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Details

Stereochemistry ABSOLUTE
Molecular Formula C12H22O11.2H2O
Molecular Weight 378.327
Optical Activity UNSPECIFIED
Defined Stereocenters 10 / 10
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of TREHALOSE DIHYDRATE

SMILES

O.O.[H][C@]2(O[C@H]1O[C@H](CO)[C@@H](O)[C@H](O)[C@H]1O)O[C@H](CO)[C@@H](O)[C@H](O)[C@H]2O

InChI

InChIKey=DPVHGFAJLZWDOC-PVXXTIHASA-N
InChI=1S/C12H22O11.2H2O/c13-1-3-5(15)7(17)9(19)11(21-3)23-12-10(20)8(18)6(16)4(2-14)22-12;;/h3-20H,1-2H2;2*1H2/t3-,4-,5-,6-,7+,8+,9-,10-,11-,12-;;/m1../s1

HIDE SMILES / InChI

Description

Trehalose, a naturally occurring disaccharide of glucose that appears to function in an anhydrobiotic capacity in many organisms. Bioblast Pharma study trehalose in Phase 2 for treating patients with Oculopharyngeal Muscular Dystrophy (OPMD) and spinocerebellar ataxia, type 3. In OPMD trehalose prevents the aggregation of the pathological protein (PABPN1) in muscle cells, the hallmark of the disease, by stabilizing the protein, reducing the formation of protein aggregations, and promoting their clearance from cells through autophagy, thus preventing muscle cell death. Trehalose induces autophagy via mTOR independent pathway. It activates TFEB, a master controller of lysosomal biogenesis and autophagy, by inhibiting AKT which is a negative regulator of TFEB that acts by direct phosphorylation (and inhibition) of TFEB. In addition, trehalose protects cells from hypoxic and anoxic injury and suppresses protein aggregation. In vivo studies with trehalose show cellular and behavioral beneficial effects in animal models of neurodegenerative diseases. Trehalose was in phase III clinical trial to study if it was possible to use the drug as add-on therapy in Bipolar Depression. Also in combination with hyaluronate, it can be used to treat dry eye syndrome. Trehalose protects the epithelial cells on the ocular surface, improving their resistance to the daily stresses of dry environments and tear film changes in a dry eye.

CNS Activity

Originator

Approval Year

Targets

Primary TargetPharmacologyConditionPotency

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Unknown
Primary
Unknown
Palliative
Unknown
Primary
Thealoz Duo

PubMed

Sample Use Guides

In Vivo Use Guide
Oculopharyngeal Muscular Dystrophy and spinocerebellar ataxia, type 3: trehalose 90 mg/mL IV solution. Bipolar Depression: trehalose 70 g/die dry eye syndrome: as one drop five times daily in both eyes for 7 days followed by a washout period of 5 days, whereupon patients switched to the alternate treatment for 5 days.
Route of Administration: Other
In Vitro Use Guide
It was found that trehalose didn’t affect the functions of human neutrophils in vitro. Human polymorphonuclear neutrophils (PMNs) were obtained from 17 healthy volunteers. Escherichia coli and Staphylococcus aureus were used for the bacterial infection model, whereas lipopolysaccharide (LPS) and interleukin (IL)-1β were used for inflammation induction model. The PMN phagocytosis rates of bacteria and apoptosis/necrosis were assessed on trehalose, maltose, and control media. There were no significant differences in the phagocytosis rates, apoptosis/necrosis rates, or levels of all cytokines or PMN-elastase among the three media in the bacterial infection model. There were also no significant differences in the levels of all cytokines and PMN-elastase among the three media in the IL-1β inflammation induction model. PMN-elastase was lower in trehalose and maltose medium after LPS stimulation, at 3 and 24 h.