Division of Hematology/Oncology


Targeted Molecular Therapy

Targeted molecular therapy at Penn Medicine is a type of personalized medical therapy designed to treat cancer by interrupting unique molecular abnormalities that drive cancer growth. Targeted therapies are drugs that are designed to interfere with a specific biochemical pathway that is central to the development, growth and spread of that particular cancer.

About Targeted Molecular Therapy

Because not every cancer develops in the same way in every person, targeted molecular therapy is personalized to the individual. In some cancers the molecular targets are known. But in other cancers these targets are still being identified. In some cases, the same types of cancer have different molecular targets. Identifying the molecular targets in any given patient's cancer requires working closely with pathologists to carefully analyze the cancer pathology.

Targeted cancer therapies such as targeted molecular therapy give medical oncologists a better way to customize cancer treatment. Advantages of molecularly targeted therapy include:

  • Potentially less harm to normal cells
  • Potentially fewer side effects
  • Improved effectiveness
  • Improved quality of life

Examples of Targeted Molecular Therapy

Some examples of targeted molecular therapy include:

  • Selective BRAF inhibitor vemurafenib for BRAF mutant melanoma targets the protein (BRAF) involved in normal cell signaling. Mutations in the gene for BRAF are found in about half of melanomas as well as some other adult cancers. In melanoma the cancers uses the mutated BRAF protein to grow and spread. By interfering with the functions of the mutated BRAF protein with drugs such as vemurafenib, the melanoma tumors often stop growing and spreading, and in some cases even shrink.
  • Imatinib and nilotinib target a protein (BCR-ABL) that is critical to the growth of chronic myelogenous leukemia cells
  • Erlotinib targets a protein called the epidermal growth factor receptor or EGFR. This protein is involved in cell signaling and is mutated in up to 25 percent of lung cancers, especially in cancers developing in never or light smokers.
  • Trastuzumab targets a cell signaling protein called HER2 that is overactive in about 25 percent of breast cancers.
  • Other examples of targeted therapies include lapatinib for breast cancer; crizotinib for lung cancer; bevacizumab for lung and colon cancer; and sorafenib for liver and kidney cancer.

The Penn Difference

At Penn, medical oncologists have experience and expertise delivering targeted molecular therapy, and are leading research to find newer and more effective ways to treat cancer with molecular targeted therapies.

At Penn, targeted cancer therapies are being studied for use alone, in combination with other targeted therapies, and in combination with other cancer treatments, such as chemotherapy and radiation.

Penn's multidisciplinary approach to medical treatment helps patients and their families achieve the best quality of life. Medical oncologists at Penn coordinate overall patient care and direct targeted molecular therapies and other related treatments.

Diseases Treated with Targeted Molecular Therapy

Targeted molecular therapy is not appropriate or feasible for all patients and all types of cancer. Hematologists/oncologists at Penn Medicine use targeted molecular therapy to treat:

  • Brain cancer
  • Breast cancer
  • Gastrointestinal (GI) cancer
  • Head and neck cancer
  • Kidney cancer
  • Leukemia
  • Lung cancer
  • Melanoma
  • Mesothelioma
  • Myeloma
  • Prostate cancer
  • Thyroid cancer

In addition to targeted molecular therapy, Penn Hematology/Oncology treatments include:

  • Bone marrow transplant and stem cell therapy
  • Chemotherapy
  • Hormone therapy
  • Immunotherapy
  • Vaccine therapy

Patients at Penn Medicine also have the expertise and support of the Abramson Cancer Center, a comprehensive cancer center recognized by the National Cancer Institute as "exceptional" that provides patients with specialized cancer treatment and support teams.