Brefeldin A was initially isolated with hopes to become an antiviral drug[3] but is now primarily used in research to study protein transport.

[5] However, upon discovery of its mechanism involving disruption of protein transport by Takatsuki and Tamura in 1985 and the cytotoxic effects observed in certain cancer cell lines, research efforts were revitalized.

[5] It is currently used solely in research mainly as an assay tool for studying membrane traffic and vesicle transport dynamics between the endoplasmic reticulum and Golgi apparatus.

[citation needed] In mammalian and yeast cells, the main target of brefeldin A appears to be a guanine nucleotide exchange factor (GEF) called GBF1.

Activated Arf1p then recruits coat protein β-COP, a subunit of the COP-I complex, to cargo-bound receptors on the membrane.

[12] Generally, antibiotic macrolides that share a similar macrocyclic lactone ring to that of brefeldin A have been shown to produce gastrointestinal discomfort as the most common side effect.

[13] Some macrolides have been shown to produce allergic reactions and though uncommon this possibility in the case of brefeldin A cannot be disregarded as of yet.

[13] Brefeldin A is not considered to be harmful from direct skin or eye exposure other than transient irritation.