Acetylleucine is a modified
amino acid
Amino acids are organic compounds that contain both amino and carboxylic acid functional groups. Although hundreds of amino acids exist in nature, by far the most important are the alpha-amino acids, which comprise proteins. Only 22 alpha a ...
used in the treatment of
vertigo
Vertigo is a condition where a person has the sensation of movement or of surrounding objects moving when they are not. Often it feels like a spinning or swaying movement. This may be associated with nausea, vomiting, sweating, or difficulties w ...
and
cerebellar ataxia
Cerebellar ataxia is a form of ataxia originating in the cerebellum. Non-progressive congenital ataxia (NPCA) is a classical presentation of cerebral ataxias.
Cerebellar ataxia can occur as a result of many diseases and may present with symptom ...
.
Acetylleucine is also being
developed as a possible treatment for several neurological disorders by IntraBio Inc. Clinical trials with acetylleucine for the treatment of three orphan, fatal, neurodegenerative disorders are underway:
Niemann-Pick disease type C,
GM2 gangliosidoses
The GM2 gangliosidoses are a group of three related genetic disorders that result from a deficiency of the enzyme beta-hexosaminidase. This enzyme catalyzes the biodegradation of fatty acid derivatives known as gangliosides. The diseases are bett ...
(
Tay-Sachs and
Sandhoff diseases), and
ataxia–telangiectasia
Ataxia–telangiectasia (AT or A–T), also referred to as ataxia–telangiectasia syndrome or Louis–Bar syndrome, is a rare, neurodegenerative, autosomal recessive disease causing severe disability. Ataxia refers to poor coordination and tela ...
. In 2020, IntraBio announced the successful multinational clinical trial results of the Niemann-Pick type C clinical trial. IntraBio is also investigating acetylleucine for the treatment of common inherited and acquired neurological diseases including
Lewy body dementia
Lewy body dementias are two similar and common subtypes of dementia—dementia with Lewy bodies (DLB) and
Parkinson's disease dementia (PDD). Both are characterized by changes in thinking, movement, behavior, and mood. The two conditions have si ...
,
amyotrophic lateral sclerosis,
restless legs syndrome
Restless legs syndrome (RLS), also known as Willis-Ekbom disease (WED), is generally a long-term disorder that causes a strong urge to move one's legs. There is often an unpleasant feeling in the legs that improves somewhat by moving them. This ...
,
multiple sclerosis, and
migraine Acetylleucine has received
orphan drug designations from the
U.S. Food & Drug Administration (FDA) and the European Commission.
See also
*
Leucine
Leucine (symbol Leu or L) is an essential amino acid that is used in the biosynthesis of proteins. Leucine is an α-amino acid, meaning it contains an α- amino group (which is in the protonated −NH3+ form under biological conditions), an α- ...
References
{{antivertigo preparations
Amino acid derivatives
Acetamides