When Will There Ever Be a Cure for Autism?

One of the problems with Hollywood, aside from the fact that it’s full of people who make a living pretending to be someone they’re not, is that it shapes the way that people see the world. When a movie comes out that features a topic that most people don’t know much about, suddenly that’s their sole source of information about the topic.

Take the 1988 movie Rain Man, for example, a classic that’s now more than 30 years old. Tell someone that your son is autistic and they’ll probably ask you if he has any special “toothpick counting powers,” or if you’ve ever taken him to clean up on some Blackjack at the nearest Indian reservation.  The truth is, there are “fewer than 100 known prodigious savants living worldwide at the present time who would meet that very high threshold of savant ability.” That’s according to an excellent academic paper on the topic titled “The savant syndrome: an extraordinary condition. A synopsis: past, present, future” which goes on to say that only 50% of people with savant capabilities are autistic, while the rest have some other condition.

When it comes to bog standard autism, a whole lot more people suffer from this condition that oftentimes prevents them from living a normal life. About 1 in 59 children are diagnosed with autism spectrum disorder with boys being four times more likely to be affected. Prevalence seems to be increasing over time, that’s according to the below table by the CDC:

The growing prevalence of autism
The growing prevalence of autism – Source: CDC

Psychology Today published an article trying to make sense of the alarming increase in prevalence, and the first bit of bad news appears. Similar to other incurable conditions we’ve talked about before like multiple sclerosis, scientists don’t know what causes autism. A huge barrier to treating any problem is not knowing the root cause. We’re treating the symptoms instead, which means that everyone will probably react differently to various treatments. To make matters worse, it’s a “spectrum disorder” which, as the name implies, means that everyone has varying degrees of severity. Like most Americans, the first question we have is, what pills can we start popping to address the problem?

Drugs for Autism

More bad news on the drug front, because according to Stanford University Professor Antonio Hardan, “companies are not interested in developing a drug for a disorder that does not have a lot of market share.” We took that statement from an article by Spectrum published way back in 2010, and the same publication published some more optimistic news this summer talking about a grant called the “Autism Innovative Medicine Studies-2-Trials” which includes 48 partners across 14 nations who are working to develop precision medicine that can target each individual differently, similar to how we will eventually develop a “cure for cancer.” It is said to be the biggest project to date in autism research. In the meantime, there are some drugs available to treat autism and some under development. You can take a look at the 2015 paper titled Pharmacological Therapies for Autism Spectrum Disorder: A Review which discusses therapies being developed at great length, or you can just read on and learn about some of the more relevant ones.

FDA-approved Drugs for Autism

Up until now, pharmaceutical treatments have largely involved the use of antipsychotics which are primarily used for schizophrenia and bipolar disorder. Two drugs – Risperidone and Aripiprazole – have been approved by the FDA to treat irritability related to autism, but there are no FDA-approved drugs for core ASD symptoms such as social interaction difficulties. Let’s take a look at some drugs in the development pipeline.

Balovaptan by Roche

Click for company websiteA drug being developed by pharmaceutical giant Roche called Balovaptan has the potential to be the first drug therapy to help improve core social interaction and communication in people with autism spectrum disorder. The FDA granted “breakthrough therapy designation” for the drug which is now in Phase II clinical trials. (If a drug is designated as breakthrough therapy, FDA will expedite the development and review of such drug.) If you’re interested in taking part in the study, there’s a website called The aV1ation Study which tells you how.

The aV1ation Study
The aV1ation Study – Source: Roche

Please note that the study is open to U.S. residents only.

Curemark and CM-AT

Founded in 2004, New Yawk startup Curemark has taken in just over $61 million in funding to develop novel therapies for the treatment of neurological disorders. Their pipeline includes lead drug candidate CM-AT which has received “Fast Track” designation from the U.S. Food and Drug Administration (FDA). The drug is in a Phase III clinical trial for children aged 3 – 8 years old which has completed enrollment and is referred to as the Blüm Study.

The Blum Study
The Blum Study – Source: Curemark

Many children with autism exhibit impaired protein digestion, which may or may not manifest in self-restricted diets. Here’s how the drug addresses that according to another informative article by Spectrum:

According to the company’s scientific officers, the idea underlying the drug is this: Amino acids serve as building blocks for chemical messengers in the brain that some children with autism may lack. By releasing essential amino acids, CM-AT restores the necessary levels of these chemicals in the children’s brains.

