Moving Toward Patient-Tailored Treatment in ALS and FTD: The Potential of Genomic Assessment as a Tool for Biological Discovery and Trial Recruitment

Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are two devastating and intertwined neurodegenerative diseases. Historically, ALS and FTD were considered distinct disorders given differences in presenting clinical symptoms, disease duration, and predicted risk of developing eac...

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Main Authors:	Iris J. Broce, Patricia A. Castruita, Jennifer S. Yokoyama
Format:	Article
Language:	English
Published:	Frontiers Media S.A. 2021-03-01
Series:	Frontiers in Neuroscience
Subjects:	clinical trials genomics precision medicine amyotrophic lateral sclerosis frontotemporal dementia
Online Access:	https://www.frontiersin.org/articles/10.3389/fnins.2021.639078/full

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spelling	doaj-be084ca48992498b9e5640cd028cf5082021-03-01T05:14:12ZengFrontiers Media S.A.Frontiers in Neuroscience1662-453X2021-03-011510.3389/fnins.2021.639078639078Moving Toward Patient-Tailored Treatment in ALS and FTD: The Potential of Genomic Assessment as a Tool for Biological Discovery and Trial RecruitmentIris J. Broce0Iris J. Broce1Patricia A. Castruita2Jennifer S. Yokoyama3Jennifer S. Yokoyama4Memory and Aging Center, Department of Neurology, Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, United StatesDepartment of Family Medicine and Public Health, University of California, San Diego, San Diego, CA, United StatesMemory and Aging Center, Department of Neurology, Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, United StatesMemory and Aging Center, Department of Neurology, Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, United StatesDepartment of Radiology and Biomedical Imaging, University of California, San Francisco, San Francisco, CA, United StatesAmyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are two devastating and intertwined neurodegenerative diseases. Historically, ALS and FTD were considered distinct disorders given differences in presenting clinical symptoms, disease duration, and predicted risk of developing each disease. However, research over recent years has highlighted the considerable clinical, pathological, and genetic overlap of ALS and FTD, and these two syndromes are now thought to represent different manifestations of the same neuropathological disease spectrum. In this review, we discuss the need to shift our focus from studying ALS and FTD in isolation to identifying the biological mechanisms that drive these diseases—both common and distinct—to improve treatment discovery and therapeutic development success. We also emphasize the importance of genomic data to facilitate a “precision medicine” approach for treating ALS and FTD.https://www.frontiersin.org/articles/10.3389/fnins.2021.639078/fullclinical trialsgenomicsprecision medicineamyotrophic lateral sclerosisfrontotemporal dementia
collection	DOAJ
language	English
format	Article
sources	DOAJ
author	Iris J. Broce Iris J. Broce Patricia A. Castruita Jennifer S. Yokoyama Jennifer S. Yokoyama
spellingShingle	Iris J. Broce Iris J. Broce Patricia A. Castruita Jennifer S. Yokoyama Jennifer S. Yokoyama Moving Toward Patient-Tailored Treatment in ALS and FTD: The Potential of Genomic Assessment as a Tool for Biological Discovery and Trial Recruitment Frontiers in Neuroscience clinical trials genomics precision medicine amyotrophic lateral sclerosis frontotemporal dementia
author_facet	Iris J. Broce Iris J. Broce Patricia A. Castruita Jennifer S. Yokoyama Jennifer S. Yokoyama
author_sort	Iris J. Broce
title	Moving Toward Patient-Tailored Treatment in ALS and FTD: The Potential of Genomic Assessment as a Tool for Biological Discovery and Trial Recruitment
title_short	Moving Toward Patient-Tailored Treatment in ALS and FTD: The Potential of Genomic Assessment as a Tool for Biological Discovery and Trial Recruitment
title_full	Moving Toward Patient-Tailored Treatment in ALS and FTD: The Potential of Genomic Assessment as a Tool for Biological Discovery and Trial Recruitment
title_fullStr	Moving Toward Patient-Tailored Treatment in ALS and FTD: The Potential of Genomic Assessment as a Tool for Biological Discovery and Trial Recruitment
title_full_unstemmed	Moving Toward Patient-Tailored Treatment in ALS and FTD: The Potential of Genomic Assessment as a Tool for Biological Discovery and Trial Recruitment
title_sort	moving toward patient-tailored treatment in als and ftd: the potential of genomic assessment as a tool for biological discovery and trial recruitment
publisher	Frontiers Media S.A.
series	Frontiers in Neuroscience
issn	1662-453X
publishDate	2021-03-01
description	Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are two devastating and intertwined neurodegenerative diseases. Historically, ALS and FTD were considered distinct disorders given differences in presenting clinical symptoms, disease duration, and predicted risk of developing each disease. However, research over recent years has highlighted the considerable clinical, pathological, and genetic overlap of ALS and FTD, and these two syndromes are now thought to represent different manifestations of the same neuropathological disease spectrum. In this review, we discuss the need to shift our focus from studying ALS and FTD in isolation to identifying the biological mechanisms that drive these diseases—both common and distinct—to improve treatment discovery and therapeutic development success. We also emphasize the importance of genomic data to facilitate a “precision medicine” approach for treating ALS and FTD.
topic	clinical trials genomics precision medicine amyotrophic lateral sclerosis frontotemporal dementia
url	https://www.frontiersin.org/articles/10.3389/fnins.2021.639078/full
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Moving Toward Patient-Tailored Treatment in ALS and FTD: The Potential of Genomic Assessment as a Tool for Biological Discovery and Trial Recruitment

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