TREHALOSE

Possibly Marketed Outside US

Details

Stereochemistry	ABSOLUTE
Molecular Formula	C12H22O11
Molecular Weight	342.2965
Optical Activity	UNSPECIFIED
Defined Stereocenters	10 / 10
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

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

InChI

InChIKey=HDTRYLNUVZCQOY-LIZSDCNHSA-N

InChI=1S/C12H22O11/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/t3-,4-,5-,6-,7+,8+,9-,10-,11-,12-/m1/s1

HIDE SMILES / InChI

Molecular Formula	C12H22O11
Molecular Weight	342.2965
Charge	0
Count

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	10 / 10
E/Z Centers	0
Optical Activity	UNSPECIFIED

Description
Sources: https://bioblastpharma.com/pipeline/iv-trehalose | https://www.ncbi.nlm.nih.gov/pubmed/28165011 | https://clinicaltrials.gov/ct2/show/study/NCT02800161

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 Target	Pharmacology	Condition	Potency
Serine/threonine-protein kinase AKT 31 Target ID: CHEMBL2111353 Sources: https://www.ncbi.nlm.nih.gov/pubmed/28165011	Inhibitor 8504

Conditions

Condition	Modality	Highest Phase	Product
Oculopharyngeal muscular dystrophy 2 Sources: https://bioblastpharma.com/pipeline	Primary	Phase II 1147	Unknown Approved Use Unknown
Spinocerebellar ataxia, type 3 2 Sources: https://bioblastpharma.com/pipeline/iv-trehalose	Primary	Phase II 1147	Unknown Approved Use Unknown
Bipolar depression 9 Sources: https://clinicaltrials.gov/ct2/show/study/NCT02800161	Palliative	Phase III 665	Unknown Approved Use Unknown
Dry eye syndrome 21 Sources: http://www.thea-pharmaceuticals.co.uk/products/thealoz-duo	Primary	Not Provided 511	Thealoz Duo Approved Use Unknown

C_max

Value	Dose	Analyte	Population
8900 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg single, oral dose: 2.67 g/kg route of administration: Oral experiment type: SINGLE co-administered:	TREHALOSE plasma	Rattus norvegicus population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
8336 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg 1 times / day multiple, oral dose: 2.67 g/kg route of administration: Oral experiment type: MULTIPLE co-administered:	TREHALOSE plasma	Rattus norvegicus population: HEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN
10918 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg single, oral dose: 2.67 g/kg route of administration: Oral experiment type: SINGLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
9578 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg 1 times / day multiple, oral dose: 2.67 g/kg route of administration: Oral experiment type: MULTIPLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
36962 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	5.34 g/kg single, oral dose: 5.34 g/kg route of administration: Oral experiment type: SINGLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
22444 ng/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	5.34 g/kg 1 times / day multiple, oral dose: 5.34 g/kg route of administration: Oral experiment type: MULTIPLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN

AUC

Value	Dose	Analyte	Population
15599 mg × h/L EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	20 mg/kg single, intramuscular dose: 20 mg/kg route of administration: Intramuscular experiment type: SINGLE co-administered:	TREHALOSE plasma	rattus population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
11136 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg single, oral dose: 2.67 g/kg route of administration: Oral experiment type: SINGLE co-administered:	TREHALOSE plasma	Rattus norvegicus population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
9876 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg 1 times / day multiple, oral dose: 2.67 g/kg route of administration: Oral experiment type: MULTIPLE co-administered:	TREHALOSE plasma	Rattus norvegicus population: HEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN
27445 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg single, oral dose: 2.67 g/kg route of administration: Oral experiment type: SINGLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
27363 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg 1 times / day multiple, oral dose: 2.67 g/kg route of administration: Oral experiment type: MULTIPLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
80040 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	5.34 g/kg single, oral dose: 5.34 g/kg route of administration: Oral experiment type: SINGLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
57172 ng × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	5.34 g/kg 1 times / day multiple, oral dose: 5.34 g/kg route of administration: Oral experiment type: MULTIPLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN

T_1/2

Value	Dose	Analyte	Population
0.73 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	20 mg/kg single, intramuscular dose: 20 mg/kg route of administration: Intramuscular experiment type: SINGLE co-administered:	TREHALOSE plasma	rattus population: HEALTHY age: ADULT sex: MALE food status: UNKNOWN
0.76 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg single, oral dose: 2.67 g/kg route of administration: Oral experiment type: SINGLE co-administered:	TREHALOSE plasma	Rattus norvegicus population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
0.89 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg 1 times / day multiple, oral dose: 2.67 g/kg route of administration: Oral experiment type: MULTIPLE co-administered:	TREHALOSE plasma	Rattus norvegicus population: HEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN
1.1 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg single, oral dose: 2.67 g/kg route of administration: Oral experiment type: SINGLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
1.5 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	2.67 g/kg 1 times / day multiple, oral dose: 2.67 g/kg route of administration: Oral experiment type: MULTIPLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
1.8 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	5.34 g/kg single, oral dose: 5.34 g/kg route of administration: Oral experiment type: SINGLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN
2.3 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30063249/	5.34 g/kg 1 times / day multiple, oral dose: 5.34 g/kg route of administration: Oral experiment type: MULTIPLE co-administered:	TREHALOSE plasma	Macaca fascicularis population: HEALTHY age: ADULT sex: FEMALE food status: UNKNOWN

