Insulin-Like Growth Factor 1 and Related Compounds in the Treatment of Childhood-Onset Neurodevelopmental Disorders

Insulin-Like Growth Factor 1 (IGF-1) is a neurotrophic polypeptide with crucial roles to play in Central Nervous System (CNS) growth, development and maturation. Following interrogation of the neurobiology underlying several neurodevelopmental disorders and Autism Spectrum Disorders (ASD), both reco...

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Main Authors: Cyrus Vahdatpour, Adam H Dyer, Daniela Tropea
Format: Article
Language:English
Published: Frontiers Media S.A. 2016-09-01
Series:Frontiers in Neuroscience
Subjects:
Online Access:http://journal.frontiersin.org/Journal/10.3389/fnins.2016.00450/full
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author Cyrus Vahdatpour
Adam H Dyer
Daniela Tropea
author_facet Cyrus Vahdatpour
Adam H Dyer
Daniela Tropea
author_sort Cyrus Vahdatpour
collection DOAJ
description Insulin-Like Growth Factor 1 (IGF-1) is a neurotrophic polypeptide with crucial roles to play in Central Nervous System (CNS) growth, development and maturation. Following interrogation of the neurobiology underlying several neurodevelopmental disorders and Autism Spectrum Disorders (ASD), both recombinant IGF-1 (mecasermin) and related derivatives, such as (1-3) IGF-1, have emerged as potential therapeutic approaches. Clinical pilot studies and early reports have supported the safety/preliminary efficacy of IGF-1 and related compounds in the treatment of Rett Syndrome, with evidence mounting for its use in Phelan McDermid Syndrome and Fragile X Syndrome. In broader ASD, clinical trials are ongoing. Here, we review the role of IGF-1 in the molecular etiologies of these conditions in addition to the accumulating evidence from early clinical studies highlighting the possibility of IGF-1 and related compounds as potential treatments for these childhood-onset neurodevelopmental disorders.
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spelling doaj.art-235cfbfcbb3d4df08f4842c038e9d0d62022-12-21T17:31:51ZengFrontiers Media S.A.Frontiers in Neuroscience1662-453X2016-09-011010.3389/fnins.2016.00450218672Insulin-Like Growth Factor 1 and Related Compounds in the Treatment of Childhood-Onset Neurodevelopmental DisordersCyrus Vahdatpour0Adam H Dyer1Daniela Tropea2Trinity College DublinTrinity College DublinSchool of MedicineInsulin-Like Growth Factor 1 (IGF-1) is a neurotrophic polypeptide with crucial roles to play in Central Nervous System (CNS) growth, development and maturation. Following interrogation of the neurobiology underlying several neurodevelopmental disorders and Autism Spectrum Disorders (ASD), both recombinant IGF-1 (mecasermin) and related derivatives, such as (1-3) IGF-1, have emerged as potential therapeutic approaches. Clinical pilot studies and early reports have supported the safety/preliminary efficacy of IGF-1 and related compounds in the treatment of Rett Syndrome, with evidence mounting for its use in Phelan McDermid Syndrome and Fragile X Syndrome. In broader ASD, clinical trials are ongoing. Here, we review the role of IGF-1 in the molecular etiologies of these conditions in addition to the accumulating evidence from early clinical studies highlighting the possibility of IGF-1 and related compounds as potential treatments for these childhood-onset neurodevelopmental disorders.http://journal.frontiersin.org/Journal/10.3389/fnins.2016.00450/fullFragile X SyndromeRett SyndromeAutism Spectrum DisordersIGF-1Phelan-McDermid Syndrome
spellingShingle Cyrus Vahdatpour
Adam H Dyer
Daniela Tropea
Insulin-Like Growth Factor 1 and Related Compounds in the Treatment of Childhood-Onset Neurodevelopmental Disorders
Frontiers in Neuroscience
Fragile X Syndrome
Rett Syndrome
Autism Spectrum Disorders
IGF-1
Phelan-McDermid Syndrome
title Insulin-Like Growth Factor 1 and Related Compounds in the Treatment of Childhood-Onset Neurodevelopmental Disorders
title_full Insulin-Like Growth Factor 1 and Related Compounds in the Treatment of Childhood-Onset Neurodevelopmental Disorders
title_fullStr Insulin-Like Growth Factor 1 and Related Compounds in the Treatment of Childhood-Onset Neurodevelopmental Disorders
title_full_unstemmed Insulin-Like Growth Factor 1 and Related Compounds in the Treatment of Childhood-Onset Neurodevelopmental Disorders
title_short Insulin-Like Growth Factor 1 and Related Compounds in the Treatment of Childhood-Onset Neurodevelopmental Disorders
title_sort insulin like growth factor 1 and related compounds in the treatment of childhood onset neurodevelopmental disorders
topic Fragile X Syndrome
Rett Syndrome
Autism Spectrum Disorders
IGF-1
Phelan-McDermid Syndrome
url http://journal.frontiersin.org/Journal/10.3389/fnins.2016.00450/full
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