open access

Vol 54, No 3 (2020)
Invited review article
Published online: 2020-06-18
Submitted: 2020-04-12
Accepted: 2020-05-03
Get Citation

Advancing gene therapies, methods, and technologies for Parkinson’s Disease and other neurological disorders

Massimo S. Fiandaca, Russell R. Lonser, J. Bradley Elder, Mirosław Ząbek, Krystof S. Bankiewicz
DOI: 10.5603/PJNNS.a2020.0046
·
Pubmed: 32557526
·
Neurol Neurochir Pol 2020;54(3):220-231.

open access

Vol 54, No 3 (2020)
Invited review article
Published online: 2020-06-18
Submitted: 2020-04-12
Accepted: 2020-05-03

Abstract

Introduction. Vector-based intracerebral gene therapies are being used to treat specific neurodegenerative conditions such as Parkinson’s Disease (PD). This review presents a basis for central nervous system (CNS) gene therapy treatments of neurodegenerative diseases such as PD, as well as the need for novel skill sets and health delivery strategies within the clinical neurosciences (neurology and neurosurgery) to meet future demand for such therapies.

State of the art. Preclinical vector-based gene therapy approaches have been translated into clinical trials for PD and other neurodegenerative conditions. Unfortunately, such trials, and parallel efforts using other therapeutics, have yet to provide a breakthrough. Image-guided convection enhanced delivery (CED) optimises the parenchymal distribution of gene therapies applied within the CNS, and may ultimately provide such a breakthrough.

Clinical implications. Currently, image-guided CED and gene therapy are not part of training programmes for most neurosurgeons and neurologists. As a result, few medical centres and hospitals have sufficiently experienced teams to participate in gene transfer clinical trials for PD or other neurological conditions. If CNS gene therapies prove to be efficacious for PD and/or other conditions, the demand for such treatments will overwhelm the available number of experienced clinical neuroscience teams and treatment centres.

Future directions. Expanded indications and demand for CNS gene therapies will require a worldwide educational effort to supplement the training of clinical neuroscience practitioners. Initially, a limited number of Centres of Excellence will need to establish relevant educational training requirements and best practice for such therapeutic approaches. Advanced technologies, including robotics and artificial intelligence, are especially germane in this regard, and will expand the treatment team’s capabilities while assisting in the safe and timely care of those afflicted.

Abstract

Introduction. Vector-based intracerebral gene therapies are being used to treat specific neurodegenerative conditions such as Parkinson’s Disease (PD). This review presents a basis for central nervous system (CNS) gene therapy treatments of neurodegenerative diseases such as PD, as well as the need for novel skill sets and health delivery strategies within the clinical neurosciences (neurology and neurosurgery) to meet future demand for such therapies.

State of the art. Preclinical vector-based gene therapy approaches have been translated into clinical trials for PD and other neurodegenerative conditions. Unfortunately, such trials, and parallel efforts using other therapeutics, have yet to provide a breakthrough. Image-guided convection enhanced delivery (CED) optimises the parenchymal distribution of gene therapies applied within the CNS, and may ultimately provide such a breakthrough.

Clinical implications. Currently, image-guided CED and gene therapy are not part of training programmes for most neurosurgeons and neurologists. As a result, few medical centres and hospitals have sufficiently experienced teams to participate in gene transfer clinical trials for PD or other neurological conditions. If CNS gene therapies prove to be efficacious for PD and/or other conditions, the demand for such treatments will overwhelm the available number of experienced clinical neuroscience teams and treatment centres.

Future directions. Expanded indications and demand for CNS gene therapies will require a worldwide educational effort to supplement the training of clinical neuroscience practitioners. Initially, a limited number of Centres of Excellence will need to establish relevant educational training requirements and best practice for such therapeutic approaches. Advanced technologies, including robotics and artificial intelligence, are especially germane in this regard, and will expand the treatment team’s capabilities while assisting in the safe and timely care of those afflicted.

Get Citation

Keywords

gene therapy, convection-enhanced delivery, Parkinson’s Disease, clinical neuroscience education, robotics, artificial intelligence

About this article
Title

Advancing gene therapies, methods, and technologies for Parkinson’s Disease and other neurological disorders

Journal

Neurologia i Neurochirurgia Polska

Issue

Vol 54, No 3 (2020)

Pages

220-231

Published online

2020-06-18

DOI

10.5603/PJNNS.a2020.0046

Pubmed

32557526

Bibliographic record

Neurol Neurochir Pol 2020;54(3):220-231.

Keywords

gene therapy
convection-enhanced delivery
Parkinson’s Disease
clinical neuroscience education
robotics
artificial intelligence

Authors

Massimo S. Fiandaca
Russell R. Lonser
J. Bradley Elder
Mirosław Ząbek
Krystof S. Bankiewicz

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