PLT Test Blog

Once anyone starts to make claims that some autism is treatable, people respond in different ways.  Those applying what has always been taught in medical school, that autism is untreatable,  will either think you are making it all up, or worse, you are some evil person taking advantage of parents in emotional distress. The very few people who read the research about things like metabolic errors and intracellular signaling may well take a different view. Also the oncology/cancer researchers who themselves think about sub-types of disease that are induced by specific signaling pathways (like RAS-induced cancers for example), may well see the sense in experimentation like that in this blog. Medicine does indeed say that autism, Down Syndrome and ID/MR are untreatable; however current science does not support this.  Your local doctor applies medicine; he is likely totally out of his depth when it comes to where science is in 2016. My posts are just my take on the science, I a...

Hardly a week goes by without somebody mentioning to me a wonder treatment or even “cure” for autism; the latest one being the GAPS diet. I think all such reports are worthy of investigation, but many lead to nowhere. Why is this?    ·        Medical science has failed to adequately define autism, so we are not all talking about the same autism ·         Many people putting forward theories have not read even the most basic (and not contested) autism research.   Some are even, apparently, qualified “doctors”.   Autism Phenotypes What is not disputed is that autism has many sub-types (phenotypes). Researchers tell us 10-20% of cases referred to as autism have a known genetic defect (Fragile X, SLOS, Timothy syndrome etc.).   80% do not have a known genetic marker/cause. Autism can be subdivided into regressive (when a child loses speech and other learnt skills) and non-regressive (early onset). ...

  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...