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Wednesday, 9 October 2019

Treating Autism – The State of Play


Image © Acabashi; Creative Commons CC-BY-SA 4.0 Source: Wikimedia Commons


I have learnt a great deal since I started to research autism treatment in 2012 and publish my notes in this blog from 2013. Prior to 2012 I had assumed that autism was not medically treatable and that there was no evidence to the contrary, so anyone claiming to treat autism must be a quack.  You are supposed to "trust your doctor" and I come from a family full of them.  It turns out that almost all treatments for conditions of the brain, ranging from depression to Multiple Sclerosis to dementia are only very partially effective, but in most cases they are better than nothing. Apply these expectations to treating autism and then read the medical research and clinical trials; you will see that autism is indeed as treatable.  

It turns out that many people have found effective medical therapies for their autism. But autism is not viewed as a public health emergency, like HIV or Ebola and no public health authority shows much interest. Don't expect that to change.

The leaves are beginning to fall and we are on our way to 2020, much time has passed but has the wider world caught on and moved at all?  Not really.

  • Enthusiastic autism pharma start-ups come and go, losing hundreds of millions of dollars
  • Autism therapies are fast-tracked by the FDA, then go nowhere fast
  • Autism treatment case histories, using existing drugs, get published but are ignored
  • Partially successful autism clinical trials of existing drugs keep getting published and are then ignored 
  • Some very bright clinicians do not publish their successful work in treating autism
  • Clinicians successfully treating their own children with autism often choose to remain silent
  • A tiny number of mainstream medical doctors do treat autism, but some cleverly call it something less controversial, like encephalopathy
  • Many of the “doctors” who do treat autism are not doctors of medicine (i.e. an MD).  This is the case in North America where the "doctor" can be a DO (Osteopathy) or a DC (Chiropractic), for example Dr Nemenchek is a DO.  I think this is partly where the crank issue has arisen from; many MDs do not seem to like DOs and DCs, particularly if they write books with the word miracle on the front cover.   
  • Treating idiopathic autism is still viewed as a dangerous/crank activity by mainstream medicine; the  best view is fringe and the worst is cringe.
  • Treating single gene autism is now viewed quite favorably, it is somehow more acceptable that treating equally severe idiopathic autism.  It attracts at least some MDs, rather than DOs and DCs.  It is seen as something clever, like treating cancer.
  • By shifting autism to a more trivial condition, by diagnosing so many people, it appears to not even need treating
  • Many things do look very odd in the world of autism. People with a sibling or child with Autism/MR/ID can react very differently, some are inspired to help others and some even look to develop medical treatments, while others either never tried, failed or gave up and are now against medical treatment (in the UK Psychologist Simon Barons Cohen and author/GP Dr Fitzpatrick, for example). The idea seems to be that because they could not help their family member, you should not try.  Very defeatist. People should declare their inherent bias and indeed their own psychiatric diagnosis, if they have one, that would rule out 90% of what is written about autism, much of which is nonsense. In the wider world, you learn from your failures, strive for success and never give up.  That is how progress is made.  Those who have failed, or given up, should move aside and give space to the next generation who will achieve more.  If it is a disease or disability, you treat it; you do not just hide it by broadening the diagnosis to include trivial cases and then "celebrate" it as just a difference.

Hopefully, Neurochlore and Servier will get Bumetanide approved for autism in Europe in the next few years.

Hopefully, doctors will actually prescribe it to European toddlers through to adults with autism.

Only then will mainstream doctors stop saying you cannot treat core autism with a pill. In reality there is evidence that dozens of pills are potentially beneficial, but there is no sure-fire way to predict which will be helpful in one specific person. 

It has been clear for years, to anyone who chooses to actually read the medical research, that many cheap existing pills can be repurposed for various sub-types of autism.  In almost all cases, polytherapy will be needed and treatment leads to improvement rather than cure. This is also the case with MS, epilepsy, depression etc.

I hope that by 2030 at least monotherapy for core autism with MR/ID will be in the mainstream.  In the meantime a small number of people, with otherwise severe autism, will continue to benefit from their science-based personalized medical treatment.




20 comments:

  1. What we diagnose as autism is not diagnosed by testing for biomarkers,but by a very limited set of behaviours.Behaviours that do not include many of the most severe neurological deficits of autism.

    Autism not only is very heterogeneous,with a wide range severity,but it has an equally broad range of causes as well.The idea is slowly being accepted that much of regressive autism is caused by inborn immune disorders.But these disorders can be very different from one individual to the next.In one patient it might be PANDAS or PANS.In another it might be Mast Cell disease.In another it might be something rare like a DNA repair disorder,like I have.The idea of autism as an immune disease,is only just starting to shed the stigma it was given by the antivaccine movement.

