Summary: While there are no specific medications for autism in the traditional sense, there are medications for autism in common use that address specific features of the disorder.
Key Points:
- People with autism spectrum disorder (ASD) show a variety of symptoms in unique combinations, with no single expression of symptoms defining the disorder.
- Providers may prescribe antipsychotic medications to help reduce features like irritability, aggression, and emotion regulation.
- Providers may prescribe antidepressants to address anxiety, anxiolytics to prevent /manage panic attacks, and stimulant or nonstimulant medication to help manage hyperactivity.
- New research shows a specific type of nutritional supplement may help reduce several types of disruptive symptoms associated with ASD.
ASD: The Search for Targets for Medication
For a basic understanding of the topic – medications for autism – we’ll start with this excellent basic definition of autism/autism spectrum disorders:
“Autism spectrum disorder (ASD) is a group of neurodevelopmental disorders characterized by impairment in social interaction, deficits in verbal and non-verbal communication and repetitive and restricted patterns of behavior and interests.”
The range of impairment in ASD can range from mild to moderate to severe, with each instance of ASD existing on a continuum of symptom severity. People with ASD may experience minimal disruption to daily life and function, or they may experience significant impairment that prevents them from participation in many of the functional necessities of daily life, from communication to cognition to basic self-care.
There is currently no known cause of ASD, but rather a host of genetic, medical environmental factors implicated in the development of ASD. In recent years, however, research shows that deficiencies in a specific type of amino acid, called branched-chain amino acids BCAAs, are associated with the presence of ASD.
These various amino acids are now the subject of studies on potential medications or autism and autism spectrum disorder (ASD).
Exploring Medications for Autism Spectrum Disorder: First Steps
Results from research on how that knowledge may help people with ASD have begun to appear. For instance, in the study “Association and Predictive Value of Blood Branched-Chain and Excitatory Amino Acids With Autism Spectrum Disorder Severity,” researchers explain the evidentiary basis and primary goal of the study:
“Abnormal amino acid metabolism may contribute to ASD pathogenesis, but the relationship between amino acid levels and severity remains unclear. This study investigated the association and predictive value of blood amino acid profiles for ASD severity.”
To be clear, this is not a study on therapeutic intervention but rather a study to confirm the correlation between the expression of specific BCAAs and autism presence and severity. To that end, the research team recruited 474 children with ASD and divided them into two groups:
- Mild-to-moderate
- Severe
Next, they examined the blood profiles of each patient in order to detect the target the BCAAs leucine and isoleucine, as well as an excitatory amino acid (EAA), glutamate.
Here’s what they found. Primary/preliminary results indicated that:
- Compared to the mild/moderate group, the severe group showed:
- Higher levels of BCAAs
- Higher levels of EAAs
After a thorough statistical analysis of the primary data, results indicated:
- Strongest association with severe ASD:
- Leucine and isoleucine
- Next strongest association with severe ASD:
- Glutamate
- Presence of BCAAs and EAAs correlated positively with increased risk of severe ASD.
- Most powerful associations appeared in children under age 3
The study authors summarize their findings as follows:
“Blood BCAAs and EAAs were closely associated with ASD severity, with leucine/isoleucine as the strongest predictor. Amino acid profile-based models provide biomarker evidence for identifying severe ASD and precision intervention.”
The logical follow up to that finding is to learn whether applying new knowledge about BCAAs/EAAs to treatment helps people with ASD manage or reduce disruptive symptoms. Fortunately, a group of researchers did exactly that.
Exploring Medications for Autism Spectrum Disorder: Targeted Treatment for ASD With Branched-Chain Amino Acids (BCAAs)
The authors of the study “Branched-Chain Amino Acids as Adjunctive-Alternative Treatment in Patients With Autism: A Pilot Study” took results from previous research – like the study we cite above – and defined the situation:
“Disruption of these systems [i.e. BCAAs] has been associated with the pathophysiology of autism while low levels of these amino acids have been discovered in patients with autism.”
And designed a study to assess the following:
“BCAA supplementation in children with autistic behavior constitutes an attempt to find an additional or alternative treatment for children who exhibit minimal to no improvement with conventional methods of treatment.”
They recruited fifty-five children with ASD, between ages 6 and 18 years of age, to examine whether treatment with BCAAs, in the manner of nutritional supplements, can help manage or reduce symptoms of ASD.
To divide the group by levels of symptom severity, all study participants completed the Childhood Autism Rating Scale (CARS) as adapted below. For each item, clinicians provide one of four values to rank symptoms:
- Typical for age: 1
- Mild disorder/disruption: 2
- Moderate disorder/disruption: 3
- Serious disorder/disruption: 4
Clinicians tally the values and assign a level of ASD severity as follows:
- Mild to moderate ASD: Score of 30-36
- Severe: Score of 37 and above
Here are the symptoms categories clinicians assess on the CARS:
- Disruptions to relationships: home, school, social
- Mimicry: amount, frequency, context
- Emotional response differences, i.e. laughing when atypical, crying when atypical
- Atypical physical movements/repetitive movements, called stereotypy, similar to perseveration
- Atypical relationships with/use of inanimate objects
- Avoidance of eye contact: amount, frequency, context
- Idiosyncratic response to sound/auditory input
- Putting objects in mouth: amount, frequency, context
- Stress reactivity: ability or lack to tolerate stress/distress
- Verbal communication differences: absence of speech, echolalia, others
- Non-verbal communication differences: use of gesture/response to gestures
- Activity levels: very high or very low
- Cognition: intellectual capacity, problems solving
- Overall impression
After administering the assessment, researchers distributed participants by CARS outcome as follows:
- Mild/moderate: 27%
- Severe: 73%
Participants who completed the study received amino acid treatment for a minimum of one month and a maximum of six (6) months.
Let’s take a look at what they found.
Can BCAAs Help People With ASD?
The short answer, for the participants in this study:
Yes.
After the treatment period, the researchers observed improvements in:
- Social behavior/interaction
- Speech
- Cooperation
- Stereotypy/perseveration
- Hyperactivity
- Overall learning capacity
Further, the researchers noted:
- One (1) participant showed a 12-unit improvement on the CARS assessment
- Seven (7) showed improvements between 8 and 12 units on the CARS assessment
- Eighteen (18) showed minor improvements of between 2 and 8 units on the CARS assessment
These outcomes are encouraging. The study authors found the improvements notable:
“Eight patients in this study had great success with BCAA supplementation; one had a considerable improvement, seven had a moderate improvement and eighteen patients had a little improvement. This is significant because the patients in question had not improved for a while.”
This is important because it can give people with ASD and their families hope that while previous treatments may not have worked, treatment with BCAAs may be effective. The study authors indicate that the earlier the treatment starts, the more favorable the outcome. In addition, they observe:
“The different degrees of improvement in the patients can be explained by the different metabolic biochemical profiles of each patient.”
This observation leads them to the following conclusions:
- Providers should consider examining the metabolic profile of all children diagnosed with ASD.
- Accurately defining a metabolic profile may help providers improve existing treatments and enable more targeted, individualized treatment for people with ASD.
There’s one contraindication for BCAA therapy for people with ASD: people with maple syrup urine disease should not engage in this form of BCAA therapy. There’s also a relevant limiting factor: the cost of BCAA therapy is high, which may eliminate it as an option for many families. However, the overall takeaway here is that while there’s not yet a medication for autism, this research shows that nutritional supplementation with BCAAs may help people with autism manage and/or reduce symptoms and minimize overall functional impairment.
That’s very good news. When we learn more, we’ll report it here first.
Gianna Melendez
Jodie Dahl, CpHT