Camptothecin and some of its derivatives, all inhibitors of topoisomer
ase I, have been found to inhibit growth and induce regression of huma
n colon carcinoma xenografts in nude mice. Some clinical trials of the
se compounds have been already completed, many more are being held. Th
e mother compound, camptothecin, which is water insoluble, has been ad
ministered orally in a Phase I clinical trial. Main toxicity encounter
ed has been diarrhea with minimal leukocytopenia. Camptothecin is now
in Phase 2 clinical trials. 9-Aminocamptothecin, a water-insoluble der
ivative, is now in Phase I trials. Topotecan and iridotecan, two water
-soluble derivatives, have undergone Phase I trials, showing mostly in
testinal toxicity, followed by bone marrow depression.