Cancer cells and normal cells have much in common in terms of the internal machinery that allows them to carryout the many activities necessary to stay alive. Chemotherapy drugs effectively target processes that cancer cells need to grow and divide, such as the ability of the cancer cells to replicate their DNA. However, many normal cells, like the cells that line the digestive tract, also need to replicate. In short, though chemotherapy drugs are particularly toxic to cancer cells, they also damage healthy cells. The use of standard chemotherapy therefore produces many, and often severe, side effects. Furthermore, these side effects sometimes prevent patients from being able to take high enough doses to fight the cancer most effectively. While chemotherapy drugs are quite effective treatments for many forms of cancer, researchers have been working diligently to produce drugs that target the processes of cancer cells specifically so as to leave healthy cells unharmed. The accumulation of knowledge about the specific differences between normal and cancerous cells has allowed for the development of treatments targeted at cancer-specific activities.
One of the most fundamental changes found in cancer cells is the presence of mutations in the genes that are responsible for causing cell growth (oncogenes). The defective proteins produced by these altered genes are prime candidates for targeted therapy. As an example, some cancers are caused in part by mutant proteins that send constant signals into the cell causing cell division. Drugs that block only the mutant form of the protein but do not interfere with the activity of the normal version would only affect cancer cells, and would leave healthy cells untouched. Alternatively, many cancers result when genes that normally prevent cell growth (tumor suppressors) are inactivated or turned off. Drugs that "fix" the activity of these proteins would repair the damaged cancer cells, but theoretically have no effect on normal cells.