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The PRECEPT clinical trial, with
CEP-1347, was watched with great hope. Pre-clinical trials indicated
it was neuroprotective. Had scientists finally come up with
something that would slow or even stop the progression of
Parkinson's disease? That was their hope, and mine as a trial
participant.
I researched CEP-1347 extensively prior to
joining the Phase II/III clinical trial. Everything indicated that
it could be a winner. What did I have to lose? Okay, there was an
increased risk of cancer because CEP-1347 stopped the mechanism for
cell death. This was a very scary possibility, but I reasoned that I
already HAD Parkinson's disease, and cancer was only a remote
possibility. I decided to do what I could NOW to fight Parkinson's
disease, and not let the cancer threat deter me. What I had to gain
was the neuroprotection: if CEP-1347 worked as hoped, I’d have the
chance to get this new treatment years before it came on the market.
I had a 75% chance of getting the drug (and a 25% chance for
placebo). So my chances of getting CEP-1347 were good, and oh,
how I hoped I would.
In May 2005, after I’d been in the trial for
nearly two years, it was abruptly halted, following scientists’
review of the 806 participants’ data after averaging 21 months on
CEP-1347. Cephalon posted a notice on its website that the
drug “was unlikely to provide evidence of significant effect,” [1]
and the trial ended just like that.
Now, 10 months later, the trial has been
unblinded. The Parkinson Study Group sent letters to their trial
centers, and participants from all over the country are learning
what dose they were on and the effects of CEP-1347. To
everyone’s great surprise: “more participants who were assigned to
CEP-1347 developed disability requiring dopaminergic treatment
(dopamine agonist or levodopa) than those who were assigned to
matching placebo.” [2] The results were confirmed by the two
SPECT (brain) scans each participant received. In plain terms,
Parkinson's disease progressed slightly faster in participants on
the drug, than it did in those on a placebo. This was the complete
opposite of what had been expected. Instead of being neuroprotective,
CEP-1347 actually caused you to get worse faster! (Perhaps
unsurprisingly, one of the people I know who did the best in the
trial found out she was on the placebo.)
One researcher who wrote to me said, “the
effects are minimal, but they appear to be real”…and … “they are not
‘statistically significant’ and “therefore, we do not put tremendous
weight on them.” [3] But the participants see the findings
differently. The majority of those with whom I am in contact are
very upset that the drug caused faster disease progression, no
matter.
These results illustrate that there is no
guarantee about the results when you join a clinical trial. Even
though I had done extensive research before joining PRECEPT,
learning the drug had shown great promise – it not only failed to be
neuroprotective, it appears to have caused faster progression of
Parkinson's disease.
Regardless, I am philosophical about my
participation in the trial. If CEP-1347 had worked as expected, I’d
have been a jubilant winner, as would have been my cohorts. As it
is, I admit I was glad to learn that I had been taking the lowest
dose. But in the end, this experience won't keep me from
joining future trial(s). I’ll do the same extensive research and
take my chances.
[1]
http://www.prnewswire.com/cgi-bin/stories.pl?ACCT=104&STORY=/www/story/05-11-2005/0003595559&EDATE=
[2] March 2006 Letter to Jean Burns from PSG
[3] Private correspondence to Jean Burns
from a researcher
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