Update on the Treatment of Ataxia: Medication and Emerging Therapies

Perlman, Susan

doi:10.1007/s13311-020-00941-3

Cited by 22 publications

(13 citation statements)

References 51 publications

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“…Yet, CNS drug developers practice their craft by adhering to two conflicting beliefs: (a) drugs for CNS disease can be developed, and (b) CNS drug development can take place in the absence of any consideration of the blood-brain barrier. These contradictory beliefs are illustrated by recent reviews of drug development for AD [1202][1203][1204][1205], PD [755,1206], stroke [1207], brain cancer [1208], Huntington's disease [1209], ALS [1210], ataxia [1211], spinal cord injury [1212], traumatic brain injury [1213], or addiction [1214]. In none of these reviews on drug development for specific brain diseases was the BBB even mentioned, so the crucial issue of brain drug delivery was uniformly in absentia in the CNS drug development process.…”

Section: Perspectivementioning

confidence: 99%

A Historical Review of Brain Drug Delivery

2022

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The history of brain drug delivery is reviewed beginning with the first demonstration, in 1914, that a drug for syphilis, salvarsan, did not enter the brain, due to the presence of a blood–brain barrier (BBB). Owing to restricted transport across the BBB, FDA-approved drugs for the CNS have been generally limited to lipid-soluble small molecules. Drugs that do not cross the BBB can be re-engineered for transport on endogenous BBB carrier-mediated transport and receptor-mediated transport systems, which were identified during the 1970s–1980s. By the 1990s, a multitude of brain drug delivery technologies emerged, including trans-cranial delivery, CSF delivery, BBB disruption, lipid carriers, prodrugs, stem cells, exosomes, nanoparticles, gene therapy, and biologics. The advantages and limitations of each of these brain drug delivery technologies are critically reviewed.

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Section: Perspectivementioning

confidence: 99%

A Historical Review of Brain Drug Delivery

2022

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“…These disorders are complicated with motor restrictions as well as autonomic dysfunction and neuropsychiatric symptoms, significantly affecting the patients' quality of life [23,24]. Unfortunately, treatment options remain limited, and development of new therapies continues to be an urgent priority [25,26].…”

Section: Discussionmentioning

confidence: 99%

Non‐invasive electrical stimulation in patients with neurodegenerative ataxia and spasticity: A systematic review and meta‐analysis of randomized controlled trials

Shengting

Yong

Lim

et al. 2022

Euro J of Neurology

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Ataxia and spasticity are manifestations of a variety of neurodegenerative diseases such as spinocerebellar ataxias, Friedreich's ataxia and multiple sclerosis, and their debilitating effects significantly affect the patients' quality of life [1][2][3]. Of note, hereditary forms of ataxia and spasticity are increasingly viewed as diseases of the same spectrum, for which management options remain poorly studied [4,5]. Unfortunately, there are currently no effective disease-modifying or curative drugs, and the heterogeneous aetiologies of neurodegenerative ataxia and spasticity create a significant challenge to the development of new therapies [5].Non-invasive electrostimulation (NES) is receiving increasing interest particularly because it is portable and cost-effective. Furthermore, it may provide new information regarding brain physiology as well as enhance neural plasticity in neurodegenerative ataxia and spasticity, without exposing the patients to the risks associated with invasive therapies such as deep brain stimulation

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“…A phase 3 study of troriluzole in the more common SCAs was recently completed, although results have not yet been published in a peer-reviewed journal. Other therapies are in earlier stages of development, 71 increasing the possibilities for new symptomatic or even disease-modifying therapies in the future.…”

Section: Treatmentmentioning

confidence: 99%

Neurodegenerative Cerebellar Ataxia

Rosenthal¹

2022

CONTINUUM: Lifelong Learning in Neurology

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PURPOSE OF REVIEW: Neurodegenerative cerebellar ataxia is a diverse collection of diseases that are unified by gait and balance abnormalities, appendicular incoordination, and abnormalities of eye movement and speech. The differential diagnosis is broad, ranging from paraneoplastic syndromes that progress quite rapidly to unidentified genetic disorders that progress slowly over the course of decades. This article highlights the diagnostic process, including the differential diagnosis, as well as treatment approaches and symptomatic management. The pillars of treatment are physical, occupational, and speech therapy as well as counseling and discussions of disease prognosis, genetics, and reproductive choices. There are many ways to help patients with neurodegenerative cerebellar ataxia and improve their quality of life.RECENT FINDINGS: Recent years have seen significant improvements in genetic testing, with reductions in cost of both Sanger sequencing and whole exome sequencing and increasing availability of the latter. These improvements increase clinicians' ability to identify the etiology of neurodegenerative cerebellar ataxia and suggest future treatments. Although no medication has been approved by the US Food and Drug Administration (FDA) for treatment of cerebellar ataxia, research and clinical trials for these diseases are increasing. SUMMARY: Neurodegenerative cerebellar ataxia is characterized by dysarthria, dysmetria, oculomotor abnormalities, and ataxic gait. It has a broad differential diagnosis, and numerous options exist for managing symptoms. Although no medications have been approved specifically for cerebellar ataxia, treatment options are available to improve patients' quality of life.

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Update on the Treatment of Ataxia: Medication and Emerging Therapies

Cited by 22 publications

References 51 publications

A Historical Review of Brain Drug Delivery

A Historical Review of Brain Drug Delivery

Non‐invasive electrical stimulation in patients with neurodegenerative ataxia and spasticity: A systematic review and meta‐analysis of randomized controlled trials

Neurodegenerative Cerebellar Ataxia

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