While it may sound invasive, gene therapies, used to alter a person’s DNA usually in the case of hereditary diseases, will be shortly approved for sale and prescription in Europe - which has been described as a ‘landmark moment’ for the field of gene therapy.
The decision to approve the use of gene therapy came as the European Medicines Agency recommended that gene therapy be approved in the case of those that suffer from lipoprotein lipase deficiency, a genetic glitch which does not allow those who suffer from it to properly digest fats. The approval for gene therapy is now pending with the European Commission, which has final said so with regards to the matter.
Gene therapies in themselves are quite tricky in that while they may be able to "correct" defective or flawed genes, there is a possibility of serious side effects, as in one gene therapy trial, one patient died, while others developed leukaemia. At present, gene therapies are mostly experimental and only available from research labs in the US and Europe, although China was the first country to approve gene therapy.
Lipoprotein lipase deficiency is a rare genetic disorder with around one in a million people having the genetic deficiency. Because they are unable to properly process fats, this leads to a build up of the substance, which can lead to lipids building up in the blood stream, abdominal pain as well as the inflammation of the pancreas which could lead to pancreatitis.
The gene therapy medicine that is up for approval is known as Glybera and what it does is infect the muscles of patients with "working copies" of the fat-processing genes to start producing them. It is being recommended to those patients who have severe cases of pancreatitis and those that cannot manage their disease simply through low fat diets.
Glybera is manufactured by the pharmaceutical company, UniQure, which said that the European Medicines Agency recommendation was a “major breakthrough" for medicine as well as sufferers of lipoprotein lipase deficiency.
Speaking about this, CEO Jorn Aldag said, "Now, for the first time, a treatment exists for patients (with lipoprotein lipase deficiency) that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection,” adding that Glybera helped to restore the “body's natural ability to break down fat particles in the blood,” and that the whole intention of gene therapy was to cure diseases at the “genetic level.”