Brilliant assessment boi568 and follow up, thank you. The learning process must and will continue from here. IMO, Dr.M. is already working down-along these lines. In the meantime, many patients and their families will be involved in each process step, some may have a longer path than others. This, IMO, is at least civilized clinical-medical science w/an excellent focus with a true clinical knowledge blessing to follow. This is how work gets done. Brilliant leadership is required. We may even see a new CNS disease FDA start to contribute, IMO.
I am not qualified to assess any endpoints yet, but I do recognize a new standard of/for processing excellence . Thanks very much for your work. WGT.
One of the inclusion criteria for the Anavex Rett trials is classic RTT.
“Diagnosis of classic RTT, according to 2010 criteria (Neul et al., 2010), and a MECP2 mutation.”
Because of course one wants homogeneity in the clinical trial population not to confound the results with too many variables. One reason that enrollment particularly for a rare disease can take time.
Very interesting and thoughtful post. You have hit the nail on the head. Diagnostic criteria in most CNS diseases are based on symptomatic descriptions rather than biometric criteria. Those symptoms have been shown to have a multiple of underlying biological causes resulting in the same or very similar symptoms. A very similar situation has been discovered in cancers. What appeared to be similar cancers in the same region of tissues has been found to be a result of different biochemical and genetic pathways, And of course have need a different treatment approach.
Anavex 2-73 being an upstream approach to treatment may well be a more general pathway treatment than previously attempted treatments. Will it be the ultimate treatment for these CNS disease? I think not. Will it be a paradigm changing approach to CNS treatments, that seems to be a high likelihood.
Additional drugs will come down the pike based on the new paradigm demonstrated by 2-73. They will be more specifically targeted than 2-73. But who knows. 2-73 or 3-71 may become the aspirin of CNS or as we have hoped a general old age tonic.
Quote: "I think this new neurodevelopmental patent application serves two purposes. First, it redefines the medical use of 2-73 on the basis of symptoms and biomarkers, rather than disease names. Second, it restarts the full patent protection clock on the medical use of 2-73 for this category of diseases.
Hello, 2040's."
Hmm...me thinks the Millionaires List is going to be quite large next year!