Gene therapy is the use of genes in the treatment of diseases in the body. Genes are made from DNA and are the basic unit of heredity. Any type of treatment that can change a gene's structure or function is considered gene therapy. Since cancer is a disease of genetic changes, gene therapy has great promise in prevention and treatment. It is being studied for use in a number of ways.

One approach to gene therapy is to supply healthy copies of missing or flawed genes. Instead of giving a patient a drug to treat or control the symptoms of the disease, researchers try to correct the basic problem by changing the genetic makeup of some of the patient's cells. Another kind of gene therapy uses genes to keep cancer cells from making new blood vessels, which helps stop cancer growth. Other gene therapies include adding genes to cancer cells to make them easier for cancer treatments or the patient's immune system to kill. Some cause the patient's white blood cells to make a special protein that helps them find and attack tumor cells. Newer gene treatments give the patient a "pro-drug" that inserts suicide genes into cancer cells. This causes the cancer cells to die.

As of early 2008, gene therapy is still experimental. You can only be treated with gene therapy in clinical trials or research studies. Gene therapy for cancer is a challenge because cancer is not caused by one single genetic flaw, but a combination of gene flaws. Many gene treatments are being studied today to find out how safe they are and how useful they might be. Your doctor or cancer care team will be able to tell you more about clinical trials using gene therapy.

If you are getting gene therapy:

What the patient can do

• Go to every scheduled appointment.
• Ask questions. Be sure you understand your treatment. Your cancer care team will help you.
• Ask about expected side effects and what to do if you have any.
• Ask when you should call your doctor.
• If you are having symptoms such as nausea or vomiting, see the related section, and let your doctor know.

What caregivers can do

• Go with the patient to appointments to learn about the gene therapy he is getting and any expected effects.
• Find out how to reach the doctor when the office is closed.
• Help the patient watch for and manage side effects.

Call the doctor if the patient:

• Has a fever (See the section on fever.)
• Has any bleeding
• Has any other side effect you've been told you should report
• Has any change in how he is feeling
• Has questions or hears things about gene therapy that cause concern

Go back to Caring for the Patient with Cancer at Home: A Guide for Patients and Families.

Last Medical Review: 04/27/2009
Last Revised: 04/27/2009