Reports this week have detailed how gene therapy has been successfully used to treat SCID (severe combined immunodeficiency disorder) in a baby.
The case study involved Nina, the child of Graeme and Aga Warnell, who had become increasingly ill from birth due to her lack of an immune system. It has seen Nina develop an immune system of a healthy new born baby with continuing improvements in her health.
Gene therapy is a method of replacing a ÔÇÿfaultyÔÇÖ gene with a healthy one by using a vector (usually a virus). Conditions called ADA SCID were first treated with gene therapy over 20 years ago, however since then it has been a rocky road with success being overshadowed by failure in the beginning. For example, 4 patients who were treated with the therapy developed Leukaemia 2 years later, and an 18 year old patient died from a liver condition that developed due to a reaction to the virus used to insert the healthy gene.
Since then, gene therapy has been used to treat conditions ranging from head and neck cancer or inherited eye diseases to haemophilia B. Treating SCID in this way is a growing success.There are 3 more children as well as Nina who have received the treatment and all are said to be doing well. Now it seems there are more successes than failures, and scientists such as Inder Verma of the Salk Institute, California, hope that gene therapy will soon become ÔÇ£a more routine modality of medicineÔÇØ.
Of course, there are still health risks to the therapy, mainly posed by the mode in which the healthy gene is inserted, as the viruses can trigger cancer and other conditions. However, successful improvements in this area have been made, and continue to develop, to make the therapy safer.
700 gene trials are currently ongoing, which should soon show worldwide success. It is hoped that gene therapy will soon be able to help more and more children just like Nina.
Bronwen Weatherby
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