one out of 12 ain't going to get NTCELL approved, the best it will do is possibly help convince regulators to approve and investors to fund another study once they find out what if anything was unique about this patient, was he/she in much better shape, did he/she have a much different diet, etc, etc, etc. did they accidentally place the NTCELL packets somewhere else in the brain?
keep in mind that an advanced pd'er can have good days and bad days and what is being measured is all based on what the neuro sees and feels, there is no biomarker for pd.
they might have made a mistake using patients that were less advanced. when i'm OFF i walk/freeze, my speech is slurred, i have painful rigidity in my feet, hands, shoulders, back, abdomen - i'm basically immobile. but when ON you wouldn't know i had pd, it's completely jeckyll/hyde. so it seems in the design of the experiment, where phase2b patients had a lower (better) baseline UPRDS motor score than in phase1, you just don't have the potential for as much improvement. to use myself as an example, i could feel normal taking 100mg l-dopa pills 3-4 times a day 7 years ago. but now i never feel normal taking 150mg 6 times a day but my ON UPRDS score is still almost as low as it was 7 years ago, why? because what they test is just very simple movements - walk up and down the hall 2 times, stand up from a sitting position, tap your fingers, rotate your wrists, push/pull against the neuro providing resistence, count backwards by 7 while walking. but 7 years agowhen ON i could jog, play a little soccer, play 1on1 in basketball. i can't anymore, my muscles are just too rigid. my point is that UPRDS is imho a crappy test and will miss out measuring more subtle movements, like can you make freethrows, kick a soccerball, throw a baseball, catch a ball, jump over an obstacle, dance.
the question that wasn't asked at the cc was did it stop progression.
fwiw, you can't measure dopamine in the brain directly, first of all impossible to take a sample, and basically you are measuring it indirectly via the UPRDS. dopamine is a simple amino acid that is tightly regulated in the brain, and is made in the brain from other amino acids like tyrosine. an advanced pd'er might take 1000 to 2000mg / day of l-dopa, an analog of dopamine, which doesn't pass the blood brain barrier but l-dopa does, and less than 5% gets into the brain. so just a little dopamine, less than 10mg, can make a big difference in the brain. with all the inherent variability between pd'ers, within a pd'er during the day, and not always being perfectly medicated, they needed really big differences, and no similar trials using 5x as many patients and were 52 weeks have been successful.
maybe they'll see improvement in 2 years but the problem is pd isn't isolated to 2 small areas of the brain,damage is in multiple areas that aren't being treated. plus there is a big race on to find a "cure", lots of trials right now including fetal cell transplants, stem cells, vaccines against alpha-sync.(?), gene therapies and repurposing existing drugs.
basically they needed as good an improvement while OFF as the best you can get with DBS imho and that can be absolutely amazing and it appears they didn't come close.
LCT Price at posting:
4.0¢ Sentiment: Sell Disclosure: Not Held
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