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 â€˜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.
Gene Therapy from Ceregene
The CEO of Ceregene, a gene therapy company based in San Diego believes an approval such as the European Medicines Agency one, â€œis really potentially going to change the way the field is looked at, there are no approved products in the major markets, they (big pharma) sell in.â€ Most pharmaceutical firms have steered clear of investing in gene therapy precisely because of the lack of approvals.
This revolutionary therapy is all about giving the body perfect versions of genes that currently defective and thereby causing health disorders. To date, gene therapy has only been used in clinical trial settings and not in normal medical practice. The European approved gene therapy is one that cure’s lipoprotein lipase deficiency. This is a genetic condition that afflicts just a few hundred patients in the EU and is similarly rare in America.
Glybera from uniQure
Sufferers of this condition have an abnormal genetic code that makes them unable to produce an enzyme that is vital for the breaking down of particular fat-conveying particles that circulate in the bloodstream after consuming food. When these particles are not absorbed, fat builds up to the concentration where blood actually turns white. This leads to periods of acute inflammation of the pancreas, and the only treatment has been a near zero-fat diet. The approved gene therapy is called â€˜glyberaâ€™ and is made by a Dutch company called â€˜uniQureâ€™.
[box type=”note”]Glybera gives proper copies of the lipoprotein lipase genes. It then lets sufferers manufacture within their bodies the required enzyme. The treatment is delivered once every few years by injection into specific zones on the leg muscles. UniQure plan at some unspecified future date to apply for approval from the FDA.[/box]
Glybera is an exception to the norm for gene therapy because most clinical trials have fallen at the hurdle of delivery and long-term effectiveness. A teenager also died during a clinical gene therapy trial in â€˜99 at the University of Pennsylvania. These two factors combined to have made pharmaceutical companies reluctant to invest sufficient funds in the therapy to bring it to a tipping point of approval. UniQure is a privately owned company and the rump of Amsterdam Molecular Therapeutics, a company that went bankrupt in the search for this medical â€˜holy grailâ€™.
Slowly, but surely gene therapy research has been showing some encouraging results. Most notable among these have been treatments for some cancers, hemophilia B, some specific immune diseases and some conditions that cause blindness.