Preferred Label : Leriglitazone Hydrochloride;
NCIt definition : The hydrochloride salt form of leriglitazone, an orally bioavailable, blood-brain-barrier
(BBB) penetrable, selective peroxisome proliferator-activated receptor (PPAR) subtype
gamma agonist, with potential neuroprotective activity that could be used for certain
central nervous system (CNS) diseases, such as adrenomyeloneuropathy, cerebral adrenoleukodystrophy
(cALD), Friedreich's ataxia, and certain other CNS diseases. Upon oral administration,
leriglitazone selectively targets, binds to and activates PPARgamma, thereby regulating
the expression of genes involved in mitochondrial biogenesis. This modulates pathways
leading to the restoration of mitochondrial function in which dysfunction is caused
by the accumulation of very long-chain fatty acids (VLCFAs), and increases energy
production, decreases oxidative stress, decreases nuclear factor kappa B (NF-kB) levels,
inhibits neuroinflammation, protects the BBB integrity, prevents demyelination and
axonal degeneration, increases neuronal survival, increases myelination and oligodendrocyte
survival and improves motor function. Mutations in the ABCD1 gene, which encodes the
peroxisomal membrane adrenoleukodystrophy protein, cause a defective function of the
ABCD1 transporter leading to an accumulation of VLCFA. VLCFA accumulation contributes
to membrane destabilization of the myelin sheath, mitochondrial dysfunction, oxidative
stress, neuroinflammation and compromised BBB integrity.;
UNII : WN9K42SAM6;
InChIKey : SBHOQYCDAHAMDW-UHFFFAOYSA-N;
CAS number : 146062-46-6;
NCI Metathesaurus CUI : CL1412427;
Origin ID : C174935;
UMLS CUI : C5421031;
Semantic type(s)
- concept_is_in_subset
- has_free_acid_or_base_form
- is_salt_form_of
