What is Targeted Therapy?

Our goal is to improve the effectiveness of cancer therapy, while at the same time minimizing drug side effects. To do this, we have partnered with pharmaceutical companies who are actively developing a new generation of cancer drugs collectively known as targeted therapy. Instead of simply acting as non-specific “cell poisons”, as do traditional chemotherapy drugs, these new inhibitors target and interfere with the activity of specific proteins that drive tumor growth and spread. Some targeted therapies are specific to a single gene or protein, whereas others can simultaneously target any one of a group of these factors. By therapeutically targeting the abnormal protein activity that is specific to the cancer cells, the impact of these drugs on normal cells is dramatically reduced, thereby limiting side effects. In general, there are two classes of targeted therapy:
Small molecule inhibitors are drugs that enter the cell and mostly block the activity of specific proteins whose activities have been abnormally turned on. Current small molecule inhibitors particularly focus on a class of proteins known as kinases, which send signals that promote cancer cell growth, division and survival. Small molecule inhibitors have been particularly successful in the treatment of patients with certain types of lung cancer (EGFR inhibitors such as erlotinib and gefintib, ALK inhibitors such as crizotinib), melanoma (BRAF inhibitors such as vemerafenib), breast cancer (HER2/ERBB2 inhibitors such as lapatinib), chronic myelogenous leukemia (BCR-ABL inhibitors such as imatinib mesylate and dasatinib) and gastrointestinal stromal tumor (KIT inhibitors such as imatinib mesylate).
Therapeutic monoclonal antibodies cannot enter the cell but instead bind to the outside of the cell and block the carcinogenic activity of proteins called receptors on the cell surface. Many of these cell surface receptors work in similar ways as the kinases described above, promoting cancer cell growth, division and survival. Therapeutic monoclonal antibodies have been particularly successful in the treatment of a subset of patients with breast cancer (HER2/ERBB2 antibodies such as trastuzumab), colon cancer (EGFR antibodies such as cetuximab and panitumumab) and squamous cell carcinoma of the head and neck cancer (EGFR antibodies such as cetuximab).
While targeted therapy represents a major advance in the treatment of cancer, it has become clear that this approach can be most successful when drug selection is matched to specific genetic signatures found in the tumor. The Targeted Cancer Care website contains the latest information related to these genetic and molecular biomarkers within the tumor that can guide the use of specific targeted anti-cancer therapies. A useful search tool is also provided that matches a known tumor genetic signature to the latest experimental drugs that are being investigated in currently available clinical trials.

How can we help?

Contact the Cancer Center to learn more about the testing and treatment options at Mass General.
  • Call 617-726-0808

How do I get tested?

Getting tested in one of the first steps in learning what targeted therapy may work for you. 

Learn more about how to get tested.