A New Paradigm for Designing Clinical Trials

As researchers have gained insight into the fact that cancer develops as a result of cellular mutations in genes that alter the normal structure of certain proteins, a new paradigm in cancer diagnosis, treatment, and prevention is now emerging.  This shift in thinking has arisen from a clearer understanding of how changes at the molecular level of the cell lead to cancer.  The alterations in normal cellular pathways from these gene mutations result in uncontrolled cellular growth and cancer.  Subsequently new anti-cancer agents are being directed toward the specific molecular targets present within the cancer cell, thus leaving the normal cells unharmed.  These agents specifically target abnormal proteins, genes, or gene products, which directly relate to the process of neoplasia, and cancer cell growth.  This methodology allows for more efficient treatment of the tumor, with less healthy tissue destruction thus greatly decreasing toxic side effects.

Some examples of clinical trials emerging from this paradigm include the treatment of chronic myeloid leukemia (CML) in adults and children with imatinib (Gleevec).  Imatinib blocks the cancer-causing effects of a genetically altered protein commonly found in CML.