They may also increase the risk of developing blood clots in the leg veins, which may require anticoagulant drug treatment. In the initial studies, drugs will be evaluated one by one. It is also a possibility that a single drug therapy may not be as effective as the approach known as combination therapy. There is experimental evidence that treatment with a chemotherapy drug followed by a differentiation inducer may be more effective than treatment with a differentiation inducer alone, for example. In fact, the strategy of designing regimens which combine drugs with different mechanisms of action and different side effects has been a successful approach to treating several different types of cancers.
Clinical trials provide a structure to test the safety and efficacy of a treatment.
Before the Food and Drug Administration can consider a drug for approval, it must go through at least three phases of clinical trials. These studies also determine how a drug acts in the body and the duration of its action. Phase III trials usually involve randomized assignment of patients to either experimental drug or an established treatment. If there is no established treatment, the drug is compared to a placebo. Prior to joining the University of Pittsburgh Cancer Istitute, Dr. Lieberman was a faculty member in the Department of Neurology and the Ruttenberg Cancer Center at Mount Sinai Medical School in New York City and he was a member of the Department of Neurology at Memorial Sloan-Kettering Cancer Center.