Gene Therapy and the Greatest Boxer of All Time

Parkinson's is a nasty, nasty disease. There is no doubt about it. Of course, any disease that is described as both 'chronic' and 'progressive' is not going fun to deal with. A degenerative disorder of the central nervous system, Parkinson's disease (PD) is currently incurable, and effective treatments are nearly non-existent.

And this is where gene therapy comes in. Gene therapy has been considered the holy grail of Parkinson's disease treatment for quite some time. Studies that have focused on use of gene replacement therapies in neural cells have shown promise, but have lacked major efficacy, which would be required for large-scale, multi-phase clinical trials. Various gene therapy strategies have been pursued, all showing positive results, but would still leave PD sufferers with continued standard therapies that can often be cumbersome, expensive, and sometimes nearly as debilitating as the disease itself. A new gene therapy, which takes three previous gene replacement strategies and combines them into one, is being reported by Nature as a potential breakthroug in PD treatment.

It makes total sense...take three different types of gene therapy treatment that have shown moderate success and combine them into one single vector. A multi-functional attack that is aimed at rehabilitating the neural cells in three ways at the exact same time. Genius. And it gets better. The therapy has already been tested on humans, and is expected to move into phase II clinical trials soon. I would love to see this treatment become standard, not only because the eradication of Parkinson's disease would be a beautiful thing, but also because it would solidify gene therapy's place as a viable treatment option for other diseases. I mean, let's face it, there's still a stigma that the public has with gene therapy. And not all of it is without reason. Gene therapy has its risks, one of the largest of which is the insertion of the target gene into a place in the genome that causes major damage to cell function or, even more likely, that causes cancer. Another issue is the current trends in gene therapy research. Everyone would like to see the creation of a treatment for PD, but what about gene therapy treatments for colorblindness or baldness? By comparison, research into these kinds of gene therapy treatments seems a little less awe-inspiring. But hey, who am I to judge? After all, gene therapy research on colorblindness could open the door for other types of ocular disorders, and curing baldness is not always just a matter of vanity; for some, it really is a quality of life issue.

Anyways, I digress. What it really comes down to is this: I want to see the great Muhammad Ali standing tall and proud, without the devastating tremors of Parkinson's racking his body, speaking about what it was like to reclaim his title as World Heavyweight Champion from George Foreman in Zaire during the 'Rumble in the Jungle.' And maybe, just maybe, gene therapy could be the key to granting me this wish.