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

Iris J. Broce¹  Jennifer S. Yokoyama²  Patricia A. Castruita³ 

[1] Department of Family Medicine and Public Health, University of California, San Diego, San Diego, CA, United States;Department of Radiology and Biomedical Imaging, University of California, San Francisco, San Francisco, CA, United States;Memory and Aging Center, Department of Neurology, Weill Institute for Neurosciences, University of California, San Francisco, San Francisco, CA, United States;

关键词: clinical trials;    genomics;    precision medicine;    amyotrophic lateral sclerosis;    frontotemporal dementia;   

DOI  :  10.3389/fnins.2021.639078

来源: DOAJ

【 摘 要 】

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.

【 授权许可】

Unknown