tranylcypromine + all-trans retinoic acid + cytarabine

This combination therapy regimen, known as the TRANSATRA regimen, consists of the LSD1 inhibitor tranylcypromine (TCP), the differentiating agent all-trans retinoic acid (ATRA), and the antimetabolite cytarabine (AraC). Developed by Michael Lübbert and colleagues at the University Medical Center Freiburg, the regimen is designed to treat patients with non-M3 acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS), particularly those who are elderly or unfit for intensive chemotherapy. The mechanism involves TCP-mediated inhibition of lysine-specific demethylase 1 (LSD1/KDM1A), which acts as an epigenetic modifier to sensitize AML blasts to the differentiating effects of ATRA, while low-dose cytarabine provides additional cytotoxic activity by inhibiting DNA synthesis. The regimen has been evaluated in a Phase I/II clinical trial (NCT02717884) for patients who have failed standard treatments like hypomethylating agents.

Other names

TCP + ATRA + AraCTCP + ATRA + low-dose cytarabine