At the PAN Forum this year, and in the above information provided by Linda, researchers and device manufacturers were still talking about targeting the putamen for gene therapy, this despite the news from Dr. Bartus as relayed in his interview with MJFF that the ceregene trial showed that the neurturin did not transfer to the nigra as it had done in animal models:

"Based on all the pre-clinical and autopsy data we have accumulated in this program, the problem is clear. Delivering CERE-120 to the terminal field (putamen), only, is sufficient in pre-clinical models, but not in the brains of people with Parkinson’s. This suggests a serious problem with axonal transporter mechanisms in Parkinson’s disease. This is a concept that’s growing in popularity and understanding. It is much more clearly established in other diseases involving neurodegeneration, such as ALS and, to some extent, Alzheimer’s disease, but it is gaining acceptance in the movement disorders and Parkinson’s fields. The concept is that, long before the cells die, there’s a loss of processes involving the axonal terminals; and that even before the loss of axonal terminals, there is a deficiency in transport ability within those terminals. While it is our best hypothesis that transport mechanisms in Parkinson’s disease are severely impaired, it is a fact that in the two patients we examined, we saw no evidence for neuturin in the nigra (where we needed it to get), despite adequate levels expressed in the terminal field (where we delivered CERE-120).

Additional support for this interpretation comes from further analysis of the human tissue and its comparison to our pre-clinical experiments. As I stated earlier, only when neurturin gets to the cell body can you expect it to begin repairing the neuron and making dopamine synthesis and other important activities more effective. One way you measure that is by looking at tyrosine hydroxylase (TH), the major synthesizing enzyme for dopamine. In all the pre-clinical studies, when you get CERE-120 in the terminal field, you not only see neurturin expressed in the terminal field and in the nigral cell body, but you also see enhanced TH in both the terminal field and in the cell body. That shows us that when you put neurturin in the terminal field and it gets carried back to the cell body, the neurons become more healthy and functional, including as a better dopamine machine. We saw no evidence for any of that in the Parkinson’s brain. Despite a good neurturin signal in the terminal field, we saw none of the secondary consequences of the neurturin expression that are required for improved function and neuronal repair that were consistently seen in the animal studies. So my point is, simply adding more protein to the terminal field likely won’t do anything because that will not ensure it also gets back to the nigral cell bodies, as is required for efficacy."

(entire interview here)

Is this being taken into consideration for these new trials?