Lycopene for Androgen-Independent Prostate Cancer. Dr. Aminah Jatoi, Mayo Clinic Dr. Jatoi presented the study design of a Phase II trial conducted by the North Central Cancer Treatment Group, an NCI-funded national cooperative group based in Rochester, Minnesota. The trial will build on prevention data; will investigate whether cancer patients, who are voluntarily supplementing with agents such as lycopene, are deriving a benefit; and will expand the limited therapeutic options in androgen-independent prostate cancer. Preliminary clinical data also support further study of lycopene for the treatment of cancer. In a study by Ansari and Gupta (8), for example, 20 androgen-independent prostate cancer patients received 10 mg per day of lycopene. The investigators found a 35 % response rate (described as a drop in PSA level) in the cohort. In this trial, 40 patients with asymptomatic, androgen-independent prostate cancer were treated with 15 mg of lycopene twice a day, administered as a tomato foodstuff. The primary endpoint is a 50 % confirmed response in PSA drop. Accrual is complete, and the data are being analyzed. The 30 mg/d lycopene dose was chosen based on preliminary efficacy data from other studies. The trial relies on a historical control group, based on a previous published and peer-reviewed study by Dr. Jatoi with the same eligibility criteria, which found that the agent being tested had no efficacy. Compliance was not monitored because the patients are highly motivated (i.e., they are not anticipating cancer, they already have hormone-refractory cancer). In addition, even without compliance data, the trial still should provide patients with some preliminary guidance as to whether to take lycopene. Discussion A participant questioned the logic/ethics of a trial without a control group. Interpretion of the outcome or making inferences for patient care will be difficult if not impossible. Dr. Jatoi replied that Phase II studies often are conducted in a cancer setting. The data can be interpreted because the natural history of hormone-refractory prostate cancer is known. Dr. Jatoi tried to include a control arm in an earlier trial, but the NCI questioned the ethics of a nontreatment arm as part of a trial in patients with active disease. It also is possible that the agent in the control arm (e.g., soy) would have therapeutic efficacy, leading to equivalence between arms. Another participant added that, in medical oncology, Phase II clinical trials often are conducted to collect preliminary data for Phase III clinical trials, not to make recommendations to the public. Furthermore, there is no effective treatment for the patients in this study, so any nontoxic agent should be explored. A suggestion was made to design trials with similar agents and control arms, so that the data have the potential to be merged. It was also noted that when exploring indications of toxicity, it is helpful to monitor side effects in the control arm. It was suggested that a decrease in the slope of rise in PSA might be useful to examine. Dr. Jatoi might observe a decrease in PSA doubling time, which will prolong the patients’ survival. The chosen endpoint, a decrease in PSA, sets up the trial for failure. Dr. Jatoi responded that, although the rate of rise in PSA was not chosen as an endpoint, this could be examined as well. 2/05 NIH Meeting Remember we are NOT Doctors and have NO medical training. This site is like an Encylopedia - there are many pages, many links on many topics. Support our work with any size DONATION - see left side of any page - for how to donate. You can help raise awareness of CAM.