Through the participants , investigators, ceregene company, [i heard the cere120 employees are mostly laid off], researchers, readers, caregivers, consumers, medical doctors for anywhere on our body....lol...can we create the story? It seems a practical way to discuss everything that is on our minds about all clinical trials with all of those involved in the cere 120 trial. The Cere 120 Reality story....everyone swears an oath to tell the truth.

This always happens; you think you've done [and had!] enough...today this idea just focused even tho others are at their own story versions quite possibly...this idea isn't original, but doing it here would be. Let's write the story. I'm starting with Carolyn. who so many of you know. I'm inviting Dr. Michael Rogan to be the MJFF observer, who is welcome to join in at anytime [I've already begged and explained enough about why I think we should communicate. ]

This kind of story you can't make up as well as the real story will be, please join us Dr. Rogan. I don't think we knew what we wanted to talk about; in spite of all the pleading and the need to have it include many disciplines. This is an ideal topic and an example of many issues on our minds .

Carolyn is ..[carolyn who are you? type it on your thread please].....she can write out how much personal info that she wants to reveal on her own post, but i will introduce her from a patient consumer and advocate viewpoint.

I have known Carolyn for quite a few years now. She is a great Internet correspondent; is not afraid of emotion, very informed and knowledgeable. She is one of the pipeliners who find and supply information; it's like our food supply in importance to us. I think I consume more information than food most days. She will correct any of my mistakes, but every attempt to be accurate will be made from this end. It will soon become apparent how little has been revealed to us, especially by those who know more...., and we think this clinical trial can be the best teachable moment in our struggles to be a part of the solution.

As you will learn about, Carolyn had a fall, which has been labeled a seizure, while in recovery from the trial's gene therapy. From brain swelling, as Carolyn called it, she was in a - would rather she label it - but it seemed semi-lucid state - for about a week, with memory lapses and many yes/no responses. Carolyn reported that she couldn't stop an activity, like brushing her teeth; she didn't know how to initiate the end of the task. Carolyn recovered; maybe she can discuss it in more depth.

Questions- I'll post and then try to categorize, each time I add another post. Some of these are freshly thought, I just talked to Carolyn on the phone. She isn't afraid. Off the cuff, first post:

Someone speak up nicely if we post something confidential, please, I will try not to.

1. seemed fast and unexpected - did the last participant just meet his year in Oct.? Then the "results" announced a month later?

2. what else will you publish and how soon?

3. Many of us have been stewing about the differences in substance between phase I and phase II in the reports. Phase I being reported in percentages of improvement, with a follow up article saying they continued to improve. Phase II was discontinued with a statement saying the placebo and treated were the same.

4. Who are the responders? Who felt better? Any patterns spotted?

paula
kindly reply

Thanks Carolyn for sharing your experiences in the CERE120 clinical trial. It might not necessarily be a bad thing that Ceregene annnouned its preliminary results so quickly. I read somewhere that if the results were good,the company was planning to move on to phase III as quickly as possible . But of course they were expecting different results. Hoping they will be further analyzing and releasing the data -- they might also find new information that could have an impact on the other gene therapy trials.

To start, my trial coordinator wrote in an email to me that "both groups (sham and treated) showed the same improvement. They compared the "off" scores at 12 months to the pre-op scores so this would have been individual scores. I would imagine more info. would be forthcoming.”

Personally, I have a hard time with this statement, as do a few other patients.

I wonder if gene therapy has more to do with the PD and the individuals brain, and maybe the surgeon. What I mean by "with the PD" is that we all are so different...some slow to progress, some significantly disabled within a few short years of dx, some with dyskinesia, some with dystonia, some with neither of the latter two.

Is is possible that gene therapy might only be a good therapy for some, while not recommended for others. Without further study and investigation, we will never know.

To "correct" some of what Paula stated above: I had my surgery on June 18,2007. I was transferred from the ICU to regular neuro on the morning of the 19th. Upon arrival to the regular neuro floor I requested a visit to the bathroom. While there I collapsed to the floor. After "taking too long" in the bathroom the nursing person decided to check on me and found me on the floor. I requested a copy of the safety report and as of this date I do not have the copy, but I will begin to pursue it more vigorously. I was told I had a seizure...not uncommon after brain surgery...and was put on anti-seizure medication. I should have gone home on the 19th, but due to this unexpected seizure I stayed until June 28th.

A safety report is the document that is filed with a government agency detailing what happened during a given incident involving a trial patient. In my case this document would tell me that an MRI was preformed to confirm the seizure, etc. I do know that my facial swelling was very bad and I am told my seizure was due to brain swelling. (The swelling would have been caused by the surgical invasion into my brain during the insertion of the therapy. Of course, neither my trial investigator nor I every spoke of this as a possible reason for the swelling. Why? Because of this was a blinded trial. I have just guessed all these months.)

I do not remember ANYTHING from the morning of the 18th until I woke up in on the Rehab floor June 25th...I lost a whole week. Those days were an ordeal for my oldest daughter, who had to deal with home care setup, etc. I could not have a conversation of any kind...just yes or no. As Paula explained above. I would start to brush my teeth, but I couldn't figure out HOW to stop brushing. My daughter had to take the brush out of my hand. This also happened with bathing. I would begin, but I didn't know how to stop bathing, turn off the water and dry myself. Suffice it to say, it was a very scarey time for my family. I was clueless, so it was not scarey for me.

On July 3rd, both my daughter's took me back to Philadelphia to see the neurosurgeon out of great concern. I was still only able to say Yes or No to anything I was asked.

I checked back into the hospital on July 3rd. I think I can home again on July 21st, but I am not confident this is the right date. I do know I was in the hospital for a month, combining both hospitalizations. This second inpatient included I was on the regular neuro floor. Those that called me on the phone will attest to how difficult it was for me to have a conversation, but as the days passed, this skill improved. By the time of my final discharge I was able to think and act normally.

I have had not problems since that last discharge on July 21st. I did spent three months on anti-seizure meds and no driving as a precaution.

Would I do it all again...yes, I would do it all again.

As we exhausted what seemed like all roads to challenge Amgen's GDNF halt, we heard about neurturin. "Word on the street" was that although neurturin was in the GDNF family, the results were not as striking. [pre-clinical research]

Later, it became a matter of who you talked to. And that's one of the purposes of clinical trials. To determine efficacy.

The cere 120 trial was actually determining much more than efficacy of neuturin. It's experimental gene therapy delivery as well.

Seems like a lot to prove. I'm glad the risks were taken and extend deep gratitude to all involved. This isn't another GDNF, at least I don't think it is. The only efficacy reports I have learned about is through word of mouth. Carolyn and one other's [rumored] capability of going off meds. And a post from Tom, who thought he improved but discovered he was on the placebo. Dottie, who is on this forum reported she was on the placebo and always thought so.

I wasn't that interested in phase III until one participant told me she went off her meds. Then I decided to go for it...a spark that soon fizzled with the announcement that only says there was no difference between placebo and treated.

All comments welcome.....trial participants I would love to hear from you.

paula

My "surgery" was June25th - a week after Carolyns' She had already fallen so my s-i-l and I went to the hospital on the 24th to see how she was doing. My participation in this trial was a nonevent after her experience.

Approximately every three months I was evaluated and lab work was done.

When there was no improvenment leading up to the 6 month evaluation
I knew i was on thhe placebo list.

I'll try to answer any questions.

Doottie

Thanks Dottie,

I wish it had done something you could feel. If rumors about a phase III possibility are true, and should that occur, are you entitled to get the actual treatment?

I really appreciate your honesty and thanks for taking the time.

paula

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