What, then, in seven months, or seven years?
Yes, wonderful therapeutic results appeared in the data from the six girls participating in the early, very short Rett syndrome clinical trial of blarcamesine; as noted, here, in the posting from which this refers:
RSBQ Total from 59 to 14 in just 7 weeks
RSBQ Hand Behaviours Subscale from 5 to zero in just 7 weeks
RSBQ Breathing Problems Subscale from 9 to 4 in just 7 weeks
% Change in Glutamate 66% reduction from baseline in just 7 week
CSHQ (Sleep) Total 20 in just 7 weeks
Glutamate µmol/L 11 µmol/L of Glutamate in just 7 weeks
% Change CSHQ Waking 1% decrease during the Night in just 7 weeks
% Change in RSBQ Total 76% reduction in just 7 weeks
% Change in GABA 80% increase in just 7 weeks
Seven weeks, about 49 days, of blarcamesine treatment caused these favorable metrics.
What would happen, however, if these girls continued their blarcamesine treatments on out chronically (in long-term continuation), first for seven months, than on out to seven years? Only three trends would be likely:
Trend A. Treatments are optimized, fully achieved after just seven weeks; but no further symptomatic improvements after that period. Continuing dosings yield no subsequent improvements. Blarcamesine reaches its full therapeutic potential in dosing periods no longer than seven weeks. No improvements after that.
Trend B. After the initial seven weeks of dosing (as shown in the trial), symptoms continue to be even more strongly suppressed on through lengthy, extended treatment periods, from seven months to seven years. Longer, time-dependent treatment periods produce stronger therapeutic results. Longer is better.
Trend C. After about seven weeks of blarcamesine dosing, after clinical metrics have improved, symptoms reverse and continue to get worse with extended blarcamesine dosings beyond seven weeks. Blarcamesine works only for short, initial dosing periods; after that things revert back to the pre-dosed pathologies.
No evidence for either A or C. The trial was too short to determine the effects, if any, of much longer, even chronic (continuing) dosing periods.
The key, for Trend B (continuing long-term symptomatic improvements) would be the drug's ability to restore debilitated organelles and cellular functions. That might well involve a period longer than seven weeks. Nerves (when they do), don't grow very fast at all. It may take many months, even several years, for blarcamesine to induce cytological "healing," with new, fully-functioning organelles and biochemical reaction sequences.
If blarcamesine can produce only Trend A, bring about favorable outcomes within short dosing periods, because of its attendant safety it will be a useful Rett syndrome therapeutic in older females with the condition. Good enough.
But, very likely, with continued, longer dosing periods, Trend B will occur. After several months, on out to several years, neurons debilitated by Rett cytological anomalies might be even more fully "repaired" by blarcamesine.
Or, at length, the drug may only prevent the worsening of Rett syndromes, as the girls age. Either way, every girl with the disease would benefit.
Of course, all of this needs to be considered for each of the other central nervous system diseases blarcamesine is being tested against. At length, might blarcamesine continue to reduce CNS disease symptoms, in time approaching normalcy?
All of this points to another potential blarcamesine process; prophylaxis, prevention, of either worsening disease symptoms or their complete prevention. For Rett, what is the likelihood that very young girls, administered blarcamesine when first discovered in them, might avoid progression to severe (and ultimately lethal) outcomes? Same for Alzheimer's and Parkinson's; prevention or progress suppression.
Given blarcamesine's unique mechanisms of action within neurons, these are all very possible.
(And, I bet that Anavex has checked all of this in murines. If blarcamesine can prevent the onset or progression of CNS diseases in murines, lab rodents, it can do that in humans.)