August 9th, 2017


Following last Friday’s PediaBlog post, “Autism: Eyes Point Internally,” a reader asks:

I have an autistic eleven-year-old son. I recently read an online article written by Emily Deans, M.D., Evolutionary Psychiatry, about various versions of the ketogenic diet having a positive effect on SOME autistic children. Does anyone have an opinion or knowledge of the efficacy of this diet upon the autistic child? (The terms mitochondrial and microbiome used by Dr. Petrosky lead me to ask this question.)


There has been some research investigating the potential benefits of using a ketogenic diet in autistic children after a small study from Greece in 2003 suggested some improvement of some autistic behaviors in some autistic children. As Dr. Deans explained in Psychology Today in 2011, the special diet was not well-tolerated by more than a third of the study’s 30 participants, but some of the kids who did tolerate it improved:

23 kids tolerated the diet beyond the initial 4 weeks, and of those, 5 more discontinued the diet due to lack of improvement during the first few cycles. Of the remaining 18 kids, two boys improved enough in symptoms to be taken out of the special school and placed in mainstream education. Overall the 18 ketogenic kids “presented with improvements in their social behavior and interactions, speech, cooperation, stereotypy, and… hyperactivity, which contributed significantly to their improvement in learning.”

The kids who did not stay on the diet were the most severely affected by autism, and the ones who had the best response were ones most mildly affected. Another interesting fact from the study is that the kids maintained their improvements through the two week washout periods and in the 6 months after the study was over. None of the kids had any complications (such as poor weight gain or selenium deficiency) seen in other trials of ketogenic diets in kids with epilepsy.

Overall (using the original sample size of 30), 26.66% of the kids benefited significantly from the diet.


Research has continued. A 2013 study, published in the journal PLOS ONE, found that a ketogenic diet improved core symptoms of autism… in mice. A 2014 review study, appearing in the open access (non-peer reviewed) journal Frontiers in Pediatrics, cites one case report and one study showing a decrease in seizures and improvement in behaviors from a ketogenic diet. (Ketogenic diets have been used for decades to treat poorly-controlled and complicated seizure disorders.) A more thorough review, published in 2015 in the open access journal Research in Autism Spectrum Disorders, concluded this about the effect of the ketogenic diet (KD) on symptoms of children with ASD:

The limited number of reports of improvements after treatment with the KD is insufficient to attest to the practicability of the KD as a treatment for ASD, but it is still a good indicator that this diet is a promising therapeutic option for this disorder.


So more research on ketogenic diets and autism is needed. (Here is one study that is currently recruiting participants.) It’s too early to say whether a ketogenic diet has merit as a credible and safe treatment option for children with autism, or whether it is, like so many sham therapies that have come before, a potentially dangerous waste of time, money, and health. Earlier this year, the U.S. Food and Drug Administration warned against bogus autism cures. Robert Preidt says the FDA cited “a long history of failed autism treatments and fads” in their warning:

Don’t fall for products claiming to cure autism, the U.S. Food and Drug Administration warns.

There’s no cure for the neurodevelopmental disorder, the agency said. Yet bogus “cures” and therapies abound — from toxin removal to raw camel milk.

Some of these fraudulent treatments could be harmful, and should be avoided, the agency said Wednesday.

Among them: chelation therapies, hyperbaric oxygen therapy and detoxifying clay baths.


The National Center for Complementary and Integrative Health (part of the National Institutes of Health) last year published evidence-based Clinical Guidelines, Scientific Literature, Info for Patients: Autism Spectrum Disorder and Complementary Health Approaches in order to evaluate the evidence, efficacy, and safety behind the use of some alternative therapies. Natural products and biologics (melatonin, omega-3 fatty acid supplementation, probiotics, secretin, vitamin B6 and magnesium, and chelation), special diets (gluten-free, casein-free, and ketogenic diets), mind and body practices (acupuncture and music therapy), and other approaches (hyperbaric oxygen therapy) were all evaluated as objectively as possible.  According to the report, support for the use of ketogenic diets in children with autism is “limited” based on the current scientific evidence. Parents who are interested in pursuing treatments with a ketogenic diet should be aware of safety concerns:

The mechanism of action of the ketogenic diet is not fully understood, and caution should be taken to avoid deleterious adverse effects or refractory outcomes. Ketogenic diets should be supervised by a nutritionist to ensure that children get the appropriate nutritional requirements for growth.  Reported adverse effects include short-term gastrointestinal-related disturbances, to longer-term cardiovascular complications.


The most effective complementary approach in treating autistic children?

A 2014 Cochrane review of 10 studies involving a total of 165 children with ASD found that music therapy was superior to “placebo” therapy or standard care for social interaction, non-verbal and verbal communication skills, initiating behavior, and social-emotional reciprocity. The review concluded that music therapy may help children with ASD to improve their skills in areas such as social interaction and communication, and may also contribute to increasing social adaptation skills in children with ASD and to promoting the quality of parent-child relationships.


