Tetrahydrobiopterin as a novel therapeutic intervention for autism.

Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics
R E FryeGlen R Elliott

Abstract

Tetrahydrobiopterin (BH(4)) is an essential cofactor for several critical metabolic pathways that have been reported to be abnormal in autism spectrum disorder (ASD). In addition, the cerebrospinal fluid concentration of BH(4) is reported to be depressed in children with ASD. Over the past 25 years, several clinical trials have suggested that treatment with BH(4) improves ASD symptomatology in some individuals. Two ongoing clinical protocols may help further define the efficacy of BH(4) treatment in children with ASD. First, children with ASD who had low concentrations of cerebrospinal fluid or urine pterins were treated in an open-label manner with 20 mg/kg per day of BH(4). The majority of children (63%) responded positively to treatment, with minimal adverse events (AEs). Second, a double-blind placebo-controlled study examining the efficacy of 20 mg/kg per day of BH(4) treatment in children with ASD is currently underway. Safety studies from the commercially available forms of BH(4) document the low incidence of AEs, particularly serious AEs. Studies have also documented the ability of BH(4) to cross the blood-brain barrier. Based on the importance of BH(4) in neurodevelopmental metabolic pathways, the safety of BH(4) treat...Continue Reading

Associated Clinical Trials

References

Apr 1, 1992·Journal of Child Neurology·A al AqeelS B Subramanyam
May 22, 1981·Science·G Kapatos, S Kaufman
Dec 31, 1997·The European Journal of Neuroscience·P Z AnastasiadisR A Levine
Dec 8, 1998·Journal of Neuroscience Research·K KoshimuraY Kato
Mar 23, 2000·The Biochemical Journal·B ThönyN Blau
Mar 6, 2004·CNS Spectrums·Benedetto Vitiello, Ann Wagner
Dec 9, 2004·The American Journal of Clinical Nutrition·S Jill JamesJames A Neubrander
Sep 15, 2005·Journal of Clinical Psychopharmacology·Torsten DanforsOrvar Eeg-Olofsson
Jan 13, 2006·International Review of Psychiatry·Carlos A PardoAndrew W Zimmerman
Mar 24, 2006·Journal of Child and Adolescent Psychopharmacology·David J PoseyChristopher J McDougle
May 3, 2006·The Journal of Clinical Psychiatry·Alexander KolevzonEric Hollander
Aug 15, 2006·Behavioral Medicine·Ludmila Cofta-WoerpelDavid W Wetter
Oct 31, 2006·Journal of Child and Adolescent Psychopharmacology·David J PoseyChristopher J McDougle
Oct 31, 2006·Journal of Child and Adolescent Psychopharmacology·Pieter W TroostPieter J Hoekstra
Nov 9, 2006·Journal of Toxicology and Environmental Health. Part B, Critical Reviews·Janet K Kern, Anne M Jones
Oct 9, 2007·Brain Pathology·Carlos A Pardo, Charles G Eberhart
Apr 19, 2008·Journal of Neurochemistry·Beat ThönyAurora Martinez
Jul 1, 2008·The European Journal of Neuroscience·John Garthwaite
Sep 9, 2008·Child and Adolescent Psychiatric Clinics of North America·Latha SooryaEric Hollander
Sep 30, 2008·Journal of Toxicology and Environmental Health. Part B, Critical Reviews·Rodney R Dietert, Janice M Dietert
Mar 25, 2009·International Journal of Immunopathology and Pharmacology·M L CastellaniM Fulcheri
Jun 29, 2013·Child and Adolescent Psychiatric Clinics of North America·Robert L Hendren

❮ Previous
Next ❯

Citations

Jun 21, 2013·Journal of Child and Adolescent Psychopharmacology·Cheryl KlaimanGlen R Elliott
Apr 10, 2014·Biomarkers in Medicine·Richard E Frye, S Jill James
Nov 20, 2012·Research in Autism Spectrum Disorders·C Kağan Gürkan, Randi J Hagerman
Feb 14, 2012·The Journal of Pediatrics·Nicola J RobertsonCarina Mallard
Jul 29, 2015·Molecular Neurobiology·Kamil K HozyaszPaweł P Jagodziński
Aug 27, 2011·The Biochemical Journal·Ernst R WernerBeat Thöny
Oct 29, 2014·Journal of Toxicology·Christopher A ShawRobert M Davidson
Jan 8, 2014·Autism Research and Treatment·Joanna C HamlinS Jill James
Apr 10, 2013·Medical Hypotheses·Mark Lucock, Robert Leeming
Apr 23, 2016·Pharmacology & Therapeutics·Hans O Kalkman, Dominik Feuerbach
Oct 24, 2013·Saudi Pharmaceutical Journal : SPJ : the Official Publication of the Saudi Pharmaceutical Society·Abdulrahman S Alanazi
Jul 16, 2014·Frontiers in Pediatrics·Richard Eugene Frye, Daniel A Rossignol
May 2, 2016·Human Genetics·Jinting GuanJames J Cai
May 18, 2016·Translational Psychiatry·R E Frye, J Slattery
Aug 5, 2017·Biomarkers in Medicine·Richard E FryeEdward V Quadros
Jan 8, 2018·Molecular Neurobiology·Dominika GładyszKamil K Hozyasz
Dec 6, 2019·Current Pharmaceutical Design·Iliyana Pacheva, Ivan Ivanov
Jun 23, 2016·Clinical Medicine Insights. Pediatrics·Richard E Frye, Daniel A Rossignol
Jun 20, 2019·Behavioral Sciences·María Andrea CastilloDario Siniscalco
Sep 3, 2021·Oxidative Medicine and Cellular Longevity·Natália Cruz-MartinsWilliam C Cho

❮ Previous
Next ❯

Related Concepts

Related Feeds

Blood Brain Barrier Regulation in Health & Disease

The blood brain barrier is essential in regulating the movement of molecules and substances in and out of the brain. Disruption to the blood brain barrier and changes in permeability allow pathogens and inflammatory molecules to cross the barrier and may play a part in the pathogenesis of neurodegenerative disorders. Here is the latest research in this field.

Anxiety Disorders

Discover the latest research on anxiety disorders including agoraphobia, panic disorder, obsessive-compulsive disorder, and post-traumatic stress disorder here.

Blood Brain Barrier

The blood brain barrier is a border that separates blood from cerebrospinal fluid. Discover the latest search on this highly selective semipermeable membrane here.

Blood Brain Barrier Chips

The blood brain barrier (BBB) is comprised of endothelial cells that regulate the influx and outflux of plasma concentrations. Lab-on-a-chip devices allow scientists to model diseases and mechanisms such as the passage of therapeutic antibodies across the BBB. Discover the latest research on BBB chips here.

CSF & Lymphatic System

This feed focuses on Cerebral Spinal Fluid (CSF) and the lymphatic system. Discover the latest papers using imaging techniques to track CSF outflow into the lymphatic system in animal models.

Autism

Autism spectrum disorder is associated with challenges with social skills, repetitive behaviors, and often accompanied by sensory sensitivities and medical issues. Here is the latest research on autism.

Blood Brain Barrier & Cytokines

Some cytokines are able to cross the blood brain barrier through transport systems and enter the cerebrospinal fluid and interstitial fluid spaces. Here is the latest research on cytokines crossing the blood brain barrier and how this can affect tissues within the CNS.