ALSUntangled 59: Tamoxifen

Richard Bedlack; Sky Kihuwa-Mani; Paul E Barkhaus; Michael Bereman; James B Caress; Jesse Crayle; Gary L Pattee; Terry Heiman Patterson; Paul Wicks; Neta Zach; Gregory T Carter; Kristiana Salmon

doi:10.1080/21678421.2021.1876731

ALSUntangled 59: Tamoxifen

Amyotroph Lateral Scler Frontotemporal Degener. 2021 Nov;22(7-8):595-598. doi: 10.1080/21678421.2021.1876731. Epub 2021 Jan 21.

Authors

Affiliations

¹ Department of Neurology, Duke University, Durham, NC, USA.
² Lloyd International Honors College, University of North Carolina at Greensboro, Greensboro, NC, USA.
³ Department of Neurology, Medical College of Wisconsin, Milwaukee, WI, USA.
⁴ Department of Biological Sciences, North Carolina State University, Raleigh, NC, USA.
⁵ Department of Neurology, Wake Forest Baptist Health, Winston-Salem, NC, USA.
⁶ Department of Neurology, University of Nebraska Medical Center, Omaha, NB, USA.
⁷ Department of Neurology, Lewis Katz School of Medicine at Temple University, Philadelphia, PA, USA.
⁸ Independent consultant, United Kingdom of Great Britain and Northern Ireland.
⁹ Department of Neuroscience, Takeda Pharmaceuticals USA Inc, Deerfield, IL, USA.
¹⁰ Department of Neurology, Saint Luke's-Elks Rehab, Boise, ID, USA.
¹¹ Department of Neurology, McGill University Health Centre, Montreal, Canada.

PMID: 33474997
DOI: 10.1080/21678421.2021.1876731

Abstract

Here we use the ALSUntangled methodology to review Tamoxifen as an ALS treatment. We show that it has plausible mechanisms, a positive preclinical study, a case report and 2 small trials suggesting benefits. We show that it appears reasonably safe, though there is a small risk of developing cancer with long term use. While we cannot yet endorse this as an ALS treatment, there is enough evidence to warrant another larger ALS trial.

Keywords: ALSUntangled; Tamoxifen; clinical trials; ethics; off-label treatment; therapy; trials.

Publication types

Case Reports
Research Support, Non-U.S. Gov't

MeSH terms

Amyotrophic Lateral Sclerosis* / drug therapy
Humans
Tamoxifen* / therapeutic use

Substances

Tamoxifen