An international, multi-center study published just yesterday in JAMA, however, has some disappointing results regarding improvisational music therapy in children with autism. Read more about the study and the methodological problems the researchers experienced with it here.

Readers wishing to learn more about autism spectrum disorder are invited to attend a workshop presented by Pediatric Alliance entitled “It Takes A Village: Autism/Learning Disabilities” this Saturday, August 12, at The Woodlands Foundation (134 Shenot Road, Wexford, PA). Here is the lineup of speakers for the event:

A Significant Difficulty – Autism Spectrum Disorder: A discussion with a focus on early diagnosis, new research, socialization, and other therapies — presented by Michael Petrosky, MD, FAAP, Pediatric Alliance — Wexford.

Executive Functioning: This encompasses cognitive, behavioral, and emotional regulation — presented by Dr. Erika Buchanan, Licensed Psychologist and Certified School Psychologist from the Center for Pediatric Neuropsychology.

Learning Disabilities and Navigating School Based Services — presented by Damian Ternullo, MD, FAAP, Pediatric Alliance — St. Clair.

Guest speaker: Kristin C. Weidus, Esq., Ruder Law – Advocates for the rights of children in public schools.

The Woodlands Foundation: Enrichment for people of all ages living with disabilities and chronic illness, while providing respite for families — presented by Jesse Solomon, Director of Programs.

Local resources will be available with materials and additional information following Q&A.

Learn more about “It Takes A Village: Autism/Learning Disabilities” and register for the class here.




(Google Images)



4 Responses to Ask Us Anything

  1. I think the statement quoted above by the FDA (“There’s no cure for the neurodevelopmental disorder”) reflects a defeatist attitude and basically a denigration of the human spirit — a rejection of “that hope that springs eternal within the human breast” (to borrow a line from the poem “Casey at the Bat.”)

    It is generally accepted that Autism is the prototype of a “multifactorial disorder” with a common endpoint”: genetically-induced auto-immune encephalitis. This process does not evolve overnight. It is accepted that “No two children with Autism are alike” and that in the area of therapy, “One size fits none.”

    In essence, I think every young child with the consideration of a diagnosis of Autism (however tenuous it may be) requires a comprehensive work-up focusing on the biomedical factors that may be at play in that particular individual; this is where microbiome, mitochondria, allergy, and the like come in. Only by this painstaking analysis (coordinated by a specialist with time, energy, and vision) can we accumulate the data needed to correct or manipulate the crucial EPIGENETIC factors that fashion the disorder in any particular patient.

    Treatment has to be greatly individualized, must supplement the conventional therapies (ABA, speech therapy, etc), and the prescriber and family must be tolerant of trial-and-error. I think Autism is such a diverse condition that it does not lend itself to the formal controlled studies that are the bedrock of scientific investigation; Autism research must focus on a “trial of one” — the unique patient.

    I truly believe that if we can intercept the evolution of Autism early-on in its formative stages, modify the epigenetic variables, and simply “never give up” we can effect a clinical amelioration tantamount to cure.

    I can state one thing with certainty: I have never seen a family of a child with Autism which fails to be a fierce advocate for their own child and for others affected. These families refuse to embrace the notion that we should throw in the towel because the condition is outright incurable. Defeat is preferable to not striving valiantly! (borrowed from Teddy Roosevelt)

  2. Right on, Dr. Kovach! When caring for the autistic child, it is important for all doctors to keep that fire in their bellies, and strive and work and strive some more, and never give up. From the outside, an autistic child showing any improvement may seem bleak. When you work intensely with them, improvement IS possible. Autism is very much a two steps forward and one step back disorder, and sometimes that improvement is fleeting, but the improvement is lasting and will surface from time to time. I prefer to fight autism while standing and fully engaged, I’ll never stop. Thank you for such a great article.

  3. Dear Pamela—thank you for your kind comments! You obviously understand well the excitement that ensues when an Autistic child makes even a meager gain, let alone a motion toward cure.
    We expect antibiotics and vaccinations to have a high (90 to 100%) efficacy and that is how they are evaluated in scientific studies. But I think a one in four benefit (as with the ketogenic diet) for any intervention in an Autistic child is more than just significant—it may be life-altering!
    It is fighters like yourself and the editor that keep the rest of us from falling into the “swamp” of indifference and cynicism.


    Ned Ketyer, M.D.

    Ned Ketyer, M.D.

    Dr. Ketyer has special interests in developmental pediatrics and preventative medicine, specifically how nutrition and the environment affect health. He earned his bachelor’s degree from the University of Vermont and his medical degree from Northwestern University Medical School. He completed his residency at Children’s Hospital of Pittsburgh.

    As one of the founding physicians of Pediatric Alliance, PC, Dr. Ketyer served as its president from 1997-2004. He has been practicing general pediatrics at Pediatric Alliance since 1990. Dr. Ketyer and his wife have three boys and live in Pittsburgh's South Hills. 

  • Note: The information included in these posts is for educational purposes only. It is not intended nor implied to be a substitute for professional medical advice.

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