Gene therapy hope for HIV

The world's largest attempt to use gene therapy to combat the Aids virus has resulted in what scientists are calling a “major advance.” The US clinical trial signals that the technique works and is and safe, scientists said.

Transplanted genes were used to roll back the human immunodeficiency virus (HIV), the virus that causes Aids. When the therapy was tested on 74 patients with HIV, it was shown to be safe and appeared to reduce the effect of the virus on the immune system, according to the report published online by the journal, Nature Medicine.

Existing treatments work fairly well for many people with HIV. But they have toxic side effects. People can also become resistant to them and have to take them for the rest of their life. So gene therapy is being tested as a possible alternative. These latest positive findings now suggest that in theory, one treatment should be enough to replace the need for a lifetime of antiretroviral therapy

Lead researcher, Professor Ronald Mitsuyasu, said the research at The University of California, was the first to come through tightly controlled trials when patients did not know whether they were getting the therapy or a placebo. "Gene therapy has the potential of needing only a one-time or infrequent administration of product,” Prof Mitsuyasu told the BBC. “It would allow the patients to control their own HIV internally without the need for continuous drug therapy.”

Stem cells are cells that pass on their genetic code when they replicate. In this trial, half the group of HIV patients were given blood stem cells containing a key gene, while the other were given a harmless lookalike substance, or placebo. The altered gene encodes a small molecule that, like a spanner thrown into a machine, is intended to block HIV from replicating, or spreading once it infects a cell. Good news came after 100 weeks into the trial: in the gene group, the viral count was significantly lower.

But a lot more work needs to be done, before the therapy can be put to use. Professor Ronald Mitsuyasu told Agence France Presse news agency: "While this treatment is far from being perfected, it is not yet as effective or as complete as current antiretroviral therapy in controlling HIV, the study did show proof of concept that inserting and administering a single anti-HIV gene in the patients' own blood stem cells and giving it back to them could reduce viral replication to some degree when anti-HIV medications are stopped."

The results from this trial, could also give hope for treating cancer and congenital defects where we know that there is a gene that might be replaced or fixed. "It's a positive finding for the field, and should move the field forward,”said prof Mitsuyasu."

SOS Children has grassroots AIDS projects all over Africa, working at a simple level giving practical support to children. Last year SOS Children supported 44,000 AIDS Orphans in their old family home in 44 countries. Read more about SOS AIDS projects and find out how you can help

Written by Hayley Jarvis for SOS Children