Clinical trials of gene/biotechnology products

Gene/biotechnology products can be either physiological peptides for the purpose of substitution of deficiencies or for therapeutic purposes in superphysiological concentrations, or nonphysiological peptides, or newer biotechnology products like monoclonal antibodies. The rules for clinical trials developed so far are also valid for clinical trials with gene/biotechnology products. Nevertheless, a major challenge for the clinical pharmacologist is the species specificity of many reactions induced by gene/biotechnology products in man. In general, animal experiments may be less predictive, so there is a greater demand for human pharmacology studies. Gene/biotechnology products offer more chances for treatment of many diseases, but during the clinical trials the clinician has to always be aware of unexpected side effects. Several newer gene technology products offer superior safety as compared to older biological products like Factor VIII preparations and human growth hormone. More than 19 therapeutics produced by gene/biotechnology have already been approved by health authorities all over the world. Major clinical benefit could be shown with hepatitis B vaccine, insulin, human growth hormone, TPA, erythropoietin, GM-CSF, G-CSF, and monoclonal antibodies for immune suppression. There is also good evidence of efficacy of interferon alpha in chronic hepatitis. So far, our knowledge about cytokines is still limited. In several cancer diseases, interferons show efficacy as well as in several autoimmune diseases. Well designed clinical pharmacology studies will be important to elaborate the therapeutic potential of drugs arising from gene/biotechnology.