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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