Details
Stereochemistry | ABSOLUTE |
Molecular Formula | C4H6O6.C2H7NS |
Molecular Weight | 227.236 |
Optical Activity | UNSPECIFIED |
Defined Stereocenters | 2 / 2 |
E/Z Centers | 0 |
Charge | 0 |
SHOW SMILES / InChI
SMILES
NCCS.O[C@H]([C@@H](O)C(O)=O)C(O)=O
InChI
InChIKey=NSKJTUFFDRENDM-ZVGUSBNCSA-N
InChI=1S/C4H6O6.C2H7NS/c5-1(3(7)8)2(6)4(9)10;3-1-2-4/h1-2,5-6H,(H,7,8)(H,9,10);4H,1-3H2/t1-,2-;/m1./s1
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 |
Molecular Formula | C4H6O6 |
Molecular Weight | 150.0868 |
Charge | 0 |
Count |
|
Stereochemistry | ABSOLUTE |
Additional Stereochemistry | No |
Defined Stereocenters | 2 / 2 |
E/Z Centers | 0 |
Optical Activity | UNSPECIFIED |
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 Date1994 |
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 |
|
Sugar-mediated crosslinking of alpha-biotinylated-Lys to cysteamine-agarose support: a method to isolate Maillard Lys-Lys-like crosslinks. | 2001 Apr |
|
An electrochemical evidence of free radicals formation from flutamide and its reactivity with endo/xenobiotics of pharmacological relevance. | 2001 Jan |
|
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 |
|
Cysteamine-colon and cysteamine-duodenum lesions in rats. Attenuation by gastric pentadecapeptide BPC 157, cimetidine, ranitidine, atropine, omeprazole, sulphasalazine and methylprednisolone. | 2001 Jan-Dec |
|
Effects of cysteamine on nociception in mice. | 2001 Jul |
|
Voltammetric behavior of L-cysteine in the presence of CPB at a silver electrode. | 2001 Mar 1 |
|
Gastric antisecretory and antiulcer activity of oxytocin in rats and guinea pigs. | 2001 Nov 21 |
|
New flavanol derivatives from grape (Vitis vinifera) byproducts. Antioxidant aminoethylthio-flavan-3-ol conjugates from a polymeric waste fraction used as a source of flavanols. | 2001 Oct |
|
[Oxidative metabolism of HIV-infected macrophages: the role of glutathione and a pharmacologic approach]. | 2001 Sep |
|
Oxidation and generation of hydrogen peroxide by thiol compounds in commonly used cell culture media. | 2001 Sep 7 |
|
Interventions for paracetamol (acetaminophen) overdoses. | 2002 |
|
Electrochemical and spectroelectrochemical behavior of the main photodegradation product of nifedipine: the nitrosopyridine derivative. | 2002 Apr |
|
The ability of hippocampal CA1 area for induction of long-term potentiation is persistently reduced by prior treatment with cysteamine: an in vitro study. | 2002 Aug |
|
Glutathione in gingival crevicular fluid and its relation to local antioxidant capacity in periodontal health and disease. | 2002 Dec |
|
A molecular CT blood pool contrast agent. | 2002 Jul |
|
Cystinosis. | 2002 Jul 11 |
|
Synthesis of (+/-)-madindolines and chemical models. Studies of chemical reactivity. | 2002 Jul 11 |
|
Gastroprotective effect of adrenomedullin administered subcutaneously in the rat. | 2002 Jun |
|
Cysteamine supplementation during in vitro maturation and embryo culture: a useful tool for increasing the efficiency of bovine in vitro embryo production. | 2002 Jun |
|
Nephropathic cystinosis associated with cardiomyopathy: a 27-year clinical follow-up. | 2002 Nov 9 |
|
Electric potential control of DNA immobilization on gold electrode. | 2003 Jan |
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
Fri Dec 15 15:58:57 GMT 2023
by
admin
on
Fri Dec 15 15:58:57 GMT 2023
|
Record UNII |
QO84GZ3TST
|
Record Status |
Validated (UNII)
|
Record Version |
|
-
Download
Name | Type | Language | ||
---|---|---|---|---|
|
Common Name | English | ||
|
Common Name | English | ||
|
Common Name | English | ||
|
Common Name | English | ||
|
Brand Name | English | ||
|
Common Name | English | ||
|
Systematic Name | English | ||
|
Common Name | English | ||
|
Code | English | ||
|
Systematic Name | English | ||
|
Common Name | English | ||
|
Systematic Name | English | ||
|
Common Name | English | ||
|
Common Name | English | ||
|
Brand Name | English |
Classification Tree | Code System | Code | ||
---|---|---|---|---|
|
NCI_THESAURUS |
C29701
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
||
|
EU-Orphan Drug |
EU/3/14/1306
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
Code System | Code | Type | Description | ||
---|---|---|---|---|---|
|
50386
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
QO84GZ3TST
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
CHEMBL602
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
27761-19-9
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
100000089423
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
81990
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | RxNorm | ||
|
248-641-7
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
DBSALT000032
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
DTXSID801026168
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
m4046
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | Merck Index | ||
|
QO84GZ3TST
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
C47467
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
SUB03168MIG
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY | |||
|
11958147
Created by
admin on Fri Dec 15 15:58:57 GMT 2023 , Edited by admin on Fri Dec 15 15:58:57 GMT 2023
|
PRIMARY |
Related Record | Type | Details | ||
---|---|---|---|---|
|
PARENT -> SALT/SOLVATE | |||
|
PARENT -> SALT/SOLVATE | |||
|
SALT/SOLVATE -> SALT/SOLVATE |
|
||
|
SOLVATE->ANHYDROUS |
Related Record | Type | Details | ||
---|---|---|---|---|
|
IMPURITY -> PARENT |
|
||
|
IMPURITY -> PARENT |
|
Related Record | Type | Details | ||
---|---|---|---|---|
|
ACTIVE MOIETY |