Hi Skint,
Hopefully your not reading my responses in a negative, talking down to you, tone, as that is not my aim. In fact its great that you have proposed these views and inputs, as it forms great discussion and promotes further research and understanding for all. If you were completely ignorant that would be a different story, and that is not how I read your current posts.
Hmmm, I see your point, although they would, I assume, and strongly hope, already, have been scored quite thoroughly through the UMSARS and biomarkers pre commencement of trial, and have a number of biomarker tests to show significant differences from placebo to ATH treatment, right throughout the study.
Another company to look at which I have a small holding with ATM is PAA, soon to change name, which has also been given OOD by the FDA for the treatment of MND (motor neuron disease), as the drug monepantol has reduced the timeframe of death by MND by over 2yrs now, in which every candidate should have been unfortunately deceased by now. As there is no drug to slow/halt or reverse progression, they have now progressed to an upcoming Healy study.
So as ATH has shown in a small number of candidates to date that there has been a reduction in a number of areas, it should work better in those less affected by MSA than those long term sufferers that were trialled in 202, and treatment is better than alternitive, hence why every drug under goes a rigorous safety profile. In which Dr Stamler had requested an independent review of the safety concerns with ATH.
I feel that even if we get a success rate of 49% (hypothetically), would it not be beneficial to help 49% of the general MSA population, or just say nope, let everyone just go on...hopefully the FDA have a bit more of a heart than to say no. But hopefully we get a much higher percentage response.
My reference to the actions of ATH434, was to your reference of the pre and post synaptic uptake. The synaptic gaps require a specific gap between each nerve ending to allow vessicles to be released and uptake between nerve endings, which is also how certain drugs are used. If asynuclien clumping due to excess ferrous iron, causes the nerves/cells to strink (atrophied) than the gap widens reducing the pre and post synaptic release and uptake, which causes a number of issues and ailments.
For example, midazolam should not be given to, or have a significant higher level of caution, pts with MS (multiple sclerosis), as midazolam is metabolised at these locations. Due to MS nerve damage, the midazolam remains active alot longer, resulting in significantly greater risk of paralysis and airway management, and longer recover post administration. Therefore visualising the images posted, if the nerves shrink (atropthy)/die, the chemical vesicles cannot be released or be uptaken by the recieving nerve ending.
Hope this makes sense.
Overall I see your point, but I believe they would be all over this, and I feel the scoring would not change if they have been on levodopa for some time or from the time of trial commencement compared to ATH treatment and other biomarkers, hopefully I am not proven wrong.
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