Very early clinical testing of a new drug will involve treatment of patients to determine the side effects of the drug, the maximum dose tolerated, and a first impression about the beneficial effects of the drug. With anti-cancer drugs, this means treating patients with a wide array of tumors, to determine whether any tumors have a better chance of responding. Since no beneficial effect can be predicted, only patients whose cancers are not responding to standard therapies are invited to participate. Both possible benefit and possible risks are quite uncertain. Before deciding to participate in phase I studies, you should obtain information about the known risks of this drug, and about other experimental treatments.
Some patients are nervous about participation in clinical studies.
These emotions are understandable, but ill-founded. All progress made in the field on cancer came from the testing of new drugs that proved more effective than older ones. Further progress will depend on patients willing to be active participants in the evaluation of the new generation of drugs. Similarly, randomized studies are the only way to select the optimal therapy from among several good therapies. In many instances, randomized clinical trials are the standard of care.