Europe's first gene therapy approval anticipated

What a milestone. After three decades of development, European regulators have recommended approval for the first commercial gene therapy in Europe and the US.

The therapy is for a rare genetic condition called lipoprotein lipase deficiency (LPLD). People with the disorder can't break down globules of fat in the bloodstream called chylomicrons. As a result, these clog up blood vessels in the gut and pancreas, often resulting in agonising visits to intensive care. The only existing treatment is for those with LPLD to avoid fat in food altogether.

In healthy people, muscle cells make an enzyme called lipoprotein lipase, which breaks down chylomicrons. People with LPLD lack the enzyme because of defects in the gene that makes it. The new treatment, called Glybera, works by replacing this gene with a healthy copy. Doctors inject into patients' upper leg muscles a harmless virus that permanently implants a working gene into muscle cells, theoretically enabling patients to make the enzyme indefinitely.

"Patients with LPLD are afraid of eating a normal meal because it can lead to acute and extremely painful inflammation of the pancreas," says J?rn Aldag, the chief executive officer of uniQure BV, the company that has developed the therapy in Naarden, the Netherlands. "Now, for the first time, a treatment exists for these patients that not only reduces this risk, but also has a multi-year beneficial effect after just one treatment," he says.

Aldag says that although the treated people can't risk eating lots of fatty food, they are likely to make around 5 to 10 per cent of the enzyme that a healthy person would make, which is enough to clear the fat they take in from a healthy diet. The treatment is still effective in people who trialled the injections six years ago.

Aldag also said the news was good for gene therapy generally. "This is phenomenal, because the promise of gene therapy is clear," he says. "It allows you to restore the natural function of the body and bring a long-term effect to patients, many of them with very severe diseases."

First for the West

Although several experimental trials of gene therapy are under way, some of them for more than a decade, uniQure is the first in the West to win regulatory approval for a commercial treatment.

The only other gene therapy available commercially is Gendicine, which was approved in China in 2004 to treat cancer.

On 20 July, experts at the European Medicines Agency recommended that Glybera be approved. The European Commission will have the final say within three months ? however, it usually follows the EMA's advice.

Although the disease affects only one or two people in every million, the treatment is valuable because no other options exist. When the company tested the treatment in 27 patients, it reduced fat concentrations in blood within one to three months, and reduced the frequency of pancreatic pain attacks by 50 to 60 per cent.

UniQure is now pursuing other gene treatments for a blood clotting disorder and Parkinson's disease.

Murky past

The approval is a landmark in the chequered history of gene therapy. A trial of a gene treatment for children with severe combined immunodeficiency, who have no functioning immune system, was called off after some of the patients developed leukaemia. Activation of cancer-causing oncogenes by the viruses that delivered the treatment was blamed. After a temporary suspension, the trial led by Alain Fischer at the Necker Hospital in Paris resumed in 2005.

The biggest setback to gene therapy was when a patient called Jessie Gelsinger died in 1999 after reacting to the virus he was injected with.

But in recent years, a multitude of trials have reported successes against HIV, Parkinson's disease and a rare form of blindness.

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