Focused Ultrasound Mediated Opening of the Blood-Brain Barrier for Neurodegenerative Diseases

The blood brain barrier (BBB) is an obstacle for the delivery of potential molecular therapies for neurodegenerative diseases such as Parkinson's disease (PD), Alzheimer's disease (AD), and amyotrophic lateral sclerosis (ALS). Although there has been a proliferation of potential disease mo...

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Main Authors: Paul S. Fishman, Jonathan M. Fischell
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-11-01
Series:Frontiers in Neurology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2021.749047/full
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author Paul S. Fishman
Jonathan M. Fischell
author_facet Paul S. Fishman
Jonathan M. Fischell
author_sort Paul S. Fishman
collection DOAJ
description The blood brain barrier (BBB) is an obstacle for the delivery of potential molecular therapies for neurodegenerative diseases such as Parkinson's disease (PD), Alzheimer's disease (AD), and amyotrophic lateral sclerosis (ALS). Although there has been a proliferation of potential disease modifying therapies for these progressive conditions, strategies to deliver these large agents remain limited. High intensity MRI guided focused ultrasound has already been FDA approved to lesion brain targets to treat movement disorders, while lower intensity pulsed ultrasound coupled with microbubbles commonly used as contrast agents can create transient safe opening of the BBB. Pre-clinical studies have successfully delivered growth factors, antibodies, genes, viral vectors, and nanoparticles in rodent models of AD and PD. Recent small clinical trials support the safety and feasibility of this strategy in these vulnerable patients. Further study is needed to establish safety as MRI guided BBB opening is used to enhance the delivery of newly developed molecular therapies.
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spelling doaj.art-0e5158b818394293a24b3466b87aae1d2023-01-13T14:28:23ZengFrontiers Media S.A.Frontiers in Neurology1664-22952021-11-011210.3389/fneur.2021.749047749047Focused Ultrasound Mediated Opening of the Blood-Brain Barrier for Neurodegenerative DiseasesPaul S. FishmanJonathan M. FischellThe blood brain barrier (BBB) is an obstacle for the delivery of potential molecular therapies for neurodegenerative diseases such as Parkinson's disease (PD), Alzheimer's disease (AD), and amyotrophic lateral sclerosis (ALS). Although there has been a proliferation of potential disease modifying therapies for these progressive conditions, strategies to deliver these large agents remain limited. High intensity MRI guided focused ultrasound has already been FDA approved to lesion brain targets to treat movement disorders, while lower intensity pulsed ultrasound coupled with microbubbles commonly used as contrast agents can create transient safe opening of the BBB. Pre-clinical studies have successfully delivered growth factors, antibodies, genes, viral vectors, and nanoparticles in rodent models of AD and PD. Recent small clinical trials support the safety and feasibility of this strategy in these vulnerable patients. Further study is needed to establish safety as MRI guided BBB opening is used to enhance the delivery of newly developed molecular therapies.https://www.frontiersin.org/articles/10.3389/fneur.2021.749047/fullFUSblood-brain barrierAlzheimer's diseaseParkinson's diseasefocused ultrasound (MRgFUS)
spellingShingle Paul S. Fishman
Jonathan M. Fischell
Focused Ultrasound Mediated Opening of the Blood-Brain Barrier for Neurodegenerative Diseases
Frontiers in Neurology
FUS
blood-brain barrier
Alzheimer's disease
Parkinson's disease
focused ultrasound (MRgFUS)
title Focused Ultrasound Mediated Opening of the Blood-Brain Barrier for Neurodegenerative Diseases
title_full Focused Ultrasound Mediated Opening of the Blood-Brain Barrier for Neurodegenerative Diseases
title_fullStr Focused Ultrasound Mediated Opening of the Blood-Brain Barrier for Neurodegenerative Diseases
title_full_unstemmed Focused Ultrasound Mediated Opening of the Blood-Brain Barrier for Neurodegenerative Diseases
title_short Focused Ultrasound Mediated Opening of the Blood-Brain Barrier for Neurodegenerative Diseases
title_sort focused ultrasound mediated opening of the blood brain barrier for neurodegenerative diseases
topic FUS
blood-brain barrier
Alzheimer's disease
Parkinson's disease
focused ultrasound (MRgFUS)
url https://www.frontiersin.org/articles/10.3389/fneur.2021.749047/full
work_keys_str_mv AT paulsfishman focusedultrasoundmediatedopeningofthebloodbrainbarrierforneurodegenerativediseases
AT jonathanmfischell focusedultrasoundmediatedopeningofthebloodbrainbarrierforneurodegenerativediseases