Hopes and concerns regarding the implementation of expanded access protocols in amyotrophic lateral sclerosis

Abstract

Hopes and concerns regarding the implementation of expanded access protocols in amyotrophic lateral sclerosis Amyotrophic lateral sclerosis (ALS) is a devastating and fatal neurodegenerative disease. The three United States Food and Drug Administration (FDA) approved therapies, although shown to slow disease progression or prolong survival or both, do not halt progression or restore strength. [1][2][3] Patients in such situations often seek treatment options beyond FDA-approved drugs. In this issue of Muscle and Nerve, Yerton et al report the experience of launching and maintaining multiple expanded access protocols (EAPs) in ALS since 2018 from a single tertiary ALS referral center in the United States, highlighting both the feasibility and challenges of developing and maintaining such programs.