune system with stem cells genetically engineered with a triple combination of HIV-resistant genes.

The team is currently seeking funding and regulatory approval to test the technique in clinical trials.
A Step Forward For Gene Therapy To Treat HIV
Shots (NPR Health Blog) |  2 May 2012
Decade-Long Safety and Function of Retroviral-Modified Chimeric Antigen Receptor T Cells
Science Translational Medicine |  2 May 2012
Generation of an HIV-1-Resistant Immune System with CD34+ Hematopoietic Stem Cells Transduced with a Triple-Combination Anti-HIV Lentiviral Vector
Journal of Virology |  03/12
Gene therapy for HIV safe, but effectiveness still unclear
CBS |  3 May 2012
Gene therapy safe, HIV study says
Boston Globe |  2 May 2012
Genetically modified T cell therapy shown to be safe, lasting in decade-long study of HIV patients
EurekAlert! |  2 May 2012
Stem Cell Therapy Shows Promise in Fight Against HIV
Science Daily |  2 May 2012
10 March 2014 - by Dr Lanay Griessner 
A potential HIV therapy where patients have their blood cells genetically modified to help them resist the virus is safe and promising as a treatment, say researchers after a small clinical trial...
8 July 2013 - by Dr James Heather 
Two HIV patients that received bone marrow transplants have been able to stop taking their anti-viral drugs without any detectable reappearance of the virus, scientists have announced....
8 April 2013 - by Matthew Thomas 
For the first time, scientists have observed how the human immune system makes antibodies capable of neutralising the human immunodeficiency virus (HIV), confirming the belief that their production occurs only after the virus has started to diversify....
28 January 2013 - by Matthew Thomas 
Altering the genetic makeup of immune cells could provide them with resistance to HIV...
5 November 2012 - by Dr Lucy Spain 
The European Medicines Agency (EMA) has approved Glybera, a gene therapy to treat a rare genetic condition, lipoprotein lipase deficiency (LPLD), for sale across Europe. Glybera is the first gene therapy to be approved for the Western market and offers the first therapeutic treatment for people with LPLD....
5 December 2011 - by Dr Tamara Hirsch 
US scientists have induced long-lasting HIV protection in mice from a single injection. Their study, published in the journal Nature, uses gene therapy to stimulate production of antibodies against the virus...
26 September 2011 - by Dr Louisa Petchey 
The success of a new gene therapy trial represents a significant step towards a 'functional cure' for HIV, US researchers announced this week. By mimicking the effects of a naturally occurring gene mutation that makes an individual resistant to infection, this therapy aims to reduce or eliminate the dependency of HIV patients on antiretroviral drugs....
7 March 2011 - by Dr Lux Fatimathas 
American researchers have successfully created immune cells resistant to HIV. T cells, which are the main target of HIV, were isolated from six HIV positive patients and genetically manipulated to confer resistance. The cells were injected back into the same patients and were able to survive and multiply...
21 June 2010 - by Dr Lux Fatimathas 
A human RNA-based gene therapy trial to combat HIV has passed the first safety test. US researchers modified human blood stem cells to make them resistant to the virus....
to add a Comment.

By posting a comment you agree to abide by the BioNews terms and conditions

Syndicate this story - click here to enquire about using this story.