An FDA panel has recommended approval of the first gene therapy drug.

[NNadir]

FDA advisers back gene therapy for rare form of blindness. (News Item, Nature, Vol. 550 Issue 7676)

Advisers to the US Food and Drug Administration (FDA) have paved the way for the agency’s first approval of a gene therapy to treat a disease caused by a genetic mutation.

On 12 October, a panel of external experts unanimously voted that the benefits of the therapy, which treats a form of hereditary blindness, outweigh its risks. The FDA is not required to follow the guidance of its advisers, but it often does. A final decision on the treatment, called voretigene neparvovec (Luxturna), is expected by 12 January.

An approval in the lucrative US drug market would be a validation that gene-therapy researchers have awaited for decades. “It’s the first of its kind,” says geneticist Mark Kay of Stanford University in California, of the treatment. “Things are beginning to look more promising for gene therapy.”

Luxturna is made by Spark Therapeutics of Philadelphia, Pennsylvania, and is designed to treat individuals who have two mutated copies of a gene called RPE65. The mutations impair the eye’s ability to respond to light, and ultimately lead to the destruction of photoreceptors in the retina.

The treatment consists of a virus loaded with a normal copy of the RPE65 gene. The virus is injected into the eye, where the gene is expressed and supplies a normal copy of the RPE65 protein.

In a randomized controlled trial that enrolled 31 people, Spark showed that, on average, patients who received the treatment improved their ability to navigate a special obstacle course1. This improvement was sustained for the full year during which the company gathered data. The control group, however, showed no improvement overall. This was enough to convince the FDA advisory committee that the benefits of the therapy outweigh the risks.

Long road

That endorsement is an important vote of confidence for a field that has struggled over the past 20 years. In the early 1990s, gene therapy was red hot, says David Williams, chief scientific officer at Boston Children’s Hospital in Massachusetts. “You couldn’t keep young people out of the field,” he says. “Everyone wanted in.” Then came the death of a young patient enrolled in a gene-therapy clinical trial, and the realization that a gene therapy used to treat children with an immune disorder could cause leukaemia.

Investors backed away from gene therapy, and some academics grew scornful of it. Although European regulators approved one such therapy in 2012, for a condition that causes severe pancreatitis, many doubted that it worked. (The company that makes it has announced that it will not renew its licence to market the drug when it expires on 25 October.) “You’re too smart to work in this field,” a colleague told Kay. “It’s a pseudoscience.”

But some researchers kept plugging away at the problem, improving the vectors that shuttle genes into human cells. Over time, new clinical trials began to show promise, and pharmaceutical companies became more interested in developing treatments for rare genetic diseases. Gradually, investors returned...

These people will be, of course, GMO, and we can look for the bourgeois assholes at Greenpeace to protest their existence.

Blindness has never worried Greenpeace types, as we can see from their awful and frankly criminal campaign against golden rice, which might have addressed vitamin A deficiencies in, um, poor people.

It is perfectly acceptable of course, to make people suffer if people who neither understand nor like nor are competent to understand science object loudly.

This is good news in any case.

I am involved (peripherally) professionally in a project involving gene therapy for a disease that kills people. Thus I find this approval encouraging.

[eppur_se_muova]

1. Know anything about this upcoming TV program ?

http://www.pbs.org/video/gene-doctors-trailer/

I can't find much info on it besides the trailer, but it might have aired already in some areas.

[NNadir]

2. Nope. I didn't know anything about it. n/t.

[eppur_se_muova]

3. Watched it this afternoon. Not too technical; encouraging as far as it goes.

Kind of hair-raising to wonder what's going to happen to such research under the current occupation of DC.

People with the hereditary blindness described in the OP, Duschene's MS, and some forms of CF were successfully treated; clinical testing continues. No treatment yet for fatal familial insomnia.

[NNadir]

4. The therapy on which I'm playing a supporting role is a disease that kills kids within two years.

As the parent of a kid born with a somatic mutation resulting from a clipped nitrogen on a nucleotide, I have some feeling for what the parents must be going through, although my kid's mutation was not going to prove even remotely deadly.

The researchers on this project I'm supporting have some flared tempers too; everybody really wants to get it done.

My clients have a very interesting vehicle, a designed vehicle, very cool.

I really can't talk too much about it, but while GMOing humans is going to make for a lot of noise, some of which will be invariably stupid, I'm proud to be around these kinds of things.

[eppur_se_muova]

5. Sounds like a very interesting project.

It is, of course, tragic that one of the early GT trials went wrong. But something is bound to go wrong when new therapies are explored. It's a shame that so many companies backed out instead of pressing on. I think one day the opponents of GT will be like an episode of Futurama :

MOM: Ah, there you are, Leela. I just wanted to thank you for helping me engineer the perfect bean. You've made me a very richer woman.
LEELA: What?! I would never help you commit your perverted crimes against nature! My marigolds!
MOM: Thanks to that DNA sample you generously donated while locked in my tower, my beanstalks now have suction cups to keep them from collapsing.
LEELA: That's horrible!
MOM: Is it? Now the world's starving masses have a cheap, abundant source of nutrition. But not too cheap. I'll take the cash, and you can have the credit.
LEELA: I don't want the credit! Genetic engineering is wrong!
MOM: Oh, really? Tell that to your old friend, the giant.
LEELA: [Screams] Where is he?! Oh, God, he's invisible now!
STAN: Fee-fi, hi. [Chuckles] I'm Stan.
LEELA: Ah-ha! You see? I freed him from your infernal contraption and he returned to normal.
STAN: On the contrary, I suffer from hereditary Gigantism. Or I did, until Mom cured me of my awful disease using genetic engineering. That's why she had me hooked up in her lab. Oh, well, congratulations. You look great.
LEELA: But, even if genetic engineering feeds the hungry, and cures some giant guy! That doesn't make it right. We have no idea what the long term effects will be. And once that genie is out of the bottle...
MOM: I can cure you too.
LEELA: Okay, I'm in.