Autism spectrum disorder (ASD) is associated with mitochondrial dysfunction but studies demonstrating the efficacy of treatments are scarce. We sought to determine whether a mitochondrial-targeted dietary supplement designed for children with ASD improved mitochondrial function and ASD symptomatology using a double-blind placebo-controlled cross-over design. Sixteen children [Mean Age 9y 4m; 88% male] with non-syndromic ASD and mitochondrial enzyme abnormalities, as measured by MitoSwab (Religen, Plymouth Meeting, PA), received weight-adjusted SpectrumNeeds (NeuroNeeds, Old Lyme, CT) and QNeeds (NeuroNeeds) and placebos matched on taste, texture and appearance during two separate 12-week blocks. Which product received first was randomized. The treatment significantly normalized citrate synthase and complex IV activity as measured by the MitoSwab. Mitochondrial respiration of peripheral blood mononuclear cell respiration, as measured by the Seahorse XFe96 (Agilent, Santa Clara, CA) with the mitochondrial oxidative stress test, became more resilient to oxidative stress after the treatment, particularly in children with poor neurodevelopment. The mitochondrial supplement demonstrated significant improvement in standardized parent-rated scales in neurodevelopment, social withdrawal, hyperactivity and caregiver strain with large effect sizes (Cohens d = 0.77-1.25), while changes measured by the clinical and psychometric instruments were not significantly different. Adverse effects were minimal. This small study on children with ASD and mitochondrial abnormalities demonstrates that a simple, well-tolerated mitochondrial-targeted dietary supplement can improve mitochondrial physiology, ASD symptoms and caregiver wellbeing. Further larger controlled studies need to verify and extend these findings. These findings are significant as children with ASD have few other effective treatments.
Competing Interest StatementThis research was supported by The Brain Foundation (Pleasanton, CA) and the EXL Foundation (Phoenix AZ). NeuroNeeds provided both active supplements and placebos only. None of the sponsors were involved with the design or conduct of the study, collection, management, analysis or interpretation of the data; or preparation, approval of the manuscript or decision to submit the manuscript for publication. Dr. Richard Boles is an owner and officer of NeuroNeeds, as well as a clinician in private practice. He served as an advisor during study design, interpretation and publication, but had no role in human subjects or study procedures. All final decisions were made solely by Dr. Richard Frye from conception to submission. Dr. Frye is a member of the Scientific Advisory Board to The Brain Foundation. All other authors have no conflicts of interest to declare.
Clinical TrialNCT03835117; IND142751
Funding StatementThis clinical trial was funded by The Brain Foundation (Pleasington, CA) and partially by the XEL Foundation (Pittsburgh, PA), both to Dr. Frye. NeuroNeeds supplied the product for testing.
Author DeclarationsI confirm all relevant ethical guidelines have been followed, and any necessary IRB and/or ethics committee approvals have been obtained.
Yes
The details of the IRB/oversight body that provided approval or exemption for the research described are given below:
IRB of Phoenix Children's Hospital gave ethical approval for this work. IRB of WCG IRB Review gave ethical approval for this work.
I confirm that all necessary patient/participant consent has been obtained and the appropriate institutional forms have been archived, and that any patient/participant/sample identifiers included were not known to anyone (e.g., hospital staff, patients or participants themselves) outside the research group so cannot be used to identify individuals.
Yes
I understand that all clinical trials and any other prospective interventional studies must be registered with an ICMJE-approved registry, such as ClinicalTrials.gov. I confirm that any such study reported in the manuscript has been registered and the trial registration ID is provided (note: if posting a prospective study registered retrospectively, please provide a statement in the trial ID field explaining why the study was not registered in advance).
Yes
I have followed all appropriate research reporting guidelines, such as any relevant EQUATOR Network research reporting checklist(s) and other pertinent material, if applicable.
Yes
Data AvailabilityAll data produced in the present study are available upon reasonable request to the authors.
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