The European Commission is close to approving a form of genetic therapy for people who are unable to properly digest fats. It would be the first time such a therapy has been approved.
Gene therapy replaces part of a patient’s genetic code that is producing an undesirable condition with a healthier code. But the practice is not so simple. An American teenager died during one treatment while other patients have developed leukemia.
The therapy under consideration uses a virus to infect muscle cells with a working copy of the faulty gene.
UniQure chief executive officer Jorn Aldag said: “Patients with lipoprotein lipase deficiency are afraid of eating a normal meal because it can lead to acute and extremely painful inflammation of the pancreas, often resulting in a visit to intensive care.
“Now, for the first time, a treatment exists for these patients that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection.
“Restoring the body’s natural ability to break down fat particles in the blood, in order to prevent pancreatitis and excruciating abdominal pain suffered by patients, is what gene therapy is all about: curing disease at the genetic level.”