Gene therapy close to approval in Europe
Gene therapy is on the verge of being approved for usage on patients in Europe, reports the BBC.
The treatment involves altering a person’s DNA so that inherited diseases and conditions can be treated.
The European Medicines Agency has recommended that the therapy be used for a rare genetic disorder which prevents suffers from being able to digest fats properly and the European Commission is set to make a decision on usage.
In theory, gene therapy is a very simple solution to a number of problems. It involves replacing a part of a person’s genetic code with DNA is working well.
However, in reality success has been mixed with one patient in the US, a teenager named Jesse Gelsinger dying during the process and several others developing leukaemia.
Despite this, The European Medicines Agency believes that the treatment will work for people suffering with lipoprotein lipase deficiency, a condition which affects one in a million people.
The condition leads to a build –up of fats in the bloodstream, pain in the abdomen and pancreatitis, which can be life-threatening.
UniQure, manufacturer of the treatment drugs to be used in the trial, described the move as a “major breakthrough”.
"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,” UniQure chief executive officer Jorn Aldag said.
"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."