Autism Therapeutics and AT001

Founded in 2010, London England startup Autism Therapeutics has taken in an undisclosed amount of funding to develop ATOO1, a drug that is in the final stages of development to treat the core symptoms of autism. The only problem is, they haven’t really provided any updates on what’s happening since 2012. It’s only worth mentioning because maybe then they’ll update the horrendous train wreck that they call a website. If you ever wanted to show someone how not to build a user interface, this would be one of the best examples you will ever find.

Yamo Pharmaceuticals and L1-79

Not a whole lot is known about Yamo Pharmaceuticals, a company that doesn’t even have a profile in Crunchbase. They’re a small clinical-stage pharmaceutical company which has developed L1-79, an oral treatment that appears to impact the core symptoms of autism, including socialization and communication. In May of last year, they also received “Fast Track” designation from the FDA and are currently preparing for a 250-patient Phase 2b study under FDA oversight that is planned to start treating patients in early 2019. The technology “integrates extensive pre-existing medical research into a more unified theory of autism than has been previously possible.”

Seaside Therapeutics and Fragile X

Founded in 2005, Massachusetts startup Seaside Therapeutics has taken in $30 million in funding to develop drug treatments to correct or improve the course of autism and Fragile X syndrome. (Fragile X syndrome is a genetic condition that causes a range of developmental problems including learning disabilities and cognitive impairment.) Here’s a look at their drug pipeline:

The Seaside Therapeutics Pipeline
The Seaside Therapeutics Pipeline – Source: Seaside Therapeutics

While most of society patiently waits for drugs like these to get approved, many parents out there don’t want to wait and turn to the Internet for help.

Autism and Doctor Google

It’s hard to fathom how difficult it is for parents who learn that their child has been diagnosed with a condition that has no cure. It’s inevitable that parents will immediately begin doing their own research to figure out what can be done to help their children, and there is an entire community of people online that have formed around this topic. Every type of home remedy you can think of will be discussed, and while some people have claimed to “cure autism” in one instance, what works for some doesn’t always work for others. There’s no harm in asking Dr. Google if there’s a cure for autism, just be realistic about what progress you’ll actually be able to make. What the community needs to focus on is looking towards finding the cause of autism, perhaps by taking an approach that involves genetics and machine learning algorithms.

Other Autism Startups

While we wanted to keep this article focused on drug treatments, we did come across some other autism-related startups worth noting. A company out of Utah called Lineagen offers a genetic testing service for children with autism spectrum disorder. We also came across a startup called Cognoa that uses artificial intelligence to diagnose autism. Lastly, a company we wrote about yesterday, Aperiomics, thinks that perhaps the answer may lie in identifying pathogens that cause autism, and they’re working towards exploring that.

We found all kinds of startups doing work around helping to treat autism using apps and such, so please don’t email us saying we “missed” your startup that is working on something related to autism. For this article, we’re mainly interested in any new autism-specific drugs being developed that have begun the FDA approval process, or any startups looking at the genetic roots of autism or how we might treat patients using personalized medicine. If you’re doing work in that area, drop us a note in the comments section or send us an email.


Whenever we do these “when will there be a cure” articles, inevitably a bunch of knuckle-draggers start posting comments about how Dr. Ifa sorted them out with some herbal cure and here’s his contact info which is in fact a direct line to the Sakawa boys who will then exhaust every option they can think of to get their hands on some of your hard-earned cash. The reason these scams work at all is because people who have a condition that cannot be cured will do just about anything to try and fix the problem, especially if it involves their child. Don’t fall for any of that garbage. While there may be home remedies out there that people swear by, we’re only interested in hearing about progress being made either by legitimate corporations – like Aperiomics for example – or progress that’s been published in an academic paper that’s been reviewed by other academics.


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  1. Let’s consider for a second the prospect that autism isn’t a condition to be cured but a normal human variation that has always been with us. When I do, this article comes across as extremely bigoted and mean spirited.

    1. Sure, that is a prospect. But the more is researched about autism, the more it looks as if there is a deficit in the connectivity of neurons in autistic brains or imbalances that prevent them from communicating properly. Sure, this can be variation that is occurring between individuals. But the fact that it is normal variation does not mean that this variation leads to desirable conditions. Or it does not mean these conditions could not be treated, as normal variation between humans also causes diabetes for example. Medicine should be used to improve the quality of life. If there is a treatment that can help an individual with autism to take care of himself and even not hurt himself, I do not see any mean spirit from attempting that result.

