Emerging therapies and recent advances for Tourette syndrome

Chou, Chih-Yi; Agin‐Liebes, Julian; Kuo, Sheng‐Han

doi:10.1016/j.heliyon.2023.e12874

Cited by 5 publications

(2 citation statements)

References 114 publications

(147 reference statements)

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“…The use of DBS for severe and pharmaco-resistant Tourette syndrome in childhood is controversial, given its potential for not only improvement but also the invasive nature, risk of complications, and the natural history of Tourette syndrome to improve with maturity [82]. Target areas include the thalamic centromedian parafascicular complex and the GPi [83]. Patient selection is key, with prior work proposing failure of behavioral and pharmacologic therapies, high tic severity and negative impact on quality of life as criteria [84].…”

Section: Deep Brain Stimulation In Tourette Syndrome and Self-injurio...mentioning

confidence: 99%

Emerging therapies for childhood-onset movement disorders

Vogt,

Quiroz,

Ebrahimi-Fakhari

2024

Current Opinion in Pediatrics

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Purpose of review We highlight novel and emerging therapies in the treatment of childhood-onset movement disorders. We structured this review by therapeutic entity (small molecule drugs, RNA-targeted therapeutics, gene replacement therapy, and neuromodulation), recognizing that there are two main approaches to treatment: symptomatic (based on phenomenology) and molecular mechanism-based therapy or ‘precision medicine’ (which is disease-modifying). Recent findings We highlight reports of new small molecule drugs for Tourette syndrome, Friedreich's ataxia and Rett syndrome. We also discuss developments in gene therapy for aromatic l-amino acid decarboxylase deficiency and hereditary spastic paraplegia, as well as current work exploring optimization of deep brain stimulation and lesioning with focused ultrasound. Summary Childhood-onset movement disorders have traditionally been treated symptomatically based on phenomenology, but focus has recently shifted toward targeted molecular mechanism-based therapeutics. The development of precision therapies is driven by increasing capabilities for genetic testing and a better delineation of the underlying disease mechanisms. We highlight novel and exciting approaches to the treatment of genetic childhood-onset movement disorders while also discussing general challenges in therapy development for rare diseases. We provide a framework for molecular mechanism-based treatment approaches, a summary of specific treatments for various movement disorders, and a clinical trial readiness framework.

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Section: Deep Brain Stimulation In Tourette Syndrome and Self-injurio...mentioning

confidence: 99%

Emerging therapies for childhood-onset movement disorders

Vogt,

Quiroz,

Ebrahimi-Fakhari

2024

Current Opinion in Pediatrics

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“…The SN connectivity changes in TD have yet to be investigated. Currently, there is a lack of sufficient treatment strategies with respect to this disorder ( Pringsheim et al, 2019 ; Chou et al, 2023 ; Johnson et al, 2023 ), indicating an urgent need for pathophysiological knowledge that can bridge the gap between behavior, the brain, and TD treatment.…”

Section: Introductionmentioning

confidence: 99%

Aberrant Functional Connectivity of the Salience Network in Adult Patients with Tic Disorders: A Resting-State fMRI Study

Orth,

Meeh,

Leiding

et al. 2024

eNeuro

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Tic disorders (TD) are characterized by the presence of motor and/or vocal tics. Common neurophysiological frameworks suggest dysregulations of the cortico-striatal-thalamo-cortical (CSTC) brain circuit that controls movement execution. Besides the common tics, there are other “non-tic” symptoms that are primarily related to sensory perception, sensorimotor integration, attention, and social cognition. The existence of these symptoms, the sensory tic triggers and the modifying effect of attention and cognitive control mechanisms on tics may indicate the salience network's involvement in the neurophysiology of TD. Resting-state functional MRI measurements were performed in 26 participants with TD and 25 healthy controls. The group differences in resting-state functional connectivity patterns were measured based on seed-to-voxel connectivity analyses. Compared to healthy controls, patients with TD exhibited altered connectivity between the core regions of the salience network (insula, ACC and TPJ) and sensory, associative, and motor-related cortices. Furthermore, connectivity changes were observed in relation to the severity of tics in the TD group. The salience network, particularly the insula, is likely to be an important site of dysregulation in TD. Our results provide evidence for large-scale neural deviations in TD beyond the CSTC pathologies. These findings may be relevant for developing treatment targets.Significance statementTic disorders (TD) are associated with a variety of symptoms beyond typical motor and vocal tics that affect sensory perception, attention, and social cognition. The presence of such non-tic symptoms suggests the potential involvement of the salience network in the pathophysiology of TD. While previous studies have predominantly focused on the cortico-striato-thalamo-cortical (CSTC) circuitry, which is known to underlie tic generation and expression, we conducted resting-state fMRI to investigate the functional connectivity of the salience network in TD. Notably, we observed impaired connectivity of the salience network with relations to the tic symptom severity. Our research provided important evidence that the pathophysiology of TD involves the salience network, which is highly relevant for developing treatment strategies.

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