Aclacinomycin A (Aclarubicin) hydrochloride is a fluorescent molecule and the first described non-peptidic inhibitor showing discrete specificity for the CTRL (chymotrypsin-like) activity of the 20S proteasome. Aclacinomycin A hydrochloride is also a dual inhibitor of topoisomerase I and II.

Aclacinomycin A could efficiently enter living cells and emit fluorescence in situ. Aclacinomycin A (10 μM for 15 min) is sufficient for clear detection of fluorescence in most of the cultured cells. Aclacinomycin A treatment (10 μM for 15 min) results in approximately 20% dead (or nearly dead) cells [1]. Aclacinomycin A effectively induces incorporation of exon 7 into SMN2 transcripts from the endogenous gene in type I SMA fibroblasts as well as into transcripts from an SMN2 minigene in the motor neuron cell line NSC34 [2].

Aclacinomycin A is very well absorbed in mice, rats, and dogs after its oral administration. The oral LD50 (76.5 mg/kg) is about twice the iv LD50 (35.6 mg/kg) in mice. Aclacinomycin A (0.75-6 mg/kg, i.p daily) dose-dependently exhibits tumor growth in mice-based Leukemia P-388 model [4].

Animal Model: DBA/2, CDF1 (BALB/c×DBA/2) mice with Leukemia P-388. Dosage: 0.75 mg/kg, 1.5 mg/kg, 3 mg/kg, 6 mg/kg. Administration: Intraperitoneal administration daily for 10 days starting 3 hr after transplantation. Result: Inhibited tumor growth [4].