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aerosolized all-trans retinoic acid + vitamin D

Development stage
Preclinical
Modality
Small Molecules
Administration
Inhalation (aerosolized)
01

Overview

This drug is a combination therapy consisting of aerosolized all-trans retinoic acid (ATRA) and vitamin D or its analogs. ATRA is a derivative of vitamin A that acts as a ligand for the retinoic acid receptor alpha (RARα), promoting differentiation and apoptosis in certain malignant cells, notably acute promyelocytic leukemia (APL) cells. Vitamin D compounds, particularly active forms like 1,25-dihydroxyvitamin D3 and its analogs, induce differentiation in myeloid leukemia cells and have antiproliferative effects on various cancer cell lines. The combination leverages the complementary mechanisms of both agents to enhance antitumor activity by promoting cellular differentiation and inhibiting proliferation without causing significant hypercalcemia. Aerosolization allows direct delivery to lung tissue or respiratory tract for localized effect or systemic absorption depending on indication under study. This combination has been investigated primarily as an adjuvant therapy in cancers such as breast cancer and acute myeloid leukemia (AML), with potential applications also explored in inflammatory conditions due to anti-inflammatory properties of ATRA[1][2][6][7].

Other names
aerosolized all trans retinoic acid + vitamin d
02

Targets

RARA (Retinoic acid receptor alpha)VDR (Vitamin D receptor)

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