I read the following grant abstract (funded by the MJFF) on another discussion list and it raised lots of questions about where the research is heading and if the scientific community is showing signs of giving up on the more advanced PWP. Full text of Grant abstract at
http://www.michaeljfox.org/research_...s_3.cfm?ID=590
Excerpt:
Immunology and Bioactivity of Regulated rAAV1-GDNF in Rodent Models of PD
Program-non- specific Funding 2009
Objective/Rationale :
“Clinical trials with a trophic factor called GDNF, where GDNF is injected directly in the brain by pumps, have not been completely successful. Many scientists believe that the delivery method is part of the problem and have been calling for gene transfer as the most viable delivery method. We further contend that GDNF will only function properly as a therapeutic when used with early stage patients. In order to perform gene therapy in early stage patients, extreme safety of the gene therapy must be demonstrated prior to use in humans. This project is part of a program to produce a viral vector to deliver GDNF but with the added safety that it can be turned off by a harmless antibiotic if side-effects appear….”
Researchers
Ronald J. Mandel, PhD
University of Florida College of Medicine
The data will be used to move the preclinical studies into a phase I clinical trial – for early stage patients only…
It seems we will be forever haunted by how the results of the Amgen GDNF trial were reported (or misreported) – was it a “failure” or “inconclusive” or “not completely successful?” The research on infusion delivery of neurotrophics seems to have been dropped, although further research was called for to resolve the issues in the Amgen trial. . We will probably never know the true potential of this treatment method.
In some articles, there is finally acknowledgment that at least some of the GDNF paritcipants ( many in advanced stages) did improve –“some dramatically”, but the consensus seems to be now that gene therapy is the delivery method of choice.
Seems we’re always going back to square one… maybe that is the way science works, but time is not neutral for PWP.
It feels lately like the funders and the researchers would prefer to just forget about the "lost generation" of now advanced patients . We’re told to take new formulations of levodopa and get a DBS to deal with the dyskinesia caused by the levodopa, as the scientist’s interests seem to be turning to non-motor symptoms.
And now they don't even want us as lab rats.
The proposed phase I clinical trial, using a new viral vector to deliver the GDNF gene would recruit only early stage patients Is the belief that “GDNF will only function properly when used with early stage patients."
Is this generally accepted or does it reflect the opinions of a few researchers? Are they attributing to all the improvements noted by advanced patients in other neurotrophic trials (GDNF, spheramiine, neurturin) to "just a placebo effect?”
ISeems to me there is going to be a recruitment problem for a trial that asks early stage PWP who are likely to be experieincing good results with drug therapies to agree to experiemental brain surgery. Is it ethical in the first place? What is the risk/benefit equation for them?
And should human gene therapy trials be on hold until a gene therapy regulator is perfected ?..
Last summer there were related discussions on PDOnlime Research . see threads at:
http://www.pdonlineresearch.org/resp...able-promoters
http://www.pdonlineresearch.org/resp...will-take-time
http://www.pdonlineresearch.org/rese...ntial-treatmen
t-parkinsons-disease#commentary
Would like to hear opinions of other PWP. Maybe after 14 years with PD I’m just becoming too cynical and and suspicious.