      1. That IS what you find. . . if you do the research backwards. It’s the same if you study any part of science backwards. Take, for example, classical physics. Nowadays we have the three laws of thermodynamics (two of which we’ve decided are actually tendencies), simple machines, and newton’s laws of motion. Go back a few hundred years, and all you have are newton’s laws and a few simple machines. That’s what you’re doing with autism research. You start in the modern day, when we know nothing is wrong with autistics, then you go back to the very first few years of the 21st century, when everyone, even those who seem so nice, like Jenny McCarthy, are nothing more than bigots seeking to confirm their beliefs, at the expense of innocent kids.

        1. The caregivers of those with severe autism may disagree with your calling research around this space bigotry.

  2. I have a 9-yr-old recently ‘officially’ diagnosed with autism, or, to use the official phrase ‘Autistic spectrum disorder.’ Since he was five, I felt that he had differences, yet a psychologist at the time said ‘No.’ My wife says that she feels that I have some of the behaviours that our 9-yr-old has, and that I may be autistic. Given I am 69 and have had a good life, including health, work, and financial success, I am unconcerned whether I may or may not be. Yes, I think that I am ‘different,’ and am very happy so to be, given how others appear to live and how they behave; but then, aren’t we all ‘different’ in our own ways?

    My autistic son has ways that make difficulties in doing things, for himself and as a family; and his verbal communication can be very challenging, especially as he has little empathy, and tends to make comments that suggest others are not right nor capable in some way or another. He is very intelligent, and some may say he has Aspergers (where that term is still recognised) and he is most definitely high functioning, with an IQ greater than 100, in my humble opinion. His special school are soon to start giving him lessons for children a year ahead, as he has done everything for his age group, and current lessons ‘bore’ him, consequently.

    Maybe autism has always been around, just not seen as such. Remembering my childhood, I was considered ‘gifted’ and offered a place at a boarding school that dealt with so-called gifted children (and which place I declined). I had my difficulties with other people – and have over the years learnt how to minimise and perhaps even remove such – and now am comfortable in my own skin. So, whether I have autistic traits or not, I understand and feel for those who do not fit into society so easily and well. Is that a problem? For me, it’s how the individual looks on it, as long as they don’t disrupt things around them too much.

    So … does the label ‘autistic’ matter that much? In my view, only when the individual cannot manage reasonably on their own; then they need help. If they can, why bother labelling: let them get on with their life in the way they like, as long as others aren’t unreasonably put about.

    1. Thank you for sharing Andrew. That make a whole lot of sense. Clearly the “cure” is for people that are looking for one. Else as you said, get on with your life! We all have problems, anxieties, difficulties in social situations, relationship problems, and the like. They don’t need a label or a pill to pop. We’re all different in our own ways as you said. We’re all unique, just like everyone else. It’s the people with autism who cannot function without a caregiver that would be grateful to live a normal life – as would the caregivers for that matter.

      1. Yes: horses for courses, and each to his own. Both old expressions with a lot of sense behind them. As long as we each do as much as we can or want to, without upsetting others; then we may just get on with living in a manner that gives us as much happiness and lack of suffering as possible. IMHO.

    2. I’m in a similar situation as you Andrew. However, each case is different, hence the term “spectrum” used to describe the condition. The frequency in which it is increasing is alarming, but our situations give us a hint as to why it is increasing. I believe it is hereditary. After my son was born, and started developing, I saw some of the traits in him that I have, more common traits in individuals with autism, and I started to wonder if I have it too. A good comparison is treacher-collins syndrome. When people are born with a very noticeable case of it, just looking at their parents may quickly give an indication where it came from. It’s something that is obvious in retrospect. What I am getting at is this is a dangerous situation, and we need to be able to test for it in order to give people this information before they decided to have children. It’s a devastating diagnosis for everyone involved. I hope for a cure, and it’s quite possible with advances in AI, that one will be developed in my sons lifetime, but the likelihood I will see one in mine is unlikely.

  3. Autism is a condition where brain function is interfered with by external radio waves or electromagnetic waves. When the brain is irradiated with such an electromagnetic wave or radio waves for a long time, the charge of the brain is changed as if it is a temporary magnet. Treatment is possible by blocking external radio waves or electromagnetic waves and rebooting (tuning) the brain function. Other psychosis also differs in brain parts and causes and treatments are similar.

    1. Thank you for commenting. Hadn’t heard this before. Is there a study you can reference that lets people learn about this aside from just a single comment in this forum?