PMID- 20643376
OWN - NLM
STAT- MEDLINE
DCOM- 20101025
LR  - 20240204
IS  - 1878-7479 (Electronic)
IS  - 1933-7213 (Print)
IS  - 1878-7479 (Linking)
VI  - 7
IP  - 3
DP  - 2010 Jul
TI  - Tetrahydrobiopterin as a novel therapeutic intervention for autism.
PG  - 241-9
LID - 10.1016/j.nurt.2010.05.004 [doi]
AB  - 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) 
      treatment, and the evidence for a therapeutic benefit of BH(4) treatment in 
      children with ASD, we conclude that BH(4) represents a novel therapy for ASD, one 
      that may gain wider use after further clinical studies have established efficacy 
      and treatment guidelines.
CI  - (c) 2010 The American Society for Experimental NeuroTherapeutics, Inc. Published 
      by Elsevier Inc. All rights reserved.
FAU - Frye, Richard E
AU  - Frye RE
AD  - Division of Child and Adolescent Neurology and The Children's Learning Institute, 
      Department of Pediatrics, University of Texas Health Science Center at Houston, 
      Houston, Texas 77030, USA. Richard.E.Frye@uth.tmc.edu
FAU - Huffman, Lynne C
AU  - Huffman LC
FAU - Elliott, Glen R
AU  - Elliott GR
LA  - eng
GR  - K23 NS046565-04/NS/NINDS NIH HHS/United States
GR  - NS046565/NS/NINDS NIH HHS/United States
GR  - K23 NS046565-05/NS/NINDS NIH HHS/United States
GR  - K23 NS046565/NS/NINDS NIH HHS/United States
GR  - K23 NS046565-03/NS/NINDS NIH HHS/United States
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
PT  - Research Support, Non-U.S. Gov't
PT  - Review
PL  - United States
TA  - Neurotherapeutics
JT  - Neurotherapeutics : the journal of the American Society for Experimental 
      NeuroTherapeutics
JID - 101290381
RN  - 0 (Neurotransmitter Agents)
RN  - 0 (Biopterins)
RN  - 31C4KY9ESH (Nitric Oxide)
RN  - EGX657432I (sapropterin)
SB  - IM
MH  - Autistic Disorder/*drug therapy/*metabolism
MH  - Biopterins/*analogs & derivatives/therapeutic use
MH  - Humans
MH  - Neurotransmitter Agents/*metabolism
MH  - Nitric Oxide/*metabolism
PMC - PMC2908599
MID - NIHMS209683
EDAT- 2010/07/21 06:00
MHDA- 2010/10/26 06:00
PMCR- 2011/07/01
CRDT- 2010/07/21 06:00
PHST- 2010/02/04 00:00 [received]
PHST- 2010/05/11 00:00 [revised]
PHST- 2010/05/22 00:00 [accepted]
PHST- 2010/07/21 06:00 [entrez]
PHST- 2010/07/21 06:00 [pubmed]
PHST- 2010/10/26 06:00 [medline]
PHST- 2011/07/01 00:00 [pmc-release]
AID - S1878-7479(23)00221-0 [pii]
AID - 70300241 [pii]
AID - 10.1016/j.nurt.2010.05.004 [doi]
PST - ppublish
SO  - Neurotherapeutics. 2010 Jul;7(3):241-9. doi: 10.1016/j.nurt.2010.05.004.