Hello, Good Morning!

As always very informative and excellent discussion. I just want to add a few words to this discussion.

Peer review and publication of data is a norm in science, as with everything you see in science, it takes time to publish a paper. Peer review examines data from different angles, validates authors' conclusions and many times reviewers ask for more explanations, expts to make the paper stronger and useful. This process becomes even more significant for high risk and high reward projects. When one is so involved in a project that has a high reward, it is easy to focus on the data that confirms one's proposed theory and miss or ignore data that "doesnt make sense". I am always uneasy about getting information from interviews or even just the abstracts of peer-reviewed papers. I am not sure partial information is necessarily a good thing!

Ceregene's interpretation of the data as quoted:

"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. 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..............

......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."


Although Ceregene's conclusions from their study suggest a defect in transport, off hand, I can think of several reasons that might contribute to their observations. They may have ruled out all the possibilities I am thinking of and hence came to their conclusions. Buf how do I know that?? If I was planning a clinical study, I would be very uneasy to design my study (like the location of injection) without seeing their data, however tempting it might be to do the "right thing".

Since Ceregenes' observations are important and may determine the outcome of any new study, is Ceregene planning to present or publish it soon (if its not done already)? Did anyone other than Ceregene and its collaborators get to see the data and evaluate it carefully? I am assuming MJFF gets scientific reports from their grantees.

I just reread the thread, instead of rambling...
I second what Linda said;
" Hopefully, the Ceregene researchers and the gene therapy consortium will be able to collaborate to possibly improve the protocol and outcome of the GDNF gene therapy study, even if not yet published in the traditional manner. Maybe they already are. If they can learn from the Ceregene trial and advance the science and bring us closer to a cure, it has not been a failure. Perhaps the Fox Foundation can play a role in facilitating communication and collaboration.[/QUOTE]


This sort of collaboration initiated by patients and the involvement of a funding agency in this process might be too much to ask for (I know my scientific community pretty well)! However, MJFF is changing the profile of a funding agency so much, there is a always a possibility for new things!!

Thanks
Girija