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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
Structure of CYSTEAMINE

SMILES

NCCS

InChI

InChIKey=UFULAYFCSOUIOV-UHFFFAOYSA-N
InChI=1S/C2H7NS/c3-1-2-4/h4H,1-3H2

HIDE SMILES / InChI

Molecular Formula C2H7NS
Molecular Weight 77.149
Charge 0
Count
MOL RATIO 1 MOL RATIO (average)
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 0
Optical Activity NONE

Description

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 TargetPharmacologyConditionPotency
178.0 µM [IC50]

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
CYSTAGON

Cmax

ValueDoseCo-administeredAnalytePopulation
2.7 mg/L
404 mg/m² 4 times / day steady-state, oral
CYSTEAMINE plasma
Homo sapiens
37.72 μM
450 mg single, oral
CYSTEAMINE plasma
Homo sapiens

AUC

ValueDoseCo-administeredAnalytePopulation
351 mg × min/L
404 mg/m² 4 times / day steady-state, oral
CYSTEAMINE plasma
Homo sapiens
96 μM × h
450 mg single, oral
CYSTEAMINE plasma
Homo sapiens

T1/2

ValueDoseCo-administeredAnalytePopulation
90 min
404 mg/m² 4 times / day steady-state, oral
CYSTEAMINE plasma
Homo sapiens

Funbound

ValueDoseCo-administeredAnalytePopulation
48%
404 mg/m² 4 times / day steady-state, oral
CYSTEAMINE plasma
Homo sapiens
48%
450 mg single, oral
CYSTEAMINE plasma
Homo sapiens

Doses

AEs

Drug as perpetrator​

Drug as victim

PubMed

Sample Use Guides

In Vivo Use Guide
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
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
Record UNII
5UX2SD1KE2
Record Status Validated (UNII)
Record Version