The Second CERE-120 Trial (Or Why I Had Brain Surgery)

Brain surgery is not one of those medical procedures that most people grow up looking forward to, nor is it something you would wish on anyone else. The second CERE-120 study, funded by Ceregene (a San Diego based company) and using Ceregene's gene therapy technology, has the potential of halting Parkinson's Disease and perhaps even restoring the cell damage that leads to Parkinson's.

First, you must understand a couple basic things. The brain contains neurons that produce dopamine, and it is dopamine that allows the brain's muscle control center to work correctly and efficiently. When the brain does not have enough dopamine to trigger everything it needs in the muscle control center, Parkinson's Disease symptoms show up. In fact, when symptoms begin to show, the brain has already lost eighty (80) percent of the neurons that produce dopamine. Most of these neurons are in the substantia nigra.

Ceregene has developed CERE-120, a gene therapy product that delivers the neurotrophic factor neurturin, a naturally occurring protein that has been demonstrated to repair damaged dopamine-producing neurons. CERE-120 is an AAV (adeno-associated virus) vector carrying neurturin. The basic concept is that the protein is delivered through a virus. If you think of the virus structure as a soccer ball, the viral shell remains, but the insides of the virus have been replaced with this protein. The virus no longer replicates as viruses normally do; it merely attaches to the cells and delivers the protein.

This is the second CERE-120 study. The first study, done over the last two or three years, delivered two drops of this formula into the putamin. After 12 months, the participants in the trial were not seeing significant improvement, and the project was considered a disappointment. Analysis by the scientists at Ceregene determined that the brain actually blocked the virus from going down to the substantia nigra, where the formula was needed the most.

The second CERE-120 study delivers ten drops directly into the substantia nigra. As my brain surgeon explained, "We're bathing the substantia nigra with this solution."

When the opportunity came up to participate, I jumped at the chance. I was diagnosed almost eleven years ago, and at the time of the diagnosis, my neurologist explained that I probably have ten to fifteen years of functional mobility left. Time, it seemed, was running out. I also had the opportunity to participate in Phase One of the study -- this is the safety and efficacy phase -- in which six of us, over 6 months, each have the procedure done. The Phase One participants get the real thing, not the placebo. If it is determined to be safe, then it will open up to a full double blind study in September, 2010.

My surgery was on March 2, 2010, and I was the third person to have this procedure done. Am I seeing any changes yet? No, not yet. When I asked the surgeon what I could expect afterward, he shrugged and said, "We really have no idea." He also explained it could be many months -- even years possibly -- before we saw any changes, if we do see any.

What was the procedure like? Well, I had two one-inch holes drilled in the top of my skull, followed by ten injections down into the substantia nigra. The holes were covered with titanium plates afterward, and I am still working on getting my hair to grow back.

Everything went smoothly. Duke is an amazing facility, and I am so blessed to live nearby (a 45 minute drive on a good day).

Will this cure Parkinson's? I don't know. I recently found out that the participants in the first CERE-120 study are actually now starting to see some benefit, though I haven't heard any qualifier or description of what the improvements are.

I feel like a kid waiting for Christmas to come. I believe something is going to happen. I just don't know what.

Is this the cure? I hope so!

Just before "going under" for the four hour surgery, I asked the Physician's Assistant if I'd be able to play the violin afterward.

She smiled and asked, "Do you play the violin now?"

I returned the smile and said, "No."

Still, even if I can't play the violin, if this slows down or stops the progression of the illness, or if it can even reverse it, I would consider it a miracle. I'll take whatever comes.

I'll keep you all posted. This is an exciting time for me!

For more information CERE-120, see this article from the Michael J. Fox Foundation.