Cysteamine

Proteostasis regulator, rescues the function of F508del-CFTR

Phase of research

FDA-/EMA-approved for Other Purpose

How it helps

Restore CFTR Function

General information

Cysteamine is a compound that is naturally present in mammalian cells. It is a radiation-protective agent that can be used to treat radiation sickness. Its bitartrate and hydrochloride salts are indicated for the treatment of neuropathic cystinosis. Cysteamine is marketed under several names, e.g. Cystagon®, which was approved in the USA in 1994. In relation to cystic fibrosis, our AIM tool found the data that cysteamine can help rescue F508del-CFTR from degradation and reduce lung inflammation.

Cysteamine on PubChem
C ysteamine on DrugBank
Cysteamine on Wikipedia

Synonyms

mercaptamine bititrate

Marketed as

Cystagon - prescribing information

Dietary sources

N/A

C₂H₇NS

Drug-Mutation Relation

results for D1152H / F508del

See all data on Cysteamine

Treats

Mutation	Link	Tested on	Impact factor	Notes
F508del	Cysteamine re-establishes the clearance of Pseudomonas aeruginosa by macrophages bearing the cystic fibrosis-relevant F508del-CFTR mutation.	murine cell cultures	5.64
F508del	Restoration of CFTR function in patients with cystic fibrosis carrying the F508del-CFTR mutation	mice	11.75	Better followed by epigallocatechin gallate treatment.

Does not treat

Mutation	Link	Tested on	Impact factor	Notes