Utility of the ketogenic diet to reverse the core deficits of autism spectrum disorder
MetadataShow full item record
Over the last decade, not only has the awareness of ASD increased dramatically, but the rates of children diagnosed with autism has increased as well. The Center of Disease Control (CDC) now estimates that autism affects approximately 1 in every 59 children, a 150% increase since 2000. Autism Spectrum Disorder is characterized by impaired social interactions and the presence of stereotyped behaviors that results in lifelong impaired functioning. There is significant clinical heterogeneity within ASD suggesting a multifactorial pathophysiology with contribution from both genetic and environmental risk factors. Given the complexity of ASD pathophysiology, existing treatments are directed at each clinical manifestation and are not considered curative. Antipsychotic medications have been shown to be efficacious in reducing the repetitive patterns of behavior, while behavioral therapy is effective in alleviating the social impairment associated with ASD. Notably, epilepsy occurs in approximately 33% of individuals with ASD. The high co-occurrence of epilepsy and autism has been hypothesized to be the result of a shared underlying pathophysiology. Current treatment guidelines for epilepsy include anti-epileptic medications with varying mechanisms of action, however approximately one-third of patients with epilepsy fail to achieve adequate seizure control with drug therapy. In these individuals with refractory epilepsy, studies have demonstrated that the ketogenic diet has proven efficacy in achieving seizure control. Numerous mouse models and several human trials have also evaluated the efficacy of the ketogenic diet in reversing the symptoms of autism, though these studies have largely been limited in generalizability, sample size, and randomization. This proposed study will be a randomized controlled study and will assess the utility of the ketogenic diet in reversing the core deficits of autism, social impairment and restricted, repetitive behaviors. Several assessment tools will be utilized to evaluate the behavioral response to the ketogenic diet. The applicability of the ketogenic diet to improve the symptoms of ASD can have a potentially profound impact on the financial burden of the disease. Additionally, it can reduce the need for both anti-epileptic and anti-psychotic medications, thus reducing both the side effects associated with those medications, while concurrently improving the symptoms of ASD.