He's standing on ME!

(Just kidding Mike...)

Hi all

Last day of school for the kids today!!!!!!! It was definitely time for school to end, as the natives were getting restless! Teachers' last day will be next Wed. Then, whatever shall I do?

We're going kayaking on Saturday. I am looking forward to it.

mary

Had 2 wk post op hyst. surgery yesterday. Dr says that I'm on schedule except for pain (unexplained). He says if neuro doesn't order sleep study he will. I said that I don't see that happening.

After 1-hour trip and back from dr, am in most pain since surgery. Even allergic to pain med he rx'd. I have one pain med plus xtra strength ibuprofen, but because of other problems have not had pain pills in over a week.

Anyway, cleared to drive and do whatever, so will try to do more.

Mamakathy

After two days of stuff I made "the cut" in Philadelphia. Lots of questions on my part that were answered either by sending an email to Ceregene or by pouring through the documentation in-office, then read to me verbatim; e.g. letters, emails, modification documents.

I almost didn't make the cut, but my tremor took me way over the top...arms very bad, legs moderate...hence, its either this or an early DBS. The scale score had to be 30 or above in "off." That score was 55... My "on" score was 28...I could increase meds, but neither I nor Dr. Shulman had wanted to do that due to the likelihood of increased dyskinesia.

I do have to say that I have been in several trials in the past, but this one went far and beyond with regard to transparency. I even had an ethics questionnaire to complete at the end of the two days. If I had answered even one question incorrectly, we would have gone over that component again as though we hadn't done it before... Also, I am told that it is highly likely that this trial/study will be audited in-house at the coordinating centers by the FDA.

The bad news is that I can't be around small children, pregnant women and elderly people for 30 days post surgery. So, my daughter and her family will move into their new house and because my youngest grandson is autistic with a compromised immune system, I won't be able to visit and hug my grandchildren. Luckily I have already been in the house once.

For the Pipeliners: I asked many, many questions that the general patient may not think to ask (antibodies, etc.), due to my exposure to hopeful future of "patient advocates" and "bill of rights," that the Project has been working so hard to bring about. This trial, more than any other, made it extremely clear of the need for patient advocates.

The lead investigator [is that his title?] , in the Ceregene neurturin trial, Dr. Ray Bartus, and the company itself, is taking great pains to not repeat any of the mistakes made in the GDNF trial. This trial is going to show many of the improvements that patients feel are needed to develop trust. Dr. Bartus attended the round table in New York held by PDF and he attended the entire PAN conference.

This should be a very significant clinical trial, and we are very fortunate to have a participant among us, who is also a patient advocate. We look forward to going through it with you Carolyn.

paula

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!

Glad you had a good time in Crete Ron.We love the Greek Islands and are off next week.We love Elounda on Crete where there is wonderful swimming.I hope you dropped in there to boat across to "The Island." which is every bit as interesting as the novel that is set there.If P.D has taught me one thing it is to make the most of every good day.At the moment I am frantically cleaning the house before my sister arrives to house sit then we are off ...Greece here we come.
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Lloyd I can assure you that being a grandpparent is one of the most wonderful experiences.The only trouble is that it signals time is passing.A sad moment was when my grandson 6 said to my husband (PWP)and me that he wanted us look after his children like we had looked after him.!! Enjoy the experience .....exhausting.exasperating enjoyableand wonderful.

You will find it interesting that the two scanner techs are Carolyn and Caroline

,,, ken

When I was in Dallas for the AWARE for All conference, at one point they invited all people who had participated in clinical trials to come to the stage. These people were called “heros” and given a standing ovation.

So Carolyn, I add you to their ranks (again - as this is not your first trial).

And I thank you and want to tell you how lucky I think you are! For the first time since GDNF, we’re seeing a trial using a neurotrophic factor. And you are one of the lucky people who will participate in the trial! I salute you & I envy you!