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Testing Sensory and Multisensory Function in Children with Autism Spectrum Disorder
Published on: April 22, 2015
A pilot open-label trial of minocycline in patients with autism and regressive features
Carlos A Pardo1, Ashura Buckley2, Audrey Thurm2
1Department of Neurology, Pathology 627, Johns Hopkins University School of Medicine, 600 North Wolfe Street, Baltimore, MD, 21287, USA.
Background:
Minocycline is a tetracycline derivative that readily crosses the blood brain barrier and appears to have beneficial effects on neuroinflammation, microglial activation and neuroprotection in a variety of neurological disorders. Both microglial activation and neuroinflammation have been reported to be associated with autism. The study was designed to evaluate the effects of minocycline treatment on markers of neuroinflammation and autism symptomatology in children with autism and a history of developmental regression.
Methods:
Eleven children were enrolled in an open-label trial of six months of minocycline (1.4 mg/kg). Ten children completed the trial. Behavioral measures were collected and cerebrospinal fluid (CSF), serum and plasma were obtained before and at the end of minocycline treatment and were analyzed for markers of neuroinflammation.
Results:
Clinical improvements were negligible. The laboratory assays demonstrated significant changes in the expression profile of the truncated form of brain derived neurotrophic factor (BDNF) (P = 0.042) and hepatic growth factor (HGF) (P = 0.028) in CSF. In serum, the ratio of the truncated BDNF form and α-2 macroglobulin (α-2 M), was also significantly lower (P = 0.028) while the mature BDNF/α-2 M ratio revealed no difference following treatment. Only the chemokine CXCL8 (IL-8) was significantly different (P = 0.047) in serum while no significant changes were observed in CSF or serum in chemokines such as CCL2 (MCP-1) or cytokines such as TNF-α, CD40L, IL-6, IFN-γ and IL-1β when pre- and post-treatment levels of these proteins were compared. No significant pre- and post-treatment changes were seen in the profiles of plasma metalloproteinases, putative targets of the effects of minocycline.
Conclusions:
Changes in the pre- and post-treatment profiles of BDNF in CSF and blood, HGF in CSF and CXCL8 (IL-8) in serum, suggest that minocycline may have effects in the CNS by modulating the production of neurotrophic growth factors. However, in this small group of children, no clinical improvements were observed during or after the six months of minocycline administration.
Trial Registration:
NCT00409747.
Insights
Minocycline treatment showed no clinical improvements in children with autism. However, it altered neurotrophic factors like BDNF and HGF in the central nervous system and CXCL8 in serum.
Area of Science:
- Neuroscience
- Pediatrics
- Pharmacology
Background:
- Autism is associated with neuroinflammation and microglial activation.
- Minocycline, a tetracycline derivative, crosses the blood-brain barrier and has shown neuroprotective effects.
- This study investigated minocycline's impact on neuroinflammation markers and autism symptoms in children with developmental regression.
Purpose of the Study:
- To evaluate the effects of minocycline on neuroinflammation markers in children with autism.
- To assess changes in autism symptomatology following minocycline treatment.
- To explore minocycline's potential to modulate neurotrophic factors and inflammatory markers.
Main Methods:
- An open-label trial involving eleven children with autism and developmental regression.
- Six months of minocycline administration (1.4 mg/kg).
- Analysis of behavioral measures, cerebrospinal fluid (CSF), serum, and plasma for neuroinflammation markers before and after treatment.
Main Results:
- No significant clinical improvements were observed.
- Significant changes in truncated brain-derived neurotrophic factor (BDNF) and hepatic growth factor (HGF) in CSF.
- Alterations in serum CXCL8 (IL-8) levels and the ratio of truncated BDNF to α-2 macroglobulin (α-2 M).
Conclusions:
- Minocycline may influence the central nervous system by modulating neurotrophic factors (BDNF, HGF) and CXCL8.
- Despite observed biomarker changes, minocycline did not yield clinical benefits in this cohort.
- Further research is needed to understand minocycline's therapeutic potential in autism.
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