Dottie, How did you do on CERE-120? I know the trial hasn't been unblinded, but did you feel like you got the treatment? Or did you think you got the placebo / sham surgery?

take care

I know you felt you got the real thing. You are totally off meds now, right?

What does this mean for the rest of us?? Is our only chance for a new treatment to take part in a clinical trial - because even if it works (for some) the majority don't get benefit ... so it turns out that only a few "lucky ones" in the trial get something that might work?

Is Parkinson's so different with each person that these substances only work on a small subset of pwp?

just wondering...

this may be old news to folks - i tune in so rarely i am a little out of touch, i am sure - but it appears that there are different types of mutations that cause different types of breast cancer - one mutation, HER2, which results in a particularly aggressive cancer, is responsible for 25% of breast cancer cases, and a drug, herceptin, has been developed to treat HER2 cancer specifically. in clinical trials, women treated with herceptin for one year had - depending on what other therapies they also had - a 33-52% lower rate of recurrence.

so, it is possible that what we call a single disease can arise from multiple sources, and it is possible to identify and treat some - and, in theory, eventually all - of those sources.

i believe this is where interest in pd subtypes comes from, in addition to the search for genetic clues.

Hi Dottie, I was reading about your experience in the clinical trial. Did this help you get off your meds? Thanks

Hii Jim -
My meds haven't been reduced -- I'm taking the same meds I've taken for years.

Duri ng the trial I was given Lorazepan so that I could have an MRI.


So, no MRI/no Lorazepan.

Boann,

Yes - and the variability is a huge problem for us. Because trial after trial will "not meet their end points" for the majority of the trial participants, but SOME of the participants will do well, and endpoints WILL be met for certain individuals.

Carolyn got neurturin in the CERE-120 phase II trial, and it DID work for her. It just didn't work for the majority of the participants.

Spheremine is another such example. It worked for Peg.

So what to do? What to do? I see promising treatments not meeting endpoints. And for some of us is a real loss because it appears that these treatments would have helped at lease SOME of us.

But how do we prove that? How are we tested to know who will be helped by what treatments?

This is a huge problem.

and have one quick comment or question for something GregW said in the MJFF disappointment thread, i.e., that CERE 120 had failed to meet its endpoint.

The press release I read from Ceregene didn't say the trial failed to meet endpoints, it said:

"The trial did not demonstrate an appreciable difference between patients treated with CERE-120 versus those in the control group."

it also said:

"Both groups showed an approximate 7 point improvement in the protocol-defined primary endpoint (Unified Parkinson's Disease Rating Scale- motor off score at 12 months), relative to a mean at baseline of approximately 39 points. "

That's an 18% improvement, on average.

The rotigotine transdermal patch trial published in 2007 had as its primary end point:

"minimum of 20% decrease in the combined Unified Parkinson's Disease Rating Scale Part II and Part III scores."

So a 20% improvement is not unheard of as an endpoint.

So, the fact that the CERE-120 group and the placebo group improved by the same amount in and of itself, in my opinion, does not necessarily indicate that CERE 120 is not effective. – degree of improvement should also be considered - what if both groups had improved by 50%?

At what point do we consider the possibility that the placebo effect might be as effective as the actual treatment as opposed to the view that the actual treatment is no better than the placebo effect? The former acknowledges both as potentially beneficial, while the latter dismisses both as useless.

Rosie:

"At what point do we consider the possibility that the placebo effect might be as effective as the actual treatment as opposed to the view that the actual treatment is no better than the placebo effect? The former acknowledges both as potentially beneficial, while the latter dismisses both as useless. "

ABSOLUTELY!!!!

The fact that the placebo effect appears to be HUGE in PD patients is something that should be pursued. I think it is a factor in our lives beyond its appearance in drug trials. How many of us use denial to our great benefit? I find it very beneficial in my life - denial - the biggest placebo of them all!

he had seen the placebo effect cause improvements in Dat scan results which should be totally objective.

He takes the placebo effect seriously and told me he is always at pains to appear totally self confident, his thinking, if the PWP sees a confident Neuro then they will share that confidence/placebo and hence improve.

Neil.

<PD, its all in your head>.

Hi Jean - At the time of my six month evaluation, I felt that I had
received the placebo/sham surgery. This was verified last week.

Dottie