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The Soluble Folate Receptor in Autism Spectrum Disorder: Relation to Autism Severity and Leucovorin Treatment
Richard E Frye1,2, Alison Lane3,4, Ashley Worner3,4
1Rossignol Medical Center, Phoenix, AZ 85050, USA.
Insights
Leucovorin (folinic acid) treatment improved autism spectrum disorder (ASD) symptoms in children with soluble folate binding proteins (sFBPs). This suggests sFBPs may indicate treatment response in ASD patients.
Area of Science:
- Neurodevelopmental Disorders
- Biochemistry
- Clinical Medicine
Background:
- Autism spectrum disorder (ASD) is a complex neurodevelopmental condition affecting numerous children.
- Folate metabolism and its role in ASD are areas of ongoing research.
- Soluble folate binding proteins (sFBPs) have been detected in some individuals with ASD, but their clinical significance remains unclear.
Purpose of the Study:
- To investigate the presence and clinical significance of sFBPs in a cohort of children with ASD.
- To evaluate the efficacy and safety of leucovorin (folinic acid) treatment in ASD patients with sFBPs.
Main Methods:
- A cohort of medically complex children with ASD and positive sFBPs was identified.
- Open-label, baseline-controlled methodology was employed.
- Standardized assessments were used to evaluate ASD symptoms before and during leucovorin treatment.
Main Results:
- Patients with ASD and sFBPs exhibited significant improvements in core and associated symptoms following leucovorin treatment.
- No adverse effects were reported during the leucovorin intervention.
- This study is the first to report on sFBPs in ASD patients and their response to leucovorin.
Conclusions:
- Leucovorin treatment appears effective and safe for children with ASD who are positive for sFBPs.
- sFBPs may serve as potential biomarkers for predicting treatment response to leucovorin in ASD.
- Further controlled studies are warranted to confirm these findings and explore the role of sFBPs in ASD management.
Abstract:
Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder with life-long consequences that affects up to 1 in 44 children. Treatment with leucovorin (folinic acid), a reduced form of folate, has been shown to improve symptoms in those with ASD and folate pathway abnormalities in controlled clinical trials. Although soluble folate binding proteins (sFBPs) have been observed in the serum of some patients with ASD, the significance of this finding has not been studied. Here, we present a cohort of ASD patients with sFBPs. These patients had severe ASD and were medically complex. Using baseline controlled open-label methodology and standardized assessments, these patients were found to improve in both core and associated ASD symptoms with leucovorin treatment. No adverse effects were related to leucovorin treatment. This is the first report of the sFBPs in ASD. This study complements ongoing controlled clinical trials and suggests that leucovorin may be effective for children with ASD who are positive for sFBPs. Further, sFBPs might be important biomarkers for treatment response to leucovorin in children with ASD. This study paves the way for further controlled studies for patients with sFBPs.
