Details
Stereochemistry | ACHIRAL |
Molecular Formula | C2H7NS |
Molecular Weight | 77.149 |
Optical Activity | NONE |
Defined Stereocenters | 0 / 0 |
E/Z Centers | 0 |
Charge | 0 |
SHOW SMILES / InChI
SMILES
NCCS
InChI
InChIKey=UFULAYFCSOUIOV-UHFFFAOYSA-N
InChI=1S/C2H7NS/c3-1-2-4/h4H,1-3H2
Molecular Formula | C2H7NS |
Molecular Weight | 77.149 |
Charge | 0 |
Count |
|
Stereochemistry | ACHIRAL |
Additional Stereochemistry | No |
Defined Stereocenters | 0 / 0 |
E/Z Centers | 0 |
Optical Activity | NONE |
DescriptionCurator's Comment: description was created based on several sources, including
https://www.ncbi.nlm.nih.gov/pubmed/6131501 | https://www.ncbi.nlm.nih.gov/pubmed/6124307 | https://www.ncbi.nlm.nih.gov/pubmed/15675041 | https://www.ncbi.nlm.nih.gov/pubmed/28384851
Curator's Comment: description was created based on several sources, including
https://www.ncbi.nlm.nih.gov/pubmed/6131501 | https://www.ncbi.nlm.nih.gov/pubmed/6124307 | https://www.ncbi.nlm.nih.gov/pubmed/15675041 | https://www.ncbi.nlm.nih.gov/pubmed/28384851
Cysteamine (trade name CYSTAGON) is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis. Cystinosis is an autosomal recessive inborn error of metabolism in which the transport of cystine out of lysosomes is abnormal; in the nephropathic form, accumulation of cystine and formation of crystals damage various organs, especially the kidney, leading to renal tubular Fanconi Syndrome and progressive glomerular failure, with end-stage renal failure by the end of the first decade of life. In four studies of cystinosis patients before cysteamine was available, renal death (need for transplant or dialysis) occurred at the median age of fewer than 10 years. Patients with cystinosis also experience growth failure, rickets, and photophobia due to cystine deposits in the cornea. With time most organs are damaged, including the retina, muscles and central nervous system. Cysteamine is an aminothiol that participates within lysosomes in a thiol-disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients with cystinosis.
CNS Activity
Originator
Approval Year
Targets
Primary Target | Pharmacology | Condition | Potency |
---|---|---|---|
Target ID: P21980 Gene ID: 7052.0 Gene Symbol: TGM2 Target Organism: Homo sapiens (Human) Sources: https://www.ncbi.nlm.nih.gov/pubmed/15675041 |
178.0 µM [IC50] |
Conditions
Condition | Modality | Targets | Highest Phase | Product |
---|---|---|---|---|
Primary | CYSTAGON Approved UsePROCYSBI is indicated for the treatment of nephropathic cystinosis in adult and pediatric patients 2 years of age and older. PROCYSBI is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adult and pediatric patients 2 years of age and older. (1) Launch Date7.7690883E11 |
Cmax
Value | Dose | Co-administered | Analyte | Population |
---|---|---|---|---|
2.7 mg/L |
404 mg/m² 4 times / day steady-state, oral dose: 404 mg/m² route of administration: Oral experiment type: STEADY-STATE co-administered: |
CYSTEAMINE plasma | Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: UNKNOWN |
|
37.72 μM |
450 mg single, oral dose: 450 mg route of administration: Oral experiment type: SINGLE co-administered: |
CYSTEAMINE plasma | Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: UNKNOWN |
AUC
Value | Dose | Co-administered | Analyte | Population |
---|---|---|---|---|
351 mg × min/L |
404 mg/m² 4 times / day steady-state, oral dose: 404 mg/m² route of administration: Oral experiment type: STEADY-STATE co-administered: |
CYSTEAMINE plasma | Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: UNKNOWN |
|
96 μM × h |
450 mg single, oral dose: 450 mg route of administration: Oral experiment type: SINGLE co-administered: |
CYSTEAMINE plasma | Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: UNKNOWN |
T1/2
Value | Dose | Co-administered | Analyte | Population |
---|---|---|---|---|
90 min |
404 mg/m² 4 times / day steady-state, oral dose: 404 mg/m² route of administration: Oral experiment type: STEADY-STATE co-administered: |
CYSTEAMINE plasma | Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: UNKNOWN |
Funbound
Value | Dose | Co-administered | Analyte | Population |
---|---|---|---|---|
48% |
404 mg/m² 4 times / day steady-state, oral dose: 404 mg/m² route of administration: Oral experiment type: STEADY-STATE co-administered: |
CYSTEAMINE plasma | Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: UNKNOWN |
|
48% |
450 mg single, oral dose: 450 mg route of administration: Oral experiment type: SINGLE co-administered: |
