[LindaH]

FYI
The NIH Recombinant DNA Advisory committee RAC) will be reviewing the trial protocol for the new CERE-120 clinical trial (Phase I/II) on Wed. , June 17 at 10:30.
You can watch a live web cast of the meeting at :

http://videocast.nih.gov/summary.asp?live=7699

The agenda is below

Wednesday, June 17, 2009 (continued)

"10:50 AM Discussion of Human Gene Transfer Protocol #0904-981: A Phase 1/2 Trial Assessing the Safety and Efficacy of Bilateral Intraputaminal and Intranigral Administration of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) in Subjects with Idiopathic Parkinson’s Disease

PIs: William J. Marks, Jr., M.D., University of California, San Francisco, CA

Mark Stacy, M.D. Duke University, Durham, NC

Sponsor: Ceregene, Inc.

RAC Reviewers: Hildegund C.J. Ertl, M.D.

Robyn S. Shapiro, J.D.

Lee-Jen Wei, Ph.D.

James R. Yankaskas, M.D., M.S.

Ad hoc Reviewers: John D. Elsworth, Ph.D., Yale University, New Haven, CT

Thomas Freeman, M.D., University

of South Florida, Tampa, FL

Tab 2561 Protocol

Tab 2562 OBA Summary

OBA Letter to PI on In-Depth RAC Review and Public Discussion

Reviews from Drs. Elsworth, Ertl, Freeman, Wei, Yankaskas and Ms. Shapiro PI’s Response

12:45 PM Public Comment

http://oba.od.nih.gov/oba/RAC/meetin...INAL%20RAC.pdf



You might also find interesting the earlier RAC meeting for the frirst recently completed CERE-120 phase II trial . It was held on Sept. 20, 2006 and is availab le on the RAC's archive of meetings at:

http://oba.od.nih.gov/rdna/rac_past_...sts.html#sep06

About the RAC

" The RAC is a technical committee whose goal is to consider the current state of knowledge and technology regarding recombinant DNA. This includes review of human gene transfer trials, and an assessment of the ability of DNA recombinants to survive in nature and the potential for transfer of genetic material to other organisms. It also considers hypothetical hazards and methods for monitoring and minimizing risks. Approximately one-third of the 15 members do not have scientific expertise but represent public interests and attitudes. This balance is intended to provide a forum for open public debate of social and scientific issues attendant to recombinant DNA research. The RAC has been overwhelmingly successful in achieving this goal. "

[paula_w]

ok - you[the forum] have lots of good ideas. so now i'm going to point out what some others have been doing all along, it's time for closure, to drop patient competition and lack of support. patient cliques in the orgs, and on and on. i'm going to share when i see something that i know to be attended by pipeliners or other real pwp without org sponsorship,

This meeting should be very intersting Two pipeliners, with the help of other pipeliners researching and pulling the information together into a backgrounder [two members are college librarians ] will be attending this meeting. They are FDA consultants and have much knowledge, but patients are often used for window dressing. These guys know their stuff and the institution.

Lack of usage of patient advisory committees is not acceptable in the online world. i am writing the book partly to tell the story of some silent warriors, who faithfully workd for the cause for no rewards.

Honest, loyal, . new groups have some big shoes to fill. This isn't criticism; it's a challenge, I wish you victory, but i'm going to point out just what has been done or is being done as an educational legacy.

Also, pdf has a training institute for patient advocates, an idea formed in part by ronnie t at pdf after attending a weekend with pipeliners to plan for the year.

paula

that's a teaser and another tally on the side of writing the book. debi, i will be emailing shortly about this book I think you will truly appreciate the angle and it does include mike, but in a very good way. this is positve. i'm hoping that you can trust us with the story. We lived this atory and it isn't over yet.

meanwhile, good luck with cere-120 and don't miss this significant webcast.

Tony and Arnie, if they are seen at all, are pipeliners.

[Stitcher]

I attend my 24-month eval/visit for CERE-120 on Wed of next week. I am full of questions for my PI. And I know that our PPP FDA Consultants, who are attending the meeting on Wed, will provide me with more questions to ask.

Air date: Tuesday, June 16, 2009, 2:00:00 PM
Time displayed is Eastern Time, Washington DC Local

I have read the RAC Meeting Minutes from Sept 2006, and had hoped to attend Wed with Tony and Arnie.
Of particular interest to me were comments:

• In the phase I trial...Regarding efficacy data, in the low dose cohort at 12 months, a mean 35% reduction in UPDRS-off scores was observed with evidence of persistence response in three of three subjects.
• For the high dose cohort at six months, a nearly 40% reduction in the UPDRS-off scores has been observed.

I will be re-consented again. The changes include: "After this visit which is month 24, there will be a month 36 and month 60. This will eliminate visits at months 42, 48, and 54. Phone calls will be made to you at month 30 and month 48. Also, the study will end at month 60 and there will not be annual visits thereafter. " (This also means that there will be no visits after month 60 for those that were treated...me for one...which were to be annual until death.)