With its p53-dependent mechanism of action, COTI-2 could represent a breakthrough therapy for many cancer patients if clinical trials confirm its’ activity in people. If we consider a specific disease such as ovarian cancer, p53 mutations are found in more than 90% of these tumors. Preclinical animal experiments with a human ovarian cancer known to have a p53 mutation and be resistant to conventional chemotherapy demonstrated that treatment with COTI-2 as a single agent either completely halted tumor growth or lead to dramatic tumor regression depending on the dose. COTI-2 was associated with no observable toxicity in these experiments.
Unlike nearly every other cancer treatment in existence today, COTI-2 is non-genotoxic. Conventional chemotherapy involves the killing of all growing and dividing cells in the body (cancer or otherwise), which often leads to significant toxic side effects in patients. By contrast, COTI-2 specifically targets and primarily destroys tumor cells.