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It is defintely important to get data out on GDNF so if something valuable is there it isn't lost. there is often a chance that if a drug were administered differently--at a different dosage or frequency or if the structure of the drug were changed slightly that it might do better in testing, however its important to know the meanings of the phases of clinical trials when reading about hem phase 1 -- test drug in a few healthy people to see if it is toxic phase 2 -- test drug in small population of afflicted people to see if it has any possitive effects phase 3 -- test drug in large population of afflicted people to compare to existing treatments and to continue analysis of adverse effects. phase 4 also exists, only 1-3 are necessary for FDA approval so if GDNF made it through phase 1, all that says is that it isn't poisonous when tested on a small group of people. of course prior to phase 1 there are animal trials and there may be (I don't know) tests on willing human subjects. without any success in animals (or with any adverse affects in animals) its not worth the investment to even go into phase 1 in humans On Mon, 18 Oct 2004 20:55:41 -0400, Linda J Herman <[log in to unmask]> wrote: > AN OPEN LETTER TO THE PARKINSON'S COMMUNITY > > From: > Parkinson's Pipeline Project > http://pdpipeline.def6.com/home_page.htm > > Grassroots Connection > http://grassrootsconnection.com > > People Living With Parkinson's > http://plwp.org > > The recent news about AMGEN halting their GDNF trials has been > disappointing and disheartening to the entire Parkinson's Community. > > Many of us viewed the preclinical report at the PAN Forum a few years > ago, including videotape, of primates who appeared symptom free after > being treated with GDNF. Phase I subjects at Frenchay Hospital in > Bristol, UK and University of Kentucky demonstrated symptom improvement > and neuron growth. Some considered GDNF research to be as important as > stem cell research. > > The news last summer that Amgen's Phase II trial had been declared a > "failure" was disappointing. Yet an Amgen press release (June 28. 2004) > stated that they would continue to analyze the data and that they were > "committed to understanding if a different approach, including evaluating > a higher dose, may yield an outcome that is consistent with the open > label study." > > Then came the unexpected news that Amgen was halting further GDNF trials > due to discovery of GDNF antibodies in four of the subjects. While we > understand the need to make patient safety the top priority, we are > concerned that complete information about the 'failure" of GDNF will not > be fully disclosed. > > Researchers, such as Dr. Clive Svendsen have raised questions about > possible effects the smaller doses of GDNF and a larger plastic delivery > tube may have had in the Amgen study. There is a strong need for the PD > community to have the following concerns addressed, along with others > that may arise. > > There is concern that: > · data from the Amgen study will not be fully analyzed and compared to > earlier, successful Phase I studies. > · AMGEN will drop all further GDNF studies – due to financial concerns – > as they have done in the past with their neuroimmunophilin and the > earlier GDNF trials, but not make the substance readily available to > other companies and researchers. > · this research halt will affect the previously announced research funded > by the Michael J. Fox Foundation on encapsulated delivery of GDNF. > > Many questions about GDNF remain, and along with Amy Comstock, Executive > Director of PAN, we also sense that "…something just doesn't feel right…. > there seems to be a disconnect in the analysis…" (Gardner) > > We trust our Parkinson's organizations to do whatever is possible to > determine whether the patient safety issues with GDNF can be resolved. > However, we see a need to involve the continuously increasing number of > knowledgeable patient advocates. Many of us have taken risks and served > as subjects in clinical trials, as even more of us will do in the future. > > Perry Cohen, director of the Parkinson's Pipeline project, has served as > a patient representative at FDA for 4 years (and is still the only > patient representative for neurology) and in this role has advocated for > increasing participation in pre-approval meetings between FDA and > industry. In a recent advocate meeting for consumer groups the Acting > Commissioner endorsed the concept of independent patient advisors to FDA > and Sponsors and the staff of the Office of Special Health Initiatives > are developing a plan to implement a pilot demonstration of this concept > for PD. > > While we recognize the ambivalence of regulators, sponsors, and even our > own charitable foundations to have another face at the table; we feel > that it is imperative that PD patients have the right to express their > own assessment of the risk benefit calculation through well-informed > representatives at scientific and regulatory meetings as well as for our > personal treatment options. We have a large audience who can be > mobilized for emails, letter writing, phone calls, and online forums. If > we used all of our resources, including the grassroots communities and > organization voices, the result will be a larger and louder message of > concern. > > In summary, we would like to: > · Form a collaborative effort, meaningfully including PD patients, within > the Parkinson's community to advocate for a full disclosure of trial > results and safety review of progressing and/or discontinued treatment > studies, beginning with Amgen and GDNF. > · Better understand Amgen's criteria for "success" and "failure." > · Urge Amgen to make GDNF available to other researchers. > > This letter will be presented to Amgen in the near future. Will you > support us by adding your endorsement to this letter? Contact: > [log in to unmask] > > For many PWP, this will become a matter of life and death. As Dr. > Langston stated (Pearson), we are well aware that if the research is > abandoned at this point, for us…"there's no sequel waiting in the wings." > We look forward to the formation of partnerships in the quest for more > timely development and approval of new and better treatments for > Parkinson's Disease. > > ------------------------------------------------------------- > Sources: > Amgen press release, "Amgen's Phase 2 Study of GDNF for Advanced > Parkinson's Disease Fails to Meet Primary Endpoint, June 28, 2004. > Retrieved online http://www.amgen.com/news/viewPR.jsp?id=585632 > > Gardner, Amanda " Trial Halted for Promising Parkinson's Drug." > Healthday News, 05 October 2004. Retrieved online through Yahoo news. > > Pearson, Helen. "Parkinson's trial halted." Nature News, published > online: 05 October 2004. Retrieved online > http://www.nature.com/news/2004/041004/full/041004-6.html > > "Profile: Clive Svendsen." Nature Medicine, 10, 659 (2004). Retrieved > online 01 July 2004 through nature.com website. > > October 17, 2004 > > ---------------------------------------------------------------------- > To sign-off Parkinsn send a message to: mailto:[log in to unmask] > In the body of the message put: signoff parkinsn > ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn