European Commission set to approve first drug based on gene therapy

The European Commission is set to approve what would be the first drug based on gene therapy globally. The idea was conceived in the 1970s but after that, a large number of clinical trials did not result in a commercial drug. Recent good results have, however, created a sense of optimism. On July 20, the European Medicine Agency recommended the use of gene therapy for treating lipoprotein (lipase) deficiency, which leads to pancreatitis. European Union normally follows this advice.

What is gene therapy?

Gene therapy treats genetic disorders by tinkering with genes, which can be in three ways: replacing the defective gene, knocking out a gene that is malfunctioning, or introducing a new gene.

What is its promise?

In principle, it can be used for treating or curing any disease with a genetic component. At the moment, it is used in experiments to treat diseases like Leber’s Congenital Amaurosis (an eye disease), some kinds of leukaemia, Parkinson’s disease and a few others.

How does it work?

Gene therapies use viruses mostly to deliver the gene to the target cell. Viruses have evolved mechanisms to deliver their own genes to foreign cells, and scientists use this method to deliver a target gene.

Why has it failed so far?

Gene therapy has failed because many reasons. The benefits have been shortlived in some cases, and sometimes viruses used for delivery become toxic. In one case, a child developed leukaemia. The immune system of patients often attacks the new gene like happens with a foreign body.