Abstract
Objective: The hypothesis of this FDA-IRB approved open-label pilot trial was that patients with ASD and mt dysfunction would improve clinically and/or biochemically following treatment with a combination of carnitine, coenzyme Q10 and alpha-lipoic acid (MitoCocktail).
Background: Emerging research has suggested that mitochondrial (mt) dysfunction may play a role in the pathogenesis of some patients with autism spectrum disorder (ASD)
Design/Methods: Participants fulfilled DSM-V diagnostic criteria for ASD and had abnormal buccal swab mt RCC (respiratory chain complexes) I and/or IV activity. They were measured through immunocapture and spectrophotometry normalized to citrate synthase activity. Eleven patients (10M, 1F), aged 5–12 years, received MitoCocktail daily during 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 (subscales/total) from ADOS-2, 3 from ASRS (subscales/total), and 5 (subscales) from ABC were contrasted at T1 vs. T2. Significant changes were compared at T2 vs. T3. RCC I and IV were measured across these time points. For statistical analysis paired t-tests were used.
Results: 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).
Conclusions: This small pilot study supports the hypothesis that in ASD MitoCocktail may have a therapeutic benefit, improving mt function and maladaptive behavior and speech. Larger placebo-controlled trials are needed to demonstrate efficacy.
Study Supported by:
Funded by the Drexel University Clinical and Translational Research Institute (CTRI)
Disclosure: Dr. Legido has nothing to disclose. Dr. Goldenthal has nothing to disclose. Dr. Garvin has nothing to disclose. Dr. Damle has nothing to disclose. Dr. Corrigan has nothing to disclose. Dr. Connell has nothing to disclose. Dr. Thao has nothing to disclose. Dr. Valencia has nothing to disclose. Dr. Melvin has nothing to disclose. Dr. Khurana has nothing to disclose. Dr. Grant has nothing to disclose. Dr. Newschaffer has nothing to disclose.
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