ICNC Abstracts, ICNC 2018

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Effect of a Combination of Carnitine, Coenzyme Q10 and Alpha-Lipoic Acid (MitoCocktail) on Mitochondrial Function and Neurobehavioral Performance in Children with Autism Spectrum Disorder
Agustin Legido, Michael Goldenthal, Brigid Garvin, Shirish Damle, Kelleen Corrigan, James Connell, Diana Thao, Ignacio Valencia, Joseph Melvin, Divya Subramanian Khurana, Mitzie Grant, Craig Newschaffer

Last modified: 2018-09-09


Emerging research suggests that mitochondrial (mt) dysfunction is important in the pathogenesis of autism spectrum disorder (ASD). The hypothesis of this open-label pilot trial was that patients with ASD and mt dysfunction would improve clinically and biochemically following treatment with combination of carnitine, coenzyme Q10 and alpha-lipoic acid (MitoCocktail).

Participants fulfilled diagnostic criteria for ASD and had abnormal buccal swab mt RCC (respiratory chain complexes) I and/or IV activity. Eleven patients aged 5-12 years, received MitoCocktail daily for 3 months. Behavioral outcome was evaluated with Autism Diagnostic Observation Schedule (ADOS-2), Autism Spectrum Rating Scale (ASRS) and Aberrant Behavior Checklist (ABC) at baseline (T1), 3 months into treatment (T2), and 3 months post-treatment (T3). Three scores from ADOS-2, 3 from ASRS, and 5 from ABC were contrasted at T1, T2 and T3. RCC I and IV were measured across these time points. For statistical analysis paired t-tests were used.

RCC I/IV ratio was significantly (p<0.02) reduced during MitoCocktail treatment. All subjects showed at least one sign of metabolic improvement, which waned 3 months post-treatment in 7/11 participants. All of the 11 total or subscale mean scores improved from T1 to T2. Significant changes were observed for Unusual Behavior subscale from ASRS (p<0.006), Lethargy subscale from ABC (p <0.01), and Inappropriate Speech subscale from ABC (p<0.02). From T2 to T3, scores worsened on these three subscales being significant for Lethargy (p<0.01) and Inappropriate Speech (p<0.007).

This small pilot study supports the hypothesis that MitoCocktail may have a therapeutic benefit in ASD.

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