Acetylleucine Explained

Acetylleucine (N-acetyl-leucine) is a modified leucine amino acid used in the treatment of vertigo[1] and cerebellar ataxia.

Two forms exist: acetyl-DL-leucine (sold under the brand Tanganil, among others) and N-acetyl-L-leucine (levacetylleucine).[2]

Acetylleucine is also being developed as a possible treatment for several neurological disorders by IntraBio Inc.[3] Clinical trials with acetylleucine for the treatment of three orphan, fatal, neurodegenerative disorders are underway: Niemann-Pick disease type C,[4] GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases),[5] and ataxia–telangiectasia.[6] In 2020, IntraBio announced the successful multinational clinical trial results of the Niemann-Pick type C clinical trial.[7] IntraBio is also investigating acetylleucine for the treatment of common inherited and acquired neurological diseases including Lewy body dementia,[8] amyotrophic lateral sclerosis, restless legs syndrome, multiple sclerosis, and migraine[9] Acetylleucine has received orphan drug designations from the U.S. Food & Drug Administration (FDA)[10] [11] [12] [13] and the European Commission.[14] [15] [16] [17]

Notes and References

  1. Web site: N07CA04 (acetylleucine) . WHO Collaborating Centre for Drug Statistics Methodology . Norwegian Institute of Public Health . 19 Dec 2016 . 26 Mar 2017.
  2. Oertel . Wolfgang H. . Janzen . Annette . Henrich . Martin T. . Geibl . Fanni F. . Sittig . Elisabeth . Meles . Sanne K. . Carli . Giulia . Leenders . Klaus . Booij . Jan . Surmeier . D. James . Timmermann . Lars . Strupp . Michael . 2024-09-02 . Acetyl-DL-leucine in two individuals with REM sleep behavior disorder improves symptoms, reverses loss of striatal dopamine-transporter binding and stabilizes pathological metabolic brain pattern—case reports . Nature Communications . 15 . 1 . 7619 . 10.1038/s41467-024-51502-7 . 39223119 . 11369233 . 2041-1723.
  3. Web site: IntraBio. 2019-08-01. https://web.archive.org/web/20190801041947/http://intrabio.com/for-medical-professionals/. 2019-08-01. dead.
  4. Web site: N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) - Full Text View - ClinicalTrials.gov. clinicaltrials.gov. 2019-08-01.
  5. Web site: N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease) - Full Text View - ClinicalTrials.gov. clinicaltrials.gov. 2019-08-01.
  6. Web site: N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T) - Full Text View - ClinicalTrials.gov. clinicaltrials.gov. 2019-08-01.
  7. Web site: IntraBio Reports Further Detail on Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C. intrabio.com. 19 October 2020 . 2021-08-01.
  8. A clinical trial to test amlodipine as a new treatment for vascular dementia. Passmore. Peter. 2014-04-15. 10.1186/isrctn31208535 . free .
  9. Strupp. Michael. Bayer. Otmar. Feil. Katharina. Straube. Andreas. 2019-02-01. Prophylactic treatment of migraine with and without aura with acetyl-dl-leucine: a case series. Journal of Neurology. 266. 2. 525–529. 10.1007/s00415-018-9155-6. 30547273. 56148131. 1432-1459.
  10. Web site: Search Orphan Drug Designations and Approvals. www.accessdata.fda.gov. 2019-08-03.
  11. Web site: Search Orphan Drug Designations and Approvals. www.accessdata.fda.gov. 2019-08-03.
  12. Web site: Search Orphan Drug Designations and Approvals. www.accessdata.fda.gov. 2019-08-03.
  13. Web site: Search Orphan Drug Designations and Approvals. www.accessdata.fda.gov. 2019-08-03.
  14. Web site: Public Health - European Commission. Union Register of medicinal products. 2019-08-03.
  15. Web site: Public Health - European Commission. Union Register of medicinal products. 2019-08-03.
  16. Web site: Public Health - European Commission. Union Register of medicinal products. 2019-08-03.
  17. Web site: Public Health - European Commission. Union Register of medicinal products. 2019-08-03.

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