PLT Test Blog

  Memantine, Arbaclofen  and now Bumetanide stumble in clinical trials (also the less well known Balovaptan, which Roche dropped in 2020). Place your bets on Suramin, anyone?   Plus ça change ,  plus  c'est la  même chose The more things change, the more they stay the same The first week of the school year brought two big surprises.  Monty, aged 18 with autism, came top of the class in the math test.  This is a big win for bumetanide treatment, because 9 years ago Monty was effectively innumerate.  With a huge effort by his Assistant, he had learnt how to read and write, but even the most basic maths was beyond him.  That all changed in 2012 thanks to Professor Ben-Ari’s published research on Bumetanide in autism. The sad news that week was that the Phase 3 clinical trial of Bumetanide for autism had been terminated early.    Servier and Neurochlore announce the main results of the two phase 3 clinical studies assessing...

This week I received a message: - “Your Bumetanide treatment is on trial among 25 teenagers here and parents are loving it” My reply, brief as usual (unlike my blog posts) “Great!” I did not mention that in the first phase of the trial 50% of the teenagers are going to be on the placebo.   It is Dr Ben-Ari’s treatment. A clinician told me that all the parents of children, to whom she has prescribed bumetanide, think their children are responders and is wondering how to deal with the parental placebo effect. I had another clinician telling me, “I guess from your experience with the blog, most people are not responders to Bumetanide”.   Then came an analysis of the recent tiny study in China that showed on average there was a measurable improvement on the CARS scale (Childhood Autism Rating Scale), but the question arose was “is this response large enough for parents to notice?” Memantine (Namenda) A few years ago, Memantine was also trialled at the University Hospital where we...

Two autism therapies mentioned in this blog have recently failed in their clinical trials. The selective mGluR5 antagonist mavoglurant failed in two trials funded by Roche and Coronado Biosciences threw in the towel with its Trichuris suis ova (“TSO”) program.  TSO are parasites that are introduced to the gut to modify the immune response, they are thought to help conditions like ulcerative colitis and some autism. "Coronado Biosciences (NASDAQ:   CNDO ) has decided to no longer pursue the development of its Trichuris suis ova (“TSO”) program. The Company is terminating all on-going TSO trials, including the Company’s Phase 2A clinical trial of TSO in pediatric patients with autism spectrum disorder. A preliminary analysis of data from this trial failed to demonstrate any signal of activity." The original user of TSO in autism documented his case here:- http://autismtso.com/ It has been a long time since the father updated his site. Does he still give TSO to his son? This ad...

Only a few months goes by without there being an uplifting report in the media of some breakthrough drug for autism.  These reports usually relate to research on mice. So where are the resulting approved drugs for use on humans? There still are no drugs approved for the core symptoms of autism.   It is quite likely that in spite of all the ongoing research, the situation will not change anytime soon. I was reading about yet another potential wonder treatment, based on research into a very old drug called Suramin .   This rather toxic drug has been shown to be effective in a particular mouse model of autism call MIA (Maternal Immune Activation).  There is some doubt as to whether the researchers have got the method of action correct, but nobody doubts the positive effect it had on some mice. Today’s post does not look at the science of Suramin, which is, by the way, another anti-parasite drug like Ivermectin , which I looked at earlier.  The subject of this ...

  In the world of clinical trials for drugs, judging success and failure can be highly subjective.   They try to make it as logical as possible and the method works pretty well for assessing things that you can measure objectively. Primary and Secondary Endpoints To quote Pfizer: A trial endpoint of a clinical trial should fulfill three criteria: (1) be measurable and interpretable , (2) sensitive to the objective of the trial, and (3) clinically relevant . The endpoint can be either clinical or surrogate in nature. If you are developing a drug to lower cholesterol or to increase survivability after a traumatic brain injury, it is pretty easy to define your endpoints. When it comes to autism, one of the major hurdles is to define objective measurable endpoints.   As it stands today, none of the assessment tools are really fit for purpose, when Big Pharma is supposed to come along and invest hundreds of millions of dollars in some bright spark’s idea. Arbaclofen, Seaside P...