    With a few exceptions,such as Dr. Ramaekers,in Belgium,the majority of the work in this area,seems to have been done in the USA.

    There can be no question,though,that by making the definition of autism so broad,so as to include so many mild,and questionable cases,that it both cheapens the definition of what autism is,and drains research away from both severe autism,and regressive autism.The fact that the neurodiversity movement has become so large,and so vocal,they are succeeding in redefining what autism is.That autism,and mental illness,is a social issue,and not a medical condition.If autism is only shown as a mild condition,indeed a positive thing,there is no need to research causes and treatments.One cannot help but wonder how many doctors are sympathetic to the neurodiverse view of autism.

    I do think a big step in the right direction,would be do redefine autism in a meaningful way.If autism truly is only what it says in the DSM,then the neurodiversity/Asperger model of autism would fit the diagnosis perfectly.In my ideal world,the autism diagnosis criteria would state that a diagnosis of autism should be ruled out,if there is profound intellectual disability,and any number of medical conditions.It makes no sense to tack all these "comorbidities" onto autism.

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    1. 'Systemic' diseases are much more of a challenge to find treatments for as things are going wrong in several body systems and most doctors/researchers specialize in only one of them.

      We can of course see autism as a number of comorbidities with specific symptoms and specific treatments, this makes it a little bit more practical to approach. But then you miss that clusters of symptoms can be caused by the same upstream dysfunction. Many genetic causes of autism, like those affecting a specific receptor, will result in ID, epilepsy, gut issues and immune dysfunction as well. The microbiota transplant study showed us that fixing a bad gut fixed autism characteristics as well. If we had the whole picture and knew all the pathways, we wouldn't call these comorbidities as they belong to the same pathology.

      /Ling

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  2. Yes the lack of translational research for autism is quite disconcerting. Also, the "silver bullet" idea for treating autism with a single blockbuster drug that cures autism is unrealistic, yet that is what big pharmaceutical companies are looking for when they decide whether to invest in drug development. The fact that any investment in autism specific drugs has occurred is remarkable in light of how the system works.

    The other problem is that moderate to severe autism will cause financial hardship on a family in the form of money spent on full-time care as well as the opportunity cost of parents in not being able to choose careers that pay well but are also inflexible with regards to family life with extreme situations. This means if parents cannot afford some expensive new autism drug, then drug companies will not make any money because the parents will not be able to afford it, unless of course they can get the government to pay for it. In the United States, the government only pays for and subsidizes the health care of the elderly. Children with disabilities and their families get paltry financial help with medical expenses compared to what baby boomer retirees receive. On top of that, because of this massive welfare state in the form of Social Security and Medicare, there is a market for investing in research for elderly diseases such as Alzheimer's because baby boomers get the lion share of medical welfare. Not so much for those with autism as very few people with severe autism will ever collect Medicare or Social Security benefits (some may get medicaid benefits, but usually that involves becoming a ward of the state) because they usually do not live that long and cannot hold down a job (necessary for Social Security).

    And as you have pointed out before many times on this blog and including in this post, practicing MD's in the United States at least just get in the way of parents trying to help their children and more often than not just give useless advice such as telling parents to go do ABA for 40 hours a week and magically their child's autism will go away.

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  3. Time to give up on Asd? ..still waiting for subgroups and biomarkers https://onlinelibrary.wiley.com/doi/full/10.1002/aur.1746?fbclid=IwAR1QGi0DlcuIknXNBLoNZwbQghNQhvn_-uWI-vZV2KGhK8P7AmvYff2hDtM
    carla marta

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    1. Carla Marta, I think it is time for self-help and not to expect too much of practical/translational substance from the full time autism researchers.

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  4. Hi Peter, my son is now with an herpes simple flare,I see autism as an immune system dysfunction, as simple as that.
    Valentina

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    1. Valentina, does the herpes make the autism worse? Does treating the herpes affect his autim?

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    2. Peter, his behavior is directly altered, since he has the virus burst shows a completly lack of attention here and at school, uncontrollable laughter, executive functiones at almost 0,worsened gross and fine motor skills, some tics appeared, lack of sleep. I am completly convinced that the nature of his autism is a neuroimmune dysfunction. I don't know for how long is safe giving Valtrex,he is taking 500 mg twice a day.
      Valentina

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  5. Good afternoon Peter and community,

    With the big Society for Neuroscience 2019 conference kicking off soon, we have access to the relevant abstracts at the following link:

    https://www.abstractsonline.com/pp8/#!/7883

    For example, if you type in "Autism" you get 630 abstracts coming up.