CYSTEAMINE plasma | Homo sapiens population: UNHEALTHY age: CHILD sex: FEMALE / MALE food status: UNKNOWN |
Doses
Dose | Population | Adverse events |
---|---|---|
0.44 % 1 times / day multiple, ophthalmic Recommended Dose: 0.44 %, 1 times / day Route: ophthalmic Route: multiple Dose: 0.44 %, 1 times / day Sources: Page: 98 EI0109S |
unhealthy, 13.4 (5.9-27.8) n = 20 Health Status: unhealthy Condition: cystinosis Age Group: 13.4 (5.9-27.8) Sex: M+F Population Size: 20 Sources: Page: 98 EI0109S |
Disc. AE: Intracranial hypertension... AEs leading to discontinuation/dose reduction: Intracranial hypertension Sources: Page: 98 EI0109S |
600 mg 1 times / day multiple, oral Recommended Dose: 600 mg, 1 times / day Route: oral Route: multiple Dose: 600 mg, 1 times / day Sources: Page: RP103-05 |
healthy, 37.5 (19-64) n = 20 Health Status: healthy Age Group: 37.5 (19-64) Sex: M+F Population Size: 20 Sources: Page: RP103-05 |
Disc. AE: Blurred vision, Hematuria... AEs leading to discontinuation/dose reduction: Blurred vision Sources: Page: RP103-05Hematuria |
AEs
AE | Significance | Dose | Population |
---|---|---|---|
Intracranial hypertension | Disc. AE | 0.44 % 1 times / day multiple, ophthalmic Recommended Dose: 0.44 %, 1 times / day Route: ophthalmic Route: multiple Dose: 0.44 %, 1 times / day Sources: Page: 98 EI0109S |
unhealthy, 13.4 (5.9-27.8) n = 20 Health Status: unhealthy Condition: cystinosis Age Group: 13.4 (5.9-27.8) Sex: M+F Population Size: 20 Sources: Page: 98 EI0109S |
Blurred vision | Disc. AE | 600 mg 1 times / day multiple, oral Recommended Dose: 600 mg, 1 times / day Route: oral Route: multiple Dose: 600 mg, 1 times / day Sources: Page: RP103-05 |
healthy, 37.5 (19-64) n = 20 Health Status: healthy Age Group: 37.5 (19-64) Sex: M+F Population Size: 20 Sources: Page: RP103-05 |
Hematuria | Disc. AE | 600 mg 1 times / day multiple, oral Recommended Dose: 600 mg, 1 times / day Route: oral Route: multiple Dose: 600 mg, 1 times / day Sources: Page: RP103-05 |
healthy, 37.5 (19-64) n = 20 Health Status: healthy Age Group: 37.5 (19-64) Sex: M+F Population Size: 20 Sources: Page: RP103-05 |
Overview
CYP3A4 | CYP2C9 | CYP2D6 | hERG |
---|---|---|---|
OverviewOther
Other Inhibitor | Other Substrate | Other Inducer |
---|---|---|
Drug as perpetrator
Drug as victim
PubMed
Title | Date | PubMed |
---|---|---|
Role of heme oxygenase-1 in the regulation of manganese superoxide dismutase gene expression in oxidatively-challenged astroglia. | 2000 Oct |
|
Pantetheinase activity of membrane-bound Vanin-1: lack of free cysteamine in tissues of Vanin-1 deficient mice. | 2000 Oct 20 |
|
Oxidative stress, glucose-6-phosphate dehydrogenase and the red cell. | 2001 |
|
Structure-activity analysis of the potentiation by aminothiols of the chromosome-damaging effect of bleomycin in G0 human lymphocytes. | 2001 |
|
Sugar-mediated crosslinking of alpha-biotinylated-Lys to cysteamine-agarose support: a method to isolate Maillard Lys-Lys-like crosslinks. | 2001 Apr |
|
Comparison of different protein immobilization methods on quartz crystal microbalance surface in flow injection immunoassay. | 2001 Dec 15 |
|
Pharmacological properties of a newly synthesized H(+)/K(+) ATPase inhibitor, 1-(2-methyl-4-methoxyphenyl)-4-. | 2001 Jan 5 |
|
Gene expression and gene therapy in experimental duodenal ulceration. | 2001 Jan-Dec |
|
Therapy effect of antiulcer agents on new chronic cysteamine colon lesion in rat. | 2001 Jan-Dec |
|
Attenuation of radiation-induced genomic instability by free radical scavengers and cellular proliferation. | 2001 Jul 1 |
|
Effect of centrally administered oxytocin on gastric and duodenal ulcers in rats. | 2001 Jun |
|
[Urease inhibition by polymer analogs of substrate and thiophosphamides]. | 2001 Mar-Apr |
|
Potentiation of NMDA receptor function through somatostatin release: a possible mechanism for the cognition-enhancing activity of GABA(B) receptor antagonists. | 2001 Sep |
|
Role of porphyrin sequestration in the biogenesis of iron-laden astrocytic inclusions in primary culture. | 2002 |
|
Effect of sperm survival and CTC staining pattern on in vitro fertilization of porcine oocytes. | 2002 Apr 15 |
|
Cysteine-mediated electron transfer in syntrophic acetate oxidation by cocultures of Geobacter sulfurreducens and Wolinella succinogenes. | 2002 Jul |
|
Synthesis of (+/-)-madindolines and chemical models. Studies of chemical reactivity. | 2002 Jul 11 |
|
Cysteamine supplementation during in vitro maturation and embryo culture: a useful tool for increasing the efficiency of bovine in vitro embryo production. | 2002 Jun |
