Anavex Life Sciences Corp (AVXL)

[falconer66a]

Mothers can tell.

If the roll over follows a blinded trial, and the patient, families, doctors and investigators don't know if the participant was receiving the drug or the placebo, they don't usually have much clarity on whether the drug is working or the placebo is working.

For girls suffering from Rett syndrome this should not be presumed.

For example, it is already known, from Phase I trials, that blarcamesine optimizes, improves gamma-aminobutyric acid (GABA) levels. GABA is an important modulator of motor nerve impulses to muscles. When lacking or deficient, as in Rett, control of muscles is compromised. Walking, grasping, and other common motions are deficient.

Would it take a trained physician to detect any improvement in a Rett girl’s muscular control, such as gait or grasping activities? The girl’s mother would see this instantly. She would tell her friends about it, including the mothers of other girls in the study, some of whom are in the placebo arm.

And no, the placebo effect generated in the placebo control arm of the study won’t generate such motor-control improvements. Thinking positive thoughts about the “drug” a Rett girl is taking can’t make up for the hyperexcitabilty in the nerves controlling her muscles, caused by a deficiency of GABA.

Other behavioral improvements may be similarly dichotomous. Improved sleep may be one of those.