Paganoni S, Macklin EA, Hendrix S, Berry JD, Elliott MA, Maiser S, Karam C, Caress JB, Owegi MA, Quick A, Wymer J, Goutman SA, Heitzman D, Heiman-Patterson T, Jackson CE, Quinn C, Rothstein JD, Kasarskis EJ, Katz J, Jenkins L, Ladha S, Miller TM, Scelsa SN, Vu TH, Fournier CN, Glass JD, Johnson KM, Swenson A, Goyal NA, Pattee GL, Andres PL, Babu S, Chase M, Dagostino D, Dickson SP, Ellison N, Hall M, Hendrix K, Kittle G, McGovern M, Ostrow J, Pothier L, Randall R, Shefner JM, Sherman AV, Tustison E, Vigneswaran P, Walker J, Yu H, Chan J, Wittes J, Cohen J, Klee J, Leslie K, Tanzi RE, Gilbert W, Yeramian PD, Schoenfeld D, Cudkowicz ME. Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis. N Engl J Med. 2020 Sep 3;383(10):919-930. PubMed.
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This study showed a significant impact of sodium phenylbutyrate/taurursodiol on ALS, based on slowing of the rate of decline in subjects versus controls as measured by the ALSFRS-R, a primary assessment tool for measuring functional change.
Overall, there was a reduction over 24 weeks of the rate of decline using this metric that was comparable to the effects seen with edaravone, recently approved by the FDA for ALS. While this was a relatively large study (given the patient population), as the authors suggest, an additional larger longitudinal study could be of value to corroborate the effect of the medication on slowing disease progression and more accurately determine any impact on survival.
To date, only riluzole has shown an impact on survival, albeit modest. Joshua Cohen and Justin Klee, the founders of Amylyx, are to be commended for their extraordinary focus, determination, and leadership in studying the neuroprotective properties of sodium phenylbutyrate/taurursodiol and then creating their company, while still undergraduates, to allow this compound to be studied successfully in patients with ALS.View all comments by Michael Weiss
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