    So if you're like me, and spend a lot of time reading through PubMed, these abstracts are going to keep you busy.

    Ling - just when we thought we didn't have enough time to read through all the papers we're interested in on PubMed, we now have access to the 2019 SfN abstracts … ;-)

    AJ

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    1. Ha, I accept the challenge! :-D

      /Ling

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    2. Some of the most interesting/useful papers often do not even have the word autism in them, so you have even more reading ahead of you.

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    3. Hi Peter and Ling,

      Ling - I think the people at SfN are probably already noting how much more traffic they have this year on their abstract site than in previous years … if they only knew it was us reading every paper …. :-)

      Peter - I agree with you 100% and that is actually a great point. Every day when I go to PubMed, I usually search under "Autism", "Neuron", "Synapse", "Dendrite"/"Dendritic spine", "neurotransmitter", etc. so there are in fact many more abstracts than the 630 under "autism" alone.

      There goes the little sleep I was already getting …

      AJ

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    4. Finished those 630 abtracts!
      OK, I cheated, I didn't read all of them but only those that spurred my interest. Here is a selection of abstracts with a short descriptive sentence for anyone coming after me:

      https://www.abstractsonline.com/pp8/#!/7883/presentation/66027
      (Excitatory GABA in acute/chronic mouse epilepsy recorded in vivo)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/73081
      (Novel, selective NKCC1 inhibitor without diuretic effect tested in ASD mice)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/66438
      (Myelin and oligodendrocytes deficits found in Pitt-Hopkins, Pten, Mecp2)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/73111
      (Size of the Corpus callosum is associated with the strength of handedness)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/55186
      (Nighttime light pollution in autism model affects sociability and repetitive behavior)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/56597
      (Prenatal exposure to maternal allergy alters microglia function, particularly in males)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/73512
      (The ketogenic diet rescues Fmr1 KO phenotypes in male mice)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/60363
      (Chronic administrations of CBD in young mice provoke sleep disturbances in adulthood)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/49874
      (Blocking CB1 receptor in monkeys (as opposed to rodents) decreases social behaviors)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/73119
      (Caffeine and A2A antagonist may recover neuronal outgrowth in ADHD)

      https://www.abstractsonline.com/pp8/#!/7883/presentation/54029
      (Purinergic P2X4Rs and D2 receptor interaction modulates PPI and sensorimotor gating - Ivermectin is mentioned)

      /Ling at your service

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    5. Ling, you have so many links Google sent your comment to the spam folder, but here it is.

      Delete
    6. Ah, I see. First time I was caught as a spammer! :-o
      Happy to hear you disagree with Google regarding me. :-)

      /Ling

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  6. Ithink the answer is yes without doubts..https://www.the-scientist.com/news-opinion/can-preventing-seizures-alter-the-course-of-autism--66545 carla marta

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    1. I agree with you Carla Marta, but I go much further than this article. By treating neuronal hyper-excitability in autism you can prevent the onset of the first seizure. In most people seizures make autism worse, but not in all cases. In most cases all you need are cheap safe generic drugs, nothing fancy.

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  7. Does the Feedbucket link work for you? It's been down for 24 hours here.

    /Ling

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    1. Ling, it sometimes stops working, it is an issue with Feedbucket not this blog.

      Delete

  8. Treating “encephalopathy” is not only less controversial, but at least it makes sense. “Autism” defined as a set of behaviors does not make sense if we are discussing medicine. You cannot treat a set of behaviors medically.
    It seems I am not alone in my view on redefining autism, but this is only one of many stumbling blocks.

    What should be the goal of self-help if full recovery is not to be expected? Personalized medicine with the available treatment options is of course urgently needed for many children or adults with autism. It is difficult to obtain, but not impossible, even if a parent is not a doctor as your blog shows best. But I can see another need: a standard of care or guidelines for clinical practice for autism (plus all these comorbidities belonging to the same pathology as Ling said) to be followed by pediatricians. Bumetanide approval may change the paradigm, but I am afraid it will not be enough to get the idea of treating autism (or encephalopathy+) into a wider world. This is a lifelong treatment, who will continue if there is no universal recommendation to treat?

    As the topic is related, I hope it’s not inappropriate to post here the link to the US parental NGO aiming for progress: https://brainfoundation.org. They organize a conference in Nov 2019 for medical professionals with a public day for everyone. This fb group accompanies the event and members can sign up for free streaming of the talks: https://www.facebook.com/groups/378157036156032/

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