|
Negligible urinary cysteamine loss in cystinosis patients with Fanconi syndrome. | 2002 May |
|
Characterization of the surfactin synthetase C-terminal thioesterase domain as a cyclic depsipeptide synthase. | 2002 Nov 12 |
|
Identification and quantitation of key aroma compounds formed in Maillard-type reactions of fructose with cysteamine or isothiaproline (1,3-thiazolidine-2-carboxylic acid). | 2002 Sep 11 |
Sample Use Guides
The recommended maintenance dose of 1.30 grams/m2/day. Patients over age 12 and over 110 pounds should receive 2.0 grams/day given in four divided doses as a starting maintenance dose. This dose should be reached after 4 to 6 weeks of incremental dosage increases as stated above. The dose should be raised if the leukocyte cystine level remains > 2 nmol/1⁄2 cystine/mg/protein.
Route of Administration:
Oral
In Vitro Use Guide
Sources: https://www.ncbi.nlm.nih.gov/pubmed/28384851
Oocytes were obtained from 4-6 weeks old Naval Medical Research Institute (NMRI) female mice, 48 hours after stimulation with Intraperitoneal (IP) injection of 10 IU Pregnant Mare Serum Gonadotropin (PMSG). Germinal Vesicle (GV) oocyte with and without cumulus cells were isolated from ovaries and cultured in Tissue Culture Medium (TCM) 199 with availability of 100 μM of CYS (Cysteamine). After 24 hours, mature oocyte in metaphase II (MII) were fertilized with sperm in In vitro Fertilization (IVF) medium (T6) and evaluated for fetal development into blastocyst. CYS could significantly (p<0.05) increase the rate of IVM and oocyte evolution, and embryo formation in medium culture.
Substance Class |
Chemical
Created
by
admin
on
Edited
Wed Jul 05 23:32:15 UTC 2023
by
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on
Wed Jul 05 23:32:15 UTC 2023
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Record UNII |
5UX2SD1KE2
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Record Status |
Validated (UNII)
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Record Version |
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LIVERTOX |
NBK548701
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WHO-ATC |
S01XA21
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FDA ORPHAN DRUG |
231006
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NCI_THESAURUS |
C29701
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NDF-RT |
N0000175544
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WHO-VATC |
QS01XA21
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FDA ORPHAN DRUG |
404613
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WHO-ATC |
A16AA04
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FDA ORPHAN DRUG |
13986
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EU-Orphan Drug |
EU/3/11/928
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FDA ORPHAN DRUG |
55690
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FDA ORPHAN DRUG |
262408
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FDA ORPHAN DRUG |
258008
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NDF-RT |
N0000187051
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NDF-RT |
N0000175549
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WHO-VATC |
QA16AA04
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FDA ORPHAN DRUG |
368712
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C61696
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CYSTEAMINE
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200-463-0
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CHEMBL602
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60-23-1
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5UX2SD1KE2
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CC-44
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58029
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M4046
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PRIMARY | Merck Index |
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BINDER->LIGAND |
BINDING
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SALT/SOLVATE -> PARENT | |||
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PRODRUG -> METABOLITE ACTIVE |
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ACTIVE MOIETY |
Name | Property Type | Amount | Referenced Substance | Defining | Parameters | References |
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Volume of Distribution | PHARMACOKINETIC |
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