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Steady_T

12/01/17 11:24 AM

#132537 RE: falconer66a #132535

The placebo effect is more complicated than your explanation.

In many cases there have been physiological changes recorded upon administration of a placebo.

From the New England Journal of Medicine....

Placebo effects rely on complex neurobiologic mechanisms involving neurotransmitters (e.g., endorphins, cannabinoids, and dopamine) and activation of specific, quantifiable, and relevant areas of the brain (e.g., prefrontal cortex, anterior insula, rostral anterior cingulate cortex, and amygdala in placebo analgesia).1 Many common medications also act through these pathways. In addition, genetic signatures of patients who are likely to respond to placebos are beginning to be identified.2 Such basic mechanistic discoveries have greatly enhanced the credibility of placebo effects. Moreover, recent clinical research into placebo effects has provided compelling evidence that these effects are genuine biopsychosocial phenomena that represent more than simply spontaneous remission, normal symptom fluctuations, and regression to the mean.1

Second, placebo effects are not just about dummy pills: the effects of symbols and clinician interactions can dramatically enhance the effectiveness of pharmaceuticals. For example, a recent study of episodic migraine demonstrated that when patients took rizatriptan (10 mg) that was labeled “placebo” (a treatment that theoretically had “pure pharmacologic effects”), the outcomes did not differ from those in patients given placebos deceptively labeled “rizatriptan” (pure expectation effect). However, when ritzatriptan was correctly labeled “rizatriptan,” its analgesic effect increased by 50%.4 Similar results have been observed when other drugs, including morphine, fentanyl, and diazepam, have been administered openly and covertly and with procedures such as deep-brain stimulation for mobility symptoms in Parkinson's disease.

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tredenwater2

12/01/17 1:43 PM

#132549 RE: falconer66a #132535



"Very similar with Rett syndrome patients. First, these are neurologically compromised little girls. Their ability, at least in some cases, to understand that pill they will be taking should suppress their epileptic fits and motor neuron hyperactivities and poor muscle control is minimal, at best. Hard to conceive how they could formulate the expectations of improved health required for the placebo effect to actually happen."

Pill? Falconer how about they arent even told they are taking a medication that is going to make them better......it is just administered to them by their parent.

See question #4 below at http://rettsyndrometrial.com/

4 ) Is your child with Rett syndrome able to take a small amount of liquid formulation (tablespoon) of investigational medicine, which can be mixed into juice or applesauce or other light food, once daily?
Yes
No
Don't know


Now if they are under a some sort of observation period documenting their daily disease symptoms and then a 4 year old is given a dose in a drink without knowing and improvement is observed (maybe drastic improvement in some cases), what is the parent/caregiver going to ask the FDA, Anavex, CRO to do at the end or during an unmasking of the trial?

I agree with Falconer that a placebo would not be applicable to a toddler, maybe candy but not a placebo.

Tred
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baltimorebullet

12/01/17 4:56 PM

#132572 RE: falconer66a #132535

I understand what you're saying but the reporting is to be done by caregivers. Probably moms and dads. People who might give everything they have to see their little girl improve.
I would never accuse them of lying but neither would I fault them for seeing what they're so desperate to see.

I'm not nearly as big a fan of the caregiver aspect as some on this board are and I expect to see scandals and/or questionable drugs approved because of it.
It should stay science only. precision to be sure, but just science.
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frrol

12/01/17 9:27 PM

#132586 RE: falconer66a #132535

No. The placebo effect is relevant to the patient and care givers and investigators. Hence double blinding.