Europe’s Committee for Medicinal Products for Human Use approved a gene therapy for a rare genetic disease, the first time a Western regulatory agency has okayed such a treatment, though gene therapies have been approved in China.
The virus-mediated treatment, called Glybera, which is being developed by Amsterdam-based UniQure, introduces a normal version of a gene needed to properly break down fats in the blood. Though the condition is rare, patients with dysfunctional copies of this gene have dramatically increased levels of fat in their blood, which can lead to fatal inflammation of the pancreas. The genetic repair lowers blood fat concentration and reduces the frequency of pancreatitis, according to clinical trial data.
Gene therapy hit a major milestone in 2012 when Glybera was approved by European authorities for the treatment of autosomal recessive lipoprotein lipase (LPL) deficiency. Gene therapy has been a promise in the making for decades, and as such, hopes are high for Glybera. Two other drugs had been approved several years ago in China as gene therapies for cancer, but with only very limited clinical success. We also had an early look at ongoing work to genetically reprogram cardiac cells to form a natural pacemaker. Meanwhile, genetic sequencing costs keep plummeting, and Life Sciences is confident that with its Ion Proton DNA sequencer it will be able to sequence so fast and cheap that it was the first to enter X-Prize 100 over 100 $1000 Genomes Challenge.
The European Medicines Agency took gene therapy a big step closer to normalization this week. The Agency reported on its website that it was recommending approval of the controversial techniques for the treatment of a very rare genetic disorder.
Providing the European Commission take on board this recommendation, and they have always listened to a medical body before, then it will make the first ever regulatory approval of a gene therapy medication, in the West. (A gene therapy medication to treat cancer has approval in China since ‘03). This will mean a metaphorical shot in the arm for a field of study that has for too long labored with too little support and funds.
The basic concept of gene therapy—replacing a defective gene or adding in a functional copy— is straightforward, but the trick is in the delivery. Gene therapies are often carried into a patient’s cells by viruses. Some of the early problems with the therapies were due to strong immune reaction to the transport viruses or cancer sparked by genomic changes induced by the viruses. But in recent years, researchers have found safer viruses and techniques for getting the replacement genes into a patient’s body.