Paula, I find it utterly amazing that in the five weeks since April 17th, the trial had recruited 18 out of the 51 patients. An while I was being evaluated I was told that there were two more patients inquiring at the Philly office about becoming involved. I believe that this is a strong indication as to the value that the PD community puts on this evolving therapy.

FYI...I am patient #18 of the 51...#1 in Philly. #2 in Philly is a week behind. (Hey, go prod Perry will ya. He emailed me last night to inquire, but is still on the fence about the trial.)

I wrote this in an email to a couple of inquiring friends Weds night after returning home.

I am very tired.

The last two days...

As with any trial, I can drop out of the trail at any point, even as they wheel me into surgery...which Dr. Stern said again today. There is no secrecy for patients with this trial...I specifically asked, since there was nothing in the consent.

The consent form review was two hours...to quote the trial coordinator, I "asked questions most patients wouldn't think to ask." Of course, this was due to my involvement with PPP that I knew what questions to answer. ALL of my questions were answered either by email to Ceregene that were read to me verbatim or by way of documents that were available in the Philly neuro office that were read to me verbatim, before I left today to come home.

I had several Phase I questions, as did my daughter; e.g. adverse effect, known migration of the gene to undesirable locations, development of antibodies (none to date). There were three patients with side effects in Phase I...side effects are considered anything that you did not report as having before the surgery; e.g. headaches, change in speech, stomach aches, etc. During the post-surgical MRI...which is immediately after surgery, before the recovery room...one Phase I patient was found to have a minute fleck of metal in his brain along the path of the injection needle, which to date has not caused him any adverse effect since his surgery...so the trial surgical cleansing protocol was modified regarding the cleaning of the wound and the cleaning of the injector prior to surgery...of course, this is something that could happen in a DBS surgery...and I saw and read the change in protocol.

This trial is likely to have FDA audits at the investigational site level. From what I heard over the past 36 hours, oversight is extensive. I had to answer a questionnaire at the end of the day today that ensures that I understand all aspects of the trail...required by an ethics "office"...the formal name of which escapes me. An incorrect answer would have meant a re-review of that component or a complete re-review of the Consent.

I hope everyone realizes that the 68 patients in the Sepheramine Phase II were also in a double-blind trial with the sham component. The final patient for that trial has her surgery in June.

I can understand the apprehension of anyone who feels uncomfortable with sham surgery. I guess in some ways it is a matter of perspective. Dr. Stern...the investigator...has done his best to explain why the sham is paramount to this gene therapy study. I know I won't explain it very well, but they feel that with the placebo effect, which is of course always a issue regardless of sham or no sham..surgery or pills...is necessary and the only way they can test efficacy is to sham.

My daughter and her husband are educated people who don't hesitate to question anything or do their own research into things; e.g. sham surgery. Until yesterday, my oldest daughter had not been exposed to PD other than the fact that I am in and out of her home as a grandmother and I have PD, take pills, my hands shake and don't walk well. With all the tests and questionnaires and more and more today and yesterday, including off and on evaluation this morning, she saw and heard it all. Twice today she told me that I was doing a good thing.

My "off" score this morning on the UPDRS had to be above 30 to qualify for the study...it was 55. My "on" score was 28. The reason it was so high "off" was due primarily to my tremor which is getting really bad and the reason I was considering early DBS. Since I have never been off meds, I didn't know what to expect this morning. I think my daughter was shocked...we look so good on meds...LOL I was a mess.

I had an email from Linda Herman regarding sham articles she found if I wanted them. She told me I was brave and that she wasn't trying to sway me away. I do have to say that every time someone tells me how brave I am, I shutter and ask myself what the heck am I doing. Saying I am brave sounds like I am doing something I shouldn't. Peg was brave a few years ago, even though it was not a blinded trial, but it was Phase I...nothing was known except animals results. At least this is Phase II and there is some known data on humans.

I am the 18th of the 51 patients, and the first at the Philly site. The second is one week behind me on the evaluation and surgical schedule (PD 27 years). There are two more interested in the Philly site...one in Philly, one from Connecticut of all places. The trial schedule begins yesterday and is extremely black and white. My surgery will be the 18th or 19th of June (within 30 days of yesterday)

The first 20 patients are being asked to go to Vancouver, Canada for a special FluoradopaPET to determine if the compound CERE-120 changes the activity in the area of the brain affected in PD...one pre surgery. So, my daughter's and I are discussing the feasibility of the trip. It has to be done before the surgery, in addition to another Philly two day visit on the June 6/7, followed by the surgical trip...yikes. This is a very complicated month since, 1) this PA daughter sold her home and is moving the week of surgery, and 2) believe it or not...crazy military...my son is coming home for leave already IN June.

Oh the dyskinesia effect. Well, it seems that the reason for the dyskinesia effect is not clearly known. It is one of two things. Either it is the result of the sudden effect of the gene on the brain tissue or it is the result of meds in combination with the effect of the gene on the tissue, since we are put on our usual meds regimen post surgery. With Phase I, once the meds are adjusted downward, the dyskinesia goes away quickly. The hospital stay is usually over night. The only reason that anyone has stayed more than this is due to urine retention, severe headache...things like this.

Probably up for a couple more hours.

Well, I have rambled long enough,

Carolyn

While I didn't find a family member to go with me to Vancouver, I did find a friend (a NT member) who has committed to go with me. I would travel alone, but the Univ of British Columbia won't allow it since the FlurodopaPET is "off"...ugh!