Bone Marrow Transplant / Cell and Gene Therapy
Bone Marrow and Peripheral Blood Hematopoietic Stem Cell Transplantation
Although stem cells are now being explored for the treatment of many diseases and conditions, one of the first clinical applications for stem cell therapy was in the treatment of cancer. Blood-forming (hematopoietic) stem cells in bone marrow are quite sensitive to the effects of chemotherapy and radiation and the loss of those essential cells may be a factor that limits the amount of chemotherapy or radiation you are able to tolerate. Stem cells (originally obtained only from bone marrow, but now also prepared from the peripheral blood) make it possible to use higher doses of radiation or chemotherapy to kill cancer cells. You receive stem cells that will repopulate your bone marrow. Following this transplant you are at risk of infection until the donor marrow has engrafted (taken hold). Largely due to this risk of infection, you will be kept in carefully controlled hospital settings, equipped with infection-fighting features such as the advanced air-filtration systems. Stem cell transplantation is a very active area of research, and you may be considered for a clinical trial in this area.
Cell and Gene Therapy
It is a top priority at Houston Methodist to make potentially life-saving treatments available to patients as soon as possible. Your doctors may discuss cell and gene therapy as a potential way to treat your tumor:
- How Cell and Gene Therapy Works
- What to Expect From Cell and Gene Therapy
- Side Effects and Risks of Cell and Gene Therapy
- After Cell and Gene Therapy
How Cell and Gene Therapy Works
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 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 viruses, which have a unique ability to recognize certain cells and insert genetic material into them.
The two general approaches in introducing vectors into the body to deliver genetic content include:
- In ex vivo genetic therapy, cells are taken from your blood or bone marrow and introduced 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.
- In in vivo genetic therapy, vectors (usually viruses) containing the desired gene are injected directly into your body.
What to Expect From Cell and Gene 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. As all current cell and gene therapies are investigational, you will receive a consent form that includes a detailed explanation of the therapy.
Side Effects and Risks of Cell and Gene Therapy
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.
After Cell and Gene 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.
Other Cancer Treatment Options
Learn more about other treatment options, such as chemotherapy, radiation therapy, or surgery. You can also learn more about the unique screening, diagnosis, and treatment for different kinds of cancer.
At Houston Methodist, we are dedicated to research and committed to a goal of making a clinical trial available to every patient who has a medical need and wants to participate. Learn more about our current cancer-related clinical trials.