Center for Cell & Gene Therapy

Your genes are the parts of your cells that contain DNA, the “code” that determines your body’s form and function. Researchers have found that certain genetic defects can cause a variety of diseases, including cancer.

Cell and gene therapy as a cancer treatment can work in many different ways:

• Changing the genes that control your normal immune cells so that they are better able to recognize and kill the cancer
• Engineering immune cells to recognize specific viruses in order to restore patient immune systems after bone marrow and stem cell transplantation
• Blocking dangerous pathways or otherwise inhibiting cancer growth using small molecule inhibitors or monoclonal antibodies

Because a gene cannot be inserted directly into a cell, it must be delivered using a carrier, also called a vector. The most commonly used vectors in cell and gene therapy are harmless viruses, which have a unique ability to recognize certain cells and insert genetic material into them.

 
The two general approaches for introducing vectors into the body to deliver genetic content include:

• Taking cells from your blood or bone marrow and introducing these cells to the vector carrying the desired gene in the laboratory. The cells are grown in a culture in the lab and then reintroduced into your body. This is called ex vivo genetic therapy.
• Delivering a synthetic vector (usually a harmless version of a virus) containing the desired gene directly into your body via injection. This is called in vivo genetic therapy.

Your specific treatment will depend on the type of cancer you have and the type of cell and gene therapy being used.

Your doctor will discuss any relevant protocols with you, including potential risks and benefits. Because all current cell and gene therapies are investigational, you will receive a consent form that includes a detailed explanation of the therapy.

As with any cancer treatment, it is important to pay close attention to the instructions from your doctor and know which warning signs to watch out for in the days and months following your procedure.

Because cell and gene therapy is still in the experimental stage, there may be risks that have not yet been discovered. Certain risks are possible, especially when viruses are used as carriers for the genetic material.

Your doctor will discuss the potential risks of the therapy with you, and any possible side effects will also be outlined in detail on the consent form you receive.