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Showing posts with the label NF-1

PAK inhibitors and potentially treating some Autism using Grandpa’s Medicine Cabinet

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I wrote several posts about why PAK1 inhibitors should be beneficial in some autism and indeed some schizophrenia. We also saw that PAK1-blocking drugs could be potentially useful for the treatment of neurofibromatosis type 2, in addition to RAS-induced cancers and neurofibromatosis type 1. One problem with drugs developed for cancer is that, even if they finally get approved, they tend to be ultra-expensive.  Production volumes are low because even if they “work” they do not prolong life for so long and cancer has numerous sub-types. Cheap drugs are ones used to treat common chronic conditions like high blood pressure, high cholesterol and indeed treatment of male lower urinary tract symptoms (LUTS), like benign prostatic hyperplasia (BPH). A small number of readers of this blog have confirmed the beneficial effect of PAK inhibitors in their specific sub-types of autism.  The problem is that there are no potent PAK1 inhibitors suitable for long term use that are readily avail...

RAS signaling, Autism, Cancer and Gingerols

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Sytrinol (Tangeretin), sacrificial Gummy Bear and Gingerol Today’s post follows on from an earlier one that introduced the term RASopathy.  A RASopathy is a disease characterized by over-activation of the RAS protein. RASopathies are of interest because if you have one, you are highly likely to also have autism. RAS dysfunction is also present in many types of cancer and there are existing drugs to inhibit RAS signaling.  It has been claimed that:- "If RAS proves to be a key player in autism …  it might suggest new treatments for autism, as many cancer drugs inhibit RAS signaling." Regular readers of the Simons Foundation autism blog may have read the following: RAS pathway, a potentially unifying theory of autism     Cancer pathway connects autism to set of rare disorders If RAS proves to be a key player in autism, she says, it might suggest new treatments for autism, as many cancer drugs inhibit RAS signaling. RAS-based interventions My Polypill already has o...