Investigational drugs in early-stage clinical trials for autism spectrum disorder

@article{Hong2019InvestigationalDI,
  title={Investigational drugs in early-stage clinical trials for autism spectrum disorder},
  author={Michael P. Hong and C. Erickson},
  journal={Expert Opinion on Investigational Drugs},
  year={2019},
  volume={28},
  pages={709 - 718}
}
ABSTRACT Introduction: Pharmacologic interventions in Autism Spectrum Disorder (ASD) have historically focused on symptom-based approaches. However, a treatment for the core social deficits has remained unidentified. While a definitive theory for the cause of ASD is not yet known, recent advances in our understanding of ASD pathophysiology have opened the door for research on new pharmaceutical methods to target core symptomology. Areas covered: Herein, we review the novel pharmacologic… Expand
Prevention in Autism Spectrum Disorder: A Lifelong Focused Approach
TLDR
This paper takes a life course approach to prevention, reviewing the possibilities of the management of risk factors from preconception until the perinatal period, that of early intervention in the first three years of life and that of effective training and support from childhood until adulthood. Expand
Methylphenidate in Autism Spectrum Disorder: A Long-Term Follow Up Naturalistic Study
TLDR
Long-term MPH was effective and well-tolerated in ADHD children and adolescents with comorbid high functioning ASD, and was associated with significative reduction of illness severity, clinical improvement and amelioration of global functioning. Expand
Bumetanide Oral Liquid Formulation for the Treatment of Children and Adolescents with Autism Spectrum Disorder: Design of Two Phase III Studies (SIGN Trials)
TLDR
Two Phase III studies evaluating the efficacy/safety of bumetanide oral liquid formulation in ASD could contribute to the first pharmacological treatment to improve social reciprocity and limit repetitive and rigid behaviors in children and adolescents with ASD. Expand
Cortical Gray Matter Injury in Encephalopathy of Prematurity: Link to Neurodevelopmental Disorders
TLDR
The current state of knowledge in this field is reviewed, amalgamating data from both clinical and preclinical studies, in the context of normal processes of developmental biology and the known pathophysiology of neurodevelopmental disorders. Expand
12953_Cortical Gray Matter Injury in Encephalopathy of Prematurity/ Link to Neurodevelopmental Disorders_GOLD VoR
Preterm-born infants frequently suffer from an array of neurological damage, collectively termed encephalopathy of prematurity (EoP). They also have an increased risk of presenting with aExpand
S0033291721000192jrv 1..14
Autism spectrum disorder (autism) is a heterogeneous group of neurodevelopmental conditions characterized by early childhood-onset impairments in communication and social interaction alongsideExpand
Association of Peripheral Blood Levels of Cytokines With Autism Spectrum Disorder: A Meta-Analysis
TLDR
The findings of the meta-analysis strengthen the evidence for an abnormal cytokine profile in ASD and these abnormal cytokines may be potential biomarkers for the diagnosis and treatment of ASD in the future. Expand
Social Cognition and Schizophrenia: Unresolved Issues and New Challenges in a Maturing Field of Research
TLDR
Continued efforts to creatively grapple with the complexities and challenges the field now faces hold great promise for helping us understand and more effectively treat a major source of functional disability in schizophrenia. Expand

References

SHOWING 1-10 OF 130 REFERENCES
Emerging drugs for the treatment of symptoms associated with autism spectrum disorders
TLDR
This review examines results from neurobiologic research in an attempt to both elucidate the pathophysiology of autism and guide the development of pharmacologic agents for the treatment of associated symptoms. Expand
Early Pharmacological Treatment of Autism: A Rationale for Developmental Treatment
TLDR
Early treatment with selective serotonin reuptake inhibitors (SSRIs) is presented as a model for pharmacologic interventions because there is evidence in autistic children for reduced brain serotonin synthesis during periods of peak synaptogenesis and serotonin is known to enhance synapse refinement. Expand
Arbaclofen in Children and Adolescents with Autism Spectrum Disorder: A Randomized, Controlled, Phase 2 Trial
Several lines of emerging data point to an imbalance between neuronal excitation and inhibition in at least a subgroup of individuals with autism spectrum disorder (ASD), including in those withExpand
The role of immune dysfunction in the pathophysiology of autism
TLDR
The current state of knowledge of immune dysfunction in ASD is discussed, how these findings may impact on underlying neuro-immune mechanisms and implicate potential areas where the manipulation of the immune response could have an impact on behavior and immunity in ASD. Expand
Immune dysfunction in autism: A pathway to treatment
TLDR
Reports suggest that autism may in fact be a systemic disorder with connections to abnormal immune responses, and how early brain development is altered, may have important therapeutic implications. Expand
Intranasal oxytocin treatment for social deficits and biomarkers of response in children with autism
TLDR
Findings indicate that OXT treatment enhances social abilities in children with ASD and that individuals with pretreatment OXT signaling deficits may stand to benefit the most from Oxt treatment. Expand
Reversal of autism-like behaviors and metabolism in adult mice with single-dose antipurinergic therapy
TLDR
It is found that disturbances in social behavior, novelty preference and metabolism are not permanent but are treatable with antipurinergic therapy (APT) in this model of ASD and schizophrenia, and that single-dose APT with suramin acutely reverses these abnormalities, even in adults. Expand
Pharmacologic treatments for the behavioral symptoms associated with autism spectrum disorders across the lifespan
TLDR
Overall, SRIs are less efficacious and more poorly tolerated in children with ASDs than in adults, and antipsychotics are the most efficacious drugs for the treatment of irritability in ASDs, and may be useful in the Treatment of other symptoms. Expand
Efficacy of Fetal Stem Cell Transplantation in Autism Spectrum Disorders: An Open-Labeled Pilot Study
TLDR
The safety and efficacy of FSC transplantations in treating children diagnosed with ASDs are investigated, and Statistically significant differences were shown on ATEC/ABC scores for the domains of speech, sociability, sensory, and overall health, as well as reductions in the total scores when compared to pretreatment values. Expand
Clinical and neural effects of six-week administration of oxytocin on core symptoms of autism.
TLDR
6-week intranasal administration of oxytocin significantly reduced autism core symptoms specific to social reciprocity, which was clinically evaluated by Autism Diagnostic Observation Scale and confirmed that the current continual administration significantly mitigated behavioural and neural responses during the task, both of which were originally impaired in autistic individuals. Expand
...
1
2
3
4
5
...