Post date: Aug 08, 2012 1:49:19 PM
The first gene therapy drug approval given by European regulators is potentially good news for those suffering from a rare syndrome which prevents their body from breaking down fat. The decision by the European Medicines Agency to allow Dutch biotech company uniQure to produce a gene therapy for LPLD sufferers opens the way for further developments in gene therapy treatments. Jim Drury reports.
REUTERS AND UNIQURE HANDOUT - Mother-of-three Jill Prawer has suffered with Lipoprotein Lipase Deficiency since she was born.
LPLD is an incredibly rare disorder which prevents sufferers from breaking down fat in their bodies. Only one-in-a-million have it and it dominates their lives.
LPLD SUFFERER JILL PRAWER (PRON: PRAVA) SAYING:
"I feel like I've had in some ways a half life in that I haven't been able to engage with what the mainstream are doing. I've felt a bit on the outside looking in."
The disease forces Jill to take extraordinary precautions with everything she eats or risk severe abdominal pain leading to life-threatening pancreatitis.
But now a cure based on gene therapy is in sight. A drug, Glybera, has been approved by the European Medicines Agency, the first time it's okayed a genetic treatment.
Dutch firm UniQure created the drug and the treatment. It works by injecting a new gene into the patients' cell through a harmless virus called a vector, according to Uniqure CEO Joern Aldag.
UNIQURE CHIEF EXECUTIVE OFFICER, JOERN ALDAG SAYING:
"The way it works in this particular case for Glybera is that we inject in an outpatient procedure at expert hospitals into the upper leg muscles and the patient, after those injections, can go home after three hours."
The new gene replaces the faulty one and instruct the patient's body to work properly.
UNIQURE CHIEF EXECUTIVE OFFICER, JOERN ALDAG SAYING:
"This lipase that we are restoring, the expression of which we are restoring, acts like a pair of scissors and it cuts fat molecules that are in your blood system after a fatty meal off so that you can pull them into the muscles for energy production. If you don't have that pair of scissors, the fat just continues to travel in your blood system and that it is terrible. It's like having ten percent cream in your blood."
Winning approval for Glybera was difficult. As the condition's rare, fewer than 30 patients could be tested, but the European Medicines Agency agreed in July there was sufficient benefit to justify using Glybera to treat the worst-affected patients.
One of the committee was gene therapy researcher Thierry VandenDriessche.
RESEARCHER IN GENE THERAPY AND EUROPEAN MEDICINES AGENCY'S COMMITTEE OF ADVANCED THERAPEUTICS MEMBER, PROF. DR. THIERRY VANDENDRIESSCHE SAYING:
"This has never happened before and I believe that this actually creates a new opportunity for commercialisation of gene therapy. The different stake holders and industry and academia will hopefully work more closely together now to expedite the marketing of gene therapy."
Jill Prawer won't be among the small number of patients receiving Glybera, as she's successfully coped with the disease for decades.
But as a prominent LPLD campaigner, she's thrilled by the news.
LPLD SUFFERER JILL PRAWER (PRON: PRAVA) SAYING:
"I think it's fantastic news, actually, especially for younger people with the condition, who maybe haven't got to grips with managing it. I think it will be a fantastic innovation for them and will help their lives immeasurably. So I'm very pleased that it's arrived and rather shocked and surprised that this condition would be the one that's been focused on."
The idea of treating disease by replacing a defective gene with a working copy gained credence in 1990, but few drugs have been successfully created and approved.
Glybera's success could change all that and push gene therapy to the forefront of new medical research.