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Gene therapy rises again

By | November 6, 2009, 12:35 PM PST

After several years of frustrating failure, gene therapy is rising again.

In theory gene therapy means finding a way to change a person’s genetic structure so that an inherited condition may be reversed. In practice, it can mean taking a leukemia or HIV virus, altering its genetic structure, and hoping the cure doesn’t turn out to be worse than the disease.

In the latest cases French scientists arrested the progress of ALD, the disease made famous by the movie “Lorenzo’s Oil,” while American scientists showed the potential of gene therapy to reverse a disease causing blindness.

In the French case, detailed at Science, scientists removed blood cells and treated them with a modified HIV virus carrying the gene for the enzyme the patients lacked. Chemotherapy wiped out the patients’ blood marrow, the new cells were infused, and in time these cells began cranking out the needed protein.

It took over a year for evidence to suggest success — one patient lost some vision, the other some points on a non-verbal IQ test — but the general course of the disease had been halted.

The European Leukodystrophies Association (ELA), which has sponsored this research to the tune of 7 million Euros (almost $11 million) was over the moon.

Shown above is one of the two French patients alongside the group’s “ambassador,” former soccer star Zinedine Zidane. (He’s the Frenchman who spat at the Italian rival during the World Cup final. Yeah, that guy. He’s usually very nice.)

The story is heartwarming but the implications are far greater. Many common diseases like cancer are being shown to have a genetic component. As the use of gene therapy increases, and becomes regularized, we could be finding cures that can be used on millions.

That is at least a decade away, but now we know it’s possible. We have a method.

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Dana Blankenhorn

About Dana Blankenhorn

Dana Blankenhorn was a contributing editor for SmartPlanet from 2009 to 2010.

Dana Blankenhorn

Dana Blankenhorn

Contributing Editor, Healthcare

Dana Blankenhorn has written for the Chicago Tribune, Advertising Age's "NetMarketing" supplement and founded the Interactive Age Daily for CMP Media. He holds degrees from Rice and Northwestern universities. He is based in Atlanta.

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Dana Blankenhorn

Dana Blankenhorn

Dana Blankenhorn has been a technology reporter since 1982, a business reporter since 1978, and a writer for as long as he can remember. His Schwab IRA has a few tech stocks in it, most notably some Intel and Applied Materials bought over 10 years ago. But the vast majority of his tiny fortune (emphasis on the word tiny) is invested in mutual funds. He presently writes for no one else but ZDNet, SmartPlanet and himself. But if you've got an opportunity let him know. If he takes the gig he"ll first add it to this disclosure page.

He writes for SmartPlanet and is not an employee of CBS.

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RE: Gene therapy rises again
Gene therapy is a step towards the Star Trek Tricoder! happy This is great; this is the kind of advancement that will finally, hopefully, take the far to prevalent hit or miss "art" out of health care / medicine. Unfortunately, it most likely will take decades to get there. But it's the "engineering" solution that's so badly needed.
Posted by GarryGR
11th Nov 2009
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RE: Gene therapy rises again
"this is the kind of advancement that will finally, hopefully, take the far to prevalent hit or miss 'art' out of health care / medicine."

Hardly, more like adding a new canvas on which to paint. The Human Genome Project would do more for engineering treatments, but even then, it would take a great deal more understanding to use genetic makeup to simulate a human body and analyze results for possible treatments. I'm afraid that's still hundreds of years in the future.
Posted by rod.boggess@...
12th Nov 2009
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RE: Gene therapy rises again
Gee, I remember visiting Disneyland in the late 1950's and learning that "hundreds of years in the future" men would walk on the moon.

In 1969, I watched it happen. Imagine.
Posted by mike@...
18th Nov 2009
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