Gene Therapy: The Future is Now

By Laura King Edwards

Predicting the future is a messy game, but “Back to the Future Part II” got a lot of things right (while we don’t have “Jaws 19,” we have plenty of big-screen TVs).

I was 7 years old when Marty McFly and Doc Brown traveled to Oct. 21, 2015 to save McFly’s kids. I dreamed about flying cars and robots and wondered how Nintendo managed to fit so many pictures on a single 8-bit video game cartridge.

When I was a kid, I wrote a lot of science fiction and fantasy stories. The iPad I use to watch movies and search for recipes and race destinations isn’t too far off from the crazy machines I loved picturing but never thought possible. It was hard enough imagining how Nintendo squeezed all of Death Mountain into my Legend of Zelda game; how could anyone possibly fit the whole world into a computer smaller than a notepad?

The world is an amazing place, and I’m astounded every day by the things humans can accomplish. We’ve landed spacecrafts on Mars. We’ve made handheld devices with more storage than early supercomputers. We’ve even made a hoverboard!

But I don’t believe anything compares to what we’re achieving with modern medicine. Think about the diseases that used to destroy entire populations. Now we can pop a pill for a lot of these illnesses and be back at work or school the next day.

And now? Gene therapy is the next game changer. Want to tackle the problems caused by a devastating disorder like Batten disease? What about fixing the disease at its root – by replacing the defective gene with a good copy of that gene, complete with a full set of instructions for making the missing enzyme that causes all of those problems?

Now, imagine swapping the Batten disease gene for a working copy of the gene for another disease like ALS (Lou Gehrig’s disease), Alzheimer’s disease or Lewy body dementia. With a few tweaks, you’ve got a treatment for another disease with tragic consequences.

The best part about gene therapy? It’s potentially a one-time fix for chronic diseases that exact huge physical, emotional and economic costs, sometimes over many years.

And it’s not even science fiction.

This May, a clinical trial for a rare childhood disease called giant axonal neuropathy (GAN) kicked off at the NIH. The trial, the world’s first for a gene therapy treatment administered directly to the spinal cord, is the result of the hard work and genius of my friend, Dr. Steven Gray of the UNC Gene Therapy Center.

Batten disease is next in line.

I still don’t understand how Nintendo managed to squeeze The Legend of Zelda onto that 8-bit game cartridge or how all of my music and thousands of photos fit on my iPhone. But gene therapy makes perfect sense to me. In fact, the only thing crazy about gene therapy is not doing everything we can to keep moving it forward.

Watch this video to meet our dream team and learn how you can help make the future now for kids like Taylor.

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