Can somebody advise...does the company actually read these chatlines.
The ramifications here based on this information BELOW, from a leading US hospital - are not open for debate by a couple of people on a chatline.
Words fail me as to what should be the immediate implications for the KZA drug and for the people who it can help. (and issues other than the SP)
I am not saying their drug paxalisib will be approved tomorrow. But this is still massive massive stuff - This type of information gets rolled out and then quickly forgotten.
I produced information here which confirmed response in some of the worlds leading diseases, clearly showing response in much lessor drugs, in the same class as paxalisib - what has happened since. The same as what always happens - nothing.
This is ridiculous. A stop needs to be made to a very wrong situation that has developed.
Don't agree - well read below and spare a thought for a possible situation where the company you have invested in....... are not giving people a chance they deserve. This is a big deal.
Suppression of PIK3CA-driven epileptiform activity by acute pathway control
View ORCID ProfileAchira Roy, Victor Z. Han, View ORCID ProfileAngela M. Bard, View ORCID ProfileDevin T. Wehle, View ORCID ProfileStephen E. P. Smith, View ORCID ProfileJan-Marino Ramirez, View ORCID ProfileFranck Kalume, View ORCID ProfileKathleen J. Millen
doi: https://doi.org/10.1101/2021.03.03.433821
This article is a preprint and has not been certified by peer review [what does this mean?].
000007
ABSTRACT
Patients harboring mutations in the PI3K-AKT-MTOR signaling pathway often develop a spectrum of neurodevelopmental disorders including epilepsy. A significant proportion of them remain unresponsive to conventional anti-seizure medications. Understanding mutation-specific pathophysiology is thus critical for molecularly targeted therapies. We previously determined that mouse models expressing patient-related activating mutation in PIK3CA are epileptic and acutely treatable with PI3K inhibition, irrespective of dysmorphology (Roy et al. 2015). Using the same mutant model, we have now identified physiological mechanisms underlying the dysregulated neuronal excitability and its acute attenuation. We show that Pik3ca-driven hyperexcitability in hippocampal pyramidal neurons is mediated by changes in multiple non-synaptic, cell-intrinsic properties. These are distinct from mechanisms driving epilepsy in TSC/RHEB models. Further, we report that acute inhibition of PI3K or AKT, but not MTOR, suppresses the intrinsic epileptiform nature of the mutant neurons. These data represent an important step towards precision therapeutics against intractable epilepsy, using pathway drugs originally developed as anti-cancer agents.
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