To evaluate the in vivo efficacy and clinical toxic effects of the 1,25-dihydroxy-16-ene-23-yne-vitamin D3 analogue in β-luteinizing hormone-Tag (LHβ-Tag) transgenic mice with heritable retinoblastoma.
Forty-two mice (8-10 weeks old), randomly assigned to experimental (n=21) or control (n=21) groups, received intraperitoneal injections of 0.05 μg of 1,25-dihydroxy-16-ene-23-yne-D3 in 0.5-mL mineral oil vehicle (experimental group) or 0.5 mL of mineral oil vehicle (control group) for 5 weeks. One experimental and 3 control animals died of injection-related trauma. Eyes were enucleated 1 week after treatment and were examined histologically in a masked fashion.
All experimental and control animals showed evidence of tumor. The tumors in the experimental mice showed a significantly smaller cross-sectional area (0.88±0.08 mm2) compared with that in the control mice (1.12 ±0.12 mm2) (P=.02). All mice completed the treatment and showed no clinical evidence of toxic effects.
Tumors in transgenic mice with retinoblastoma treated with 1,25(OH)2-16-ene-23-yne-D3 showed a 21% smaller cross-sectional area compared with that in the control mice, without producing clinically apparent toxic effects. This compound may be useful as adjunctive therapy in the treatment of retinoblastoma.
Shternfeld IS, Lasudry JGH, Chappell RJ, Darjatmoko SR, Albert DM. Antineoplastic Effect of 1,25-Dihydroxy-16-ene-23-yne-Vitamin D3 Analogue in Transgenic Mice With Retinoblastoma. Arch Ophthalmol. 1996;114(11):1396-1401. doi:10.1001/archopht.1996.01100140596012