That is a really great question Steady. There are tens of hundreds of trials that have been ran now and have failed across many different diseases. We have enough placebo data to fill a football stadium for each disease.
That being said I would like to think that soon we will be advanced enough in our basic knowledge of safety, gaba and glutamate levels in some cases, and quality of daily living or RWD as fair starting points for a general trial with blarcasamine. Who knows the patients better than the family members? Let them be the Dr. for once and monitor their loved ones trial. After an initial consultation with a Dr. and perhaps a blood test, just allow the patient to slowly titrate up to dizziness and then backing off and use this as the trial “starting point” and then caregivers/family members keep a daily diary. Weekly checkins through zoom meetings and a monthly visit to test the blood or as necessary.
With Alzheimers I think we will have a blood test measurement within the next year or two which may allow the Dr visit be avoided all together. Just have a nurse stop by and pick up a sample and drop off your kit. Again slow titration, daily diaries, weekly, zoom visits, monthly check ups etc.
Should data continue to show efficacy, especially in the PDD with actigraph data for secondary endpoints, how awesome would that be for a loved one to journal.....” my little bobby continues to not have as many tremors week over week and actually sleeps all through the night now and actually remembers my name again after just a few months of being on the drug at maximum levels! I can finally get some rest now myself and quit worrying.”
Run everything through the non profits (after they pass the “sniff” test of who their allegiance is to) who have spent years of raising money and organizing networks compiling patients names to help combat the dsease. Completely cut out the middle person and the greedy corporate raiders. Partner with a manufacturer to supply the drug and step back and watch how humanity starts to grow.
Uhhmmm this sounds way too familiar and may just be where Anavex is headed, I may photo this post for future reflection.
Cheers
