Living with Leukemia : Bone Marrow Transplants, Biologic Therapy

by Food and Drug Administration (FDA)

(Page 3 of 3)

Bone Marrow Transplants

Bone marrow transplants offer some people like 44-year-old Tom Kochanowicz of Omaha, Neb., the best chance of survival and, in his case, a cure. Kochanowicz was diagnosed with CML at age 38 when his doctor detected a lump in his side. "Fortunately," he says, "mine was the slow-moving kind and initially was managed with medications to bring my white blood cell count under control."

However, Kochanowicz says the side effects of the radiation and chemotherapy treatments took their toll. "The aggressive treatment was wiping out my immune system," he remembers. Consequently, despite hopes that the drug treatments would conquer, or at least manage, his leukemia, Kochanowicz admits he had always known that his life ultimately would depend on a bone marrow transplant.

In this procedure, existing abnormal bone marrow is eliminated through radiation treatments or chemotherapy. Healthy marