When met with the term ‘HIV’, the mind, understandably, associates it with the terrible scenes witnessed in the news about the devastating effect HIV/AIDs has had around the world. It is therefore a big leap to then consider the use of such an agent in the treatment, let alone curing, of a disease. Wiskott-Aldrich syndrome (WAS) is a rare recessive genetic disease predominantly affecting males at a prominence of around one in 100,000. It is a disease characterised by eczema, low platelet count and immune deficiency. The immune deficiency is a particularly debilitating symptom that arises from a decrease in body antibodies and a decrease in a particular white blood cell activity. Its severity is variable and mutation dependent bubt can be deadly. However, scientists have been able to cure six boys of their condition following treatment using a tamed HIV.
HIV, as is widely known, is the human immunodeficiency virus associated with further development of AIDs. The virus is therefore best known for causing a considerable detrimental effect to the body, for which there is no cure. The therapy, trialed in Great Ormond Street Hospital, London, and Necker Children’s Hospital in France, involved first removing a sample of the patient’s bone marrow. This was then analysed to find the cells capable of generating the immune system. Once the cells were found, a tamed HIV variety was used to transfer the correction for the mutant gene in WAS.
The trial found that six out of seven boys showed reversed symptoms and reduced hospital time. One child is no longer confined to a wheelchair. Another child, the seventh, unfortunately acquired a resistant strain of the herpes virus as a result of compromised immunity; however, the infection was contracted before the trial was carried out.
Professor Adrian Thrasher, from Great Ormond Street Hospital, said: “I think it is very significant, it is another clear and powerful demonstration that a gene therapy approach is an effective one.
ne consistent problem with gene therapy is that you must overcome the challenge of fixing the genes responsible in every cell that is causing the problem, or at least the majority- circumstances depending. The advantage with the treatment for this disorder is that the cells will continue to self-propagate as well as producing specific cell types, giving rise to a more effective immune system in patients, if not quite as effective as those of the general population.
The world of gene therapy is a vast realm of clinical application potential. With a great deal more diseases becoming more comprehensively studied and understood, numerous new potential gene therapies are being researched for those diseases found to have a genetic abnormality component to them – promising a more prosperous future for single gene disorder-bearing patients.