Doses

Dose	Population	Adverse events
100 g 1 times / day multiple, oral Highest studied dose Dose: 100 g, 1 times / day Route: oral Route: multiple Dose: 100 g, 1 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/27208415/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/27208415/	Other AEs: Bloating, Flatulence... Other AEs: Bloating (grade 1-2) Flatulence (grade 1-2) Loose stools (grade 1-2) Sources: https://pubmed.ncbi.nlm.nih.gov/27208415/
68.5 g 6 times / day multiple, topical Studied dose Dose: 68.5 g, 6 times / day Route: topical Route: multiple Dose: 68.5 g, 6 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/12414409/ https://pubmed.ncbi.nlm.nih.gov/15295655/	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/12414409/ https://pubmed.ncbi.nlm.nih.gov/15295655/	Disc. AE: Conjunctivitis... AEs leading to discontinuation/dose reduction: Conjunctivitis (20%) Sources: https://pubmed.ncbi.nlm.nih.gov/12414409/ https://pubmed.ncbi.nlm.nih.gov/15295655/

AEs

AE	Significance	Dose	Population
Bloating	grade 1-2	100 g 1 times / day multiple, oral Highest studied dose Dose: 100 g, 1 times / day Route: oral Route: multiple Dose: 100 g, 1 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/27208415/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/27208415/
Flatulence	grade 1-2	100 g 1 times / day multiple, oral Highest studied dose Dose: 100 g, 1 times / day Route: oral Route: multiple Dose: 100 g, 1 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/27208415/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/27208415/
Loose stools	grade 1-2	100 g 1 times / day multiple, oral Highest studied dose Dose: 100 g, 1 times / day Route: oral Route: multiple Dose: 100 g, 1 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/27208415/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/27208415/
Conjunctivitis	20% Disc. AE	68.5 g 6 times / day multiple, topical Studied dose Dose: 68.5 g, 6 times / day Route: topical Route: multiple Dose: 68.5 g, 6 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/12414409/ https://pubmed.ncbi.nlm.nih.gov/15295655/	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M+F Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/12414409/ https://pubmed.ncbi.nlm.nih.gov/15295655/

PubMed

Title	Date	PubMed
Rapana thomasiana hemocyanin (RtH): dissociation and reassociation behavior of two isoforms, RtH1 and RtH2.	2002	11473809
Trehalose-6-phosphate phosphorylase is part of a novel metabolic pathway for trehalose utilization in Lactococcus lactis.	2001-11-16	11553642
Chemical chaperones regulate molecular chaperones in vitro and in cells under combined salt and heat stresses.	2001-10-26	11517217
Towards more active biocatalysts in organic media: increasing the activity of salt-activated enzymes.	2001-10-20	11536141
Phosphoglucomutase is an in vivo lithium target in yeast.	2001-10-12	11500487
Urea increases tolerance of yeast inorganic pyrophosphatase activity to ethanol: the other side of urea interaction with proteins.	2001-10-01	11566028
Trehalose sensitivity in Drosophila correlates with mutations in and expression of the gustatory receptor gene Gr5a.	2001-09-18	11566105
Trehalose metabolism in Arabidopsis: occurrence of trehalose and molecular cloning and characterization of trehalose-6-phosphate synthase homologues.	2001-09	11520870
Mechanism of pressure-induced thermostabilization of proteins: studies of glutamate dehydrogenases from the hyperthermophile Thermococcus litoralis.	2001-09	11514665
Colorimetric determination of active alpha-glucoside transport in Saccharomyces cerevisiae.	2001-09	11438190
Infrared spectroscopic study on the properties of the anhydrous form II of trehalose. Implications for the functional mechanism of trehalose as a biostabilizer.	2001-08-30	11513830
Reversible dehydration of trehalose and anhydrobiosis: from solution state to an exotic crystal?	2001-08-30	11513823
Of tardigrades, trehalose, and tissue engineering.	2001-08-04	11502330
Alterations in the levels of glycogen and glycogen synthase transcripts during desiccation in the insect-killing nematode Steinernema feltiae IS-6.	2001-08	11534633
Haemolymph sugar levels in foraging honeybees (Apis mellifera carnica): dependence on metabolic rate and in vivo measurement of maximal rates of trehalose synthesis.	2001-08	11533121
Prevention of structural perturbations and aggregation upon encapsulation of bovine serum albumin into poly(lactide-co-glycolide) micropheres using the solid-in-oil-in water technique.	2001-08	11518019
Pseudoalteromonas ulvae sp. nov., a bacterium with antifouling activities isolated from the surface of a marine alga.	2001-07	11491351
Purification and characterization of the heterologously expressed trehalose/maltose ABC transporter complex of the hyperthermophilic archaeon Thermococcus litoralis.	2001-07	11453995
Osmoregulation in Onymacris rugatipennis, a free-ranging tenebrionid beetle from the Namib Desert.	2001-07	11440873
Trehalose-hydroxyethylcellulose microspheres containing vancomycin for topical drug delivery.	2001-07	11438427
Trehalose is required for the acquisition of tolerance to a variety of stresses in the filamentous fungus Aspergillus nidulans.	2001-07	11429462
Cryptococcomas distinguished from gliomas with MR spectroscopy: an experimental rat and cell culture study.	2001-07	11425983
Some observations in freeze-drying of recombinant bioluminescent Escherichia coli for toxicity monitoring.	2001-06-15	11403844
Fungal trehalose phosphorylase: kinetic mechanism, pH-dependence of the reaction and some structural properties of the enzyme from Schizophyllum commune.	2001-06-15	11389683
Comparison of the decameric structure of peroxiredoxin-II by transmission electron microscopy and X-ray crystallography.	2001-06-11	11410278
Global gene expression during short-term ethanol stress in Saccharomyces cerevisiae.	2001-06-01	11389906
Rigid multilamellar bilayer cooperativity is modified by non covalently linked neuraminic-5-acid: a spectrophotometric determination.	2001-06	11519684
New cyclodextrin derivatives as chiral selectors in capillary electrophoresis.	2001-06	11495056
Trehalose-producing operon treYZ from Arthrobacter ramosus S34.	2001-06	11471747
Innovative fermentation strategies for the production of extremophilic enzymes.	2001-06	11453463
Roles of trehalose phosphate synthase in yeast glycogen metabolism and sporulation.	2001-06	11442833
Granulomatous tissue formation of shikon and shikonin by air pouch method.	2001-06	11411553
Cloning, expression, and characterization of the hxk-1 Gene from the white truffle Tuber borchii vittad.: A first step toward understanding sugar metabolism.	2001-06	11407882
Induction of ApL3 expression by trehalose complements the starch-deficient Arabidopsis mutant adg2-1 lacking ApL1, the large subunit of ADP-glucose pyrophosphorylase.	2001-06	11402215
The osmotic stress response and virulence in pyelonephritis isolates of Escherichia coli: contributions of RpoS, ProP, ProU and other systems.	2001-06	11390697
Genetic localization and regulation of the maltose phosphorylase gene, malP, in Lactococcus lactis.	2001-06	11390687
Separating structure and dynamics in CSA/DD cross-correlated relaxation: a case study on trehalose and ubiquitin.	2001-06	11384172
Miniaturized electrospraying as a technique for the production of microarrays of reproducible micrometer-sized protein spots.	2001-05-15	11393839
Development of poly-(D,L-lactide--coglycolide) microsphere formulations containing recombinant human vascular endothelial growth factor to promote local angiogenesis.	2001-05-14	11389981
[The metabolism of trehalose and intracellular glycerol in Candida krusei responding to high osmosis].	2001-05	11517613
Trehalose becomes the most abundant non-structural carbohydrate during senescence of soybean nodules.	2001-05	11432911
Burkholderia fungorum sp. nov. and Burkholderia caledonica sp. nov., two new species isolated from the environment, animals and human clinical samples.	2001-05	11411678
Multiple effects of protein phosphatase 2A on nutrient-induced signalling in the yeast Saccharomyces cerevisiae.	2001-05	11401708
Long-circulating monensin nanoparticles for the potentiation of immunotoxin and anticancer drugs.	2001-05	11370701
Effect of sucrose/raffinose mass ratios on the stability of co-lyophilized protein during storage above the Tg.	2001-04	11451034
Molecular characteristics and physiology of microsporidia.	2001-04	11369278
Human platelets loaded with trehalose survive freeze-drying.	2001-03	11448110
Gemini surfactants with a disaccharide spacer.	2001-02-07	11456621
Mycolic acids from Mycobacterium tuberculosis: purification by countercurrent distribution and T-cell stimulation.	2001	11491525
Acquisition of tolerance against oxidative damage in Saccharomyces cerevisiae.	2001	11483159

Patents

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.

Substance Class	Chemical Created by `admin` on `Mon Mar 31 18:16:32 GMT 2025` Edited by `admin` on `Mon Mar 31 18:16:32 GMT 2025`
Record UNII	B8WCK70T7I
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

CABALETTA

Preferred Name

English

TREHALOSE

Official Name

English

MYCOSE

Common Name

English

MUSHROOM SUGAR

Common Name

English

NSC-2093

Code

English

.ALPHA.-D-GLUCOPYRANOSYL-.ALPHA.-D-GLUCOPYRANOSIDE

Common Name

English

TREHALOSE [USP-RS]

Common Name

English

.ALPHA.,.ALPHA.-TREHALOSE

Common Name

English

TREHALOSE [FCC]

Common Name

English

Trehalose [WHO-DD]

Common Name

English

TREHALOSE [MI]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

781920