First curative gene therapy awaiting approval in Europe
Pharmaceuticals giant GlaxoSmithKline is awaiting approval to sell a gene therapy across Europe which offers a complete cure to severe combined immune deficiency. The therapy, called Strimvelis, uses a “repair and replace” system, taking out cells and fixing them in a test tube before returning them to the human body. Marcia Boyle, founder and president of the Immune Deficiency Foundation said: “the idea that you don’t have to worry about it and can be normal is extremely exciting for people.” Strimvelis was first developed by the San Raffaele Telethon Institute for Gene Therapy in Italy, and could pave the way for similar cancer therapies.

Called Strimvelis, and owned by drug giant GlaxoSmithKline, the treatment is for severe combined immune deficiency, a rare disease that leaves newborns with almost no defense against viruses, bacteria, or fungi and is sometimes called “bubble boy” disease after an American child whose short life inside a protective plastic shield was described in a 1976 movie.

The treatment is different from any that’s come before because it appears to be an outright cure carried out through a genetic repair. The therapy was tested on 18 children, the first of them 15 years ago. All are still alive.

“I would be hesitant to call it a cure, although there’s no reason to think it won’t last,” says Sven Kili, the executive who heads gene-therapy development at GSK.

The British company licensed the treatment in 2010 from the San Raffaele Telethon Institute for Gene Therapy, in Milan, Italy, where it was developed and first tested on children.

On April 1, European advisors recommended that Strimvelis be allowed on the market. If, as expected, GSK wins formal authorization, it can start selling the drug in 27 European countries. GSK plans to seek U.S. marketing approval next year.

An artist’s illustration of gene therapy shows a retrovirus harboring a correct copy of a human gene.

GSK is the first large drug company to seek to market a gene therapy to treat any genetic disease. If successful, the therapeutic could signal a disruptive new phase in medicine in which one-time gene fixes replace trips to the pharmacy or lifelong dependence on medication.

“The idea that you don’t have to worry about it and can be normal is extremely exciting for people,” says Marcia Boyle, founder and president of the Immune Deficiency Foundation, whose son was born with a different immune disorder, one of more than 200 known to exist. “I am a little guarded on gene therapy because we were all excited a long time ago, and it was not as easy to fool Mother Nature as people had hoped.”

Today, several hundred gene therapies are in development, and many aspire to be out-and-out cures for one of about 5,000 rare diseases caused by errors in a single gene.