Parkinson's Disease: Hope at Last?

Up to this point in time, there has been nothing found (and scientifically demonstrated) to slow down or stop the progression of Parkinson's Disease. The words "you have Parkinson's Disease" have been synonymous with telling the patient that he or she is going downhill and all that can be done is treat the symptoms.

I was diagnosed more than ten years ago at the ripe old age of thirty-eight (yeah, ancient, I know). Since that diagnosis, several things have come along that grabbed the spotlight for a few days, but nothing has actually been substantiated in clinical trials as an effective solution to this insidious disease.

People are affected differently by Parkinson's Disease. I don't have much of a tremor, but I have a pronounced stutter (pun intended), a shuffling gait, a twitching countenance, an impressive drool at times (especially while I am sleeping), loss of flexibility, "freezing" while trying to walk, and a sense of balance that would not do well in the Olympics. I know what you're thinking. My, what glamorous symptoms these are.

Perhaps the most significant impact on me has been cognitive impairment in the "executive functioning" portion of my brain. That is, my organizational skills; my problem solving abilities; and my attention to detail and logistics are all suffering. That has not boded well for my career in software development. I have worked in that field for the past twenty-five years and recently discovered I can no longer "get it done." It's terribly frustrating to realize that something I used to do (with great success) is now beyond my reach.

There is always hope, of course, and now it looks as if the potential for a cure (or a fix, if you prefer) could be just around the corner. Gene therapy has looked promising for several diseases, and in recent years some research involving gene therapy and Parkinson's Disease has been done. Over the past three years, a clinical trial of CERE-120 was held (gene therapy involving the injection of a virus in the brain), and though some of the participants of the study reported experiencing improvement in their symptoms, the data in the double blind study did not hold up. Investigation by scientists led the medical community to believe that perhaps not enough of this drug was used and that it was not injected deep enough in the brain.

A new study of CERE-120 has begun, and the same drug will be used again. This time, though, much more of it will be injected into the brain, and the injections will be deep, actually in the part of the brain where the dopamine-producing neurons live. I am fortunate and blessed to be part of it. I am participating in Phase 1, the "safety and efficacy" phase, and I am the third person who will have had this done. Six people will go through Phase 1 during a six month (or longer) period. If it is shown to be safe and reasonable, then Phase 2 will open up with a double blind study of fifty-eight people.

Is this going to work? We don't know. But in laboratory animals, the dopamine-producing neurons that have "died" and / or are dormant have actually been restored. If this works in humans, it would be the first time that the disease can be addressed and not just the symptoms.

Keep in mind that when a person is diagnosed with Parkinson's Disease, he or she has already lost eighty percent or more of the dopamine-producing neurons that are used for movement. Restoring some or most of those neurons could actually reverse the damaging effects of the disease.

Now that would be cool.

Assuming everything progresses as planned, I will be having brain surgery on Tuesday, March 2nd, 2010. That's less than three days away from now, as I sit and write this. Two one-inch holes will be drilled in my head, and a series of injections reaching down to the substantia nigra (where the bulk of these neurons reside) will be done.

How will my body respond? I don't know.

Will my cognitive abilities return? I don't know.

The surgeon says it could be many months before any benefit is realized. It could be longer.

But we're trying. The medical community has come a long ways since the mid-1970s, when I watched my grandmother wither away from the ravages of both the disease and the massive amount of medication the doctors pumped into her.

Are there risks with the surgery and the virus? Sure there are. But there's a chance it will work. And boy, am I ready for Joel Revision II to be released.