The West's First Gene Therapy Goes On Sale Mid-2013

We're tampering with genes to cure a rare metabolic disorder--and that's likely just the beginning.

By Clay Dillow Posted 11.06.2012 at 3:45 pm 8 Comments

Gene Therapy: Tampering With DNA Using genes to generate protein therapies in the body. National Institutes of Health

The first gene therapy to be approved in the West will hit the market by the middle of next year, opening the masses to a controversial treatment that directly alters a patient’s own DNA. Dutch biotech uniQure’s Glybera was approved for sale by the European Commission late last month.

Gene therapies emerged--appropriately--about the same time the first human genome was being mapped during the 1990s, though the study of gene therapies goes back as far as the 1970s. They work by actually tampering with a person’s DNA--usually by encoding a functioning gene to replace a mutated one, or by introducing DNA that encodes a therapeutic protein into the body. Clinical trials have gone on for years. Early on, failures caused many to dismiss the idea of tampering with genes. But later--in the following decade--many in the medical community changed their minds after witnessing a series of successes. Among them: China, which approved its first gene therapy in 2003.

In the West, however, gene therapy has been a source of constant debate, and that’s what makes this development notable. Gene therapies carry a lot of promise, including the ability to treat any number of inherited diseases that have few treatment options. They are a way to literally tinker with the fundamental material that tells our cells how to function, so their potential is indeed vast--if we can make them work.

Glybera will treat lipoprotein lipase deficiency (LPLD), an extremely rare inherited disorder affecting the metabolism of certain fat particles. It affects something like one or two people in a million, so it’s not like we’re curing blindness here. Gene therapy is still limited to single-gene disorders--and most common diseases are more complex multi-gene problems.

Still, the acceptance of the first gene therapy into Western medicine could mark a turning point for gene medicine, provided nothing goes wrong. Many labs are still working on gene therapies for a number of conditions, and uniQure itself is working on additional genetic therapies for everything from hemophilia to Parkinson’s. Even limited success there would naturally be a huge leap forward for medical science.

[Cosmos]

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8 Comments

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Shakouhousha

11/06/12 at 5:04 pm

That is both incredible and freaky. This could be the future of medicine or the undoing of mankind, we'll see.

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Greenmatrix

11/06/12 at 5:19 pm

Kudo's to these Dutch scientists for leading the way. That must have been a lot of work and headaches developing this, testing it, proving it works and finally getting this all approved for the market.

So many things can be cured with this...eventually.

It`s like the quantum computer power of the medical world.

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Greenmatrix

11/06/12 at 5:34 pm

It's hard to understate just how much of a major step forward for genetic engineering this is, if they get to the point where they can easily pick and choose the DNA to replace then genetic diseases could all be eliminated. Just think about that for a moment, we'd be able to correct Down's syndrome, Cystic fibrosis and Turner syndrome to name but a few. I can even see a few ways that this could be used as an effective cancer treatment. This is massive!

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Mukuro Holmes

11/06/12 at 10:11 pm

I surprised this didn't get approved earlier. Goes to show how inefficient bureaucracy is. Still, at least we making some baby steps in the right direction. If this develops for another 100-1000 years (providing that there are medical advancements) we may actually be able to change ourselves to become superhuman. Then controversial issues start to erupt like the problem of "have-access to gene therapy" and "have-nots". Sigh...why are humans so shortsighted? The benefits obviously outweigh the risks.

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Robot

11/07/12 at 8:51 am

I hope this does help people, still I am having flash backs to the movie, "I am legend".

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Volt

11/07/12 at 10:32 am

Someday... Some day I shall see red and I will be happy.

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Robot

11/07/12 at 3:39 pm

D13,
Sadly, there is truth to your words. It is less profitable to cure the sick, but more profitable to just ease their pain.

And with health care being funded so much by government funded programs (that have hidden them, cost of living increases), health care will always raise their cost, knowing in advance they will get the money. There is no reason to lessen the cost of health care.

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