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.