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Monday, 13 March 2017

Meaningful Autism Treatments?




 Edinburgh

The Simons Foundation just keeps giving to fund autism research.  They have just given $25 million to the university in Edinburgh, Scotland.
Big donations are not rare in the US; the CEO of Broadcom recently gave $20 million to fund autism research at MIT.
The United Kingdom has very highly rated medical research, but not when it comes to autism.  The US is by far the dominant centre for the study of autism and I expect China to become the clear, if distant, number two.
I liked the quote that was attributed to Mr Simons, even if it was written by the PR department:-

"We are confident that the great scientists already in place, coupled with the comprehensive facility being developed, will accelerate understanding of autism and hasten the development of meaningful treatments."


Meaningful Treatments

How would you know a Meaningful Treatment if you stumbled upon one?
It does help if you know what you are looking for.
To the general public a Meaningful Treatment is seen automatically as a “cure”. 
This then implies you just need one and you are “fixed”.
For most people with autism, Meaningful Treatments will be just incremental steps towards a better life.  Cognitive function will be key in severe autism, but irrelevant to many with Asperger’s.
Controlling tics might be life changing for some, for others the key might be halting aggression and self-injury.
In the new mild autism the issues are more subtle and varied, but when things go wrong the result can be more dramatic than in severe autism. This is the group that sometimes does not want treatment.
I hope Mr Simons knows what he is looking for. He has given so much money.


This Blog
There are many Meaningful Treatments mentioned in this blog, but for the great majority there are no cures.
If in severe autism you can raise IQ by 30 points, I think that is terrific, to use a Trumpism, but you will still be autistic and still be more autistic than a totally untreated person with Asperger’s. So to most observers, not much of a recovery at all.





9 comments:

  1. All this calls for personalized medicine and biomarkers/subgroups based studies.

    It is good to hear about new million dollar research in autism and at the same time there is something strange when you compare it with Bumetanide cost, less than 2 EUR per month.

    New multicenter Bumetanide RCT results have just been published:
    https://www.ncbi.nlm.nih.gov/pubmed/28291262

    With the conclusion: "Bumetanide improves the core symptoms of ASD and presents a favorable benefit/risk ratio particularly at 1.0 mg twice daily."

    Peter, do you know if this is enough for the French team to get the EU Bumetanide approval for autism?












    ReplyDelete
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    1. Agnieszka, if you read the full text of the study it refers to the next step being the phase III trial. They are dropping the 2mg twice a day dose, even though it gave the best result.

      Probably once the parents have figured out how manage the side effects, the 2mg twice a day is the better dose for adult sized people.

      http://www.nature.com/tp/journal/v7/n3/full/tp201710a.html

      The current study is based on data now a year old.

      Delete
  2. Tyler, I wanted to ask you about l serine,what are your thoughts about it, if it can be added to my son´s BCAAs treatment. I don´t know if it helps to decrease dopamine levels and or is an NMDA agonist.I also wanted to tell you that my son is very thin and don´t know if it because of BCAAs,he can´t gain weight.Apart from this, I would like to try l serine, but in addition to my folate deficency I have the V Leiden Factor, a clotting disorder and I think I can´t take it.I am not taking warfarin.
    Valentina

    ReplyDelete
    Replies
    1. Well first of all, I am not sure which "Serine" you are talking about. Serine is just an amino acid, but depending on its chirality (whether it is D isoform or L isoform) it can have much different effects on body chemistry. L-Serine is a non-essential amino acid which is naturally produced in the body and D-Serine which is also naturally produced in the human body (although at much lower levels) is a powerful neuromodulator of the glycine site of the NMDA receptor.

      In the paper I mentioned a few posts back, I believe they were just using L-Serine and not for any direct neuromodulatory reason, rather more for its role in glutathione homeostasis. When people talk about serine and autism or schizophrenia, they are usually talking about D-Serine or else get confused with D-Cycloserine which is a totally different thing.

      I would not take D-Serine unless you want to potentiate NMDA receptors which you probably would not want to do in a broad way as reducing glutamatergic hyperactivity (i.e. NMDA antagonists or negative allosteric modulators) is where most of the approaches seem to be (or so I have read).

      Delete
    2. Tyler,ok, D Serine can be a very dangerous approach, on the contrary, L Serine could work in my son as it is a motor neuron protector and also boosts the immune system.
      Valentina

      Delete
  3. Thanks, I didn't notice this. There's another Bumetanide trial listed here:

    https://clinicaltrials.gov/ct2/show/NCT02947880

    "Evaluation of the Efficiency of Treatment by BUMETANIDE on Autistic Children With a Known Ethiology"

    Is that phase III? What does "known ethiology" means here? The trial ends in 2020. It's a long time again.

    The good news is that no child in the the published study developed arrhythmia or neuromuscular complications due to potassium loss even with higher dose. And they suggest that a gradual Bumetanide dose increase might reduce hypokalemia risk. I also increased the dose gradually with no potassium issues.

    I believe all adverse events reported are manageable and the good effect is worth the effort needed to control hydration or prepare the child psychologically for increased diuresis.

    ReplyDelete
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    1. It say phase 2. It also says CARS>= 30 which is not the severe autism as in the current trial. The EMA must have told them to start with severe autism.

      The current trial was not registered on that site.

      One reader of this blog was offered to join a trial in Spain, so I think they may have multiple trials in progress.

      Delete
  4. Well it seems like it's me, not only my son, who needs additional reading lessons ;-)

    I just hope very much for the moment of official approval of Bumetanide for core autism symptoms. Personally I don't need it as in paediatrics many drugs are used off label, so why not Bumetanide, but neither my opinion nor documented IQ increase in my son is an argument for those who stick to "autism is untreatable", "different brain wiring" etc mantra.

    In the current study, withdrawal rate due to adverse events with the highest dose was 27%. On one hand it is high number, on the other it means that the majority of children completed the trial even with 2 mg twice daily. So, as you pointed, it might be worth considering in older ones.

    ReplyDelete
  5. We can no more treat Autism than we can treat "Fever" - we need to focus on treating the myriad underlying CAUSES of behavior not the symptom.

    ReplyDelete

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