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
Cysteamine on DrugBank
Cysteamine on Wikipedia

Synonyms

mercaptamine bititrate

Marketed as

Dietary sources

N/A

Structure not available

C2H7NS

Drug-Mutation Relation

Treats

Mutation Number of sources Average impact factor
F508del
2 8.70
Link Tested on Impact factor Notes
Cysteamine re-establishes the clearance of Pseudomonas aeruginosa by macrophages bearing the cystic fibrosis-relevant F508del-CFTR mutation. murine cell cultures 5.64
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 Number of sources Average impact factor