Targeted therapy as a treatment for rare cancer

Targeted therapy is a personalized drug treatment for cancer. It focuses on the cancer’s genetic mutations. Sometimes referred to as precision medicine, targeted therapies can be especially helpful for rare cancers that have no standard of care. This is because they are used based on the presence of a genetic mutation rather than the type of rare cancer. Targeted therapy works by attacking the specific drivers of cancer cell growth in your body. It is different from chemotherapy, which attacks all cells that grow and divide quickly. Targeted therapy is often more precise and less toxic than traditional chemotherapy. It can lead to better outcomes and have fewer side effects.

Small-molecule drug – A drug that can enter cells easily because it has a low molecular weight. Once inside the cells, it can affect other molecules, such as proteins, and may cause cancer cells to die. Most small-molecule drugs can be administered orally, as a pill you can swallow.

Monoclonal antibody – A type of protein that is made in the laboratory and can bind to certain targets in the body, such as antigens on the surface of cancer cells. Antigens are markers that tell your immune system whether something in your body is harmful or not. Monoclonal antibodies are given intravenously (injected into a vein) and can be used alone or to carry drugs, toxins, or radioactive substances directly to cancer cells.

Antibody-drug conjugates (ADC) – A substance that combines a monoclonal antibody with a cytotoxic drug, like chemotherapy. The monoclonal antibody binds to specific proteins or receptors found on cancer cells, allowing the cytotoxic drug to enter these cells and destroy them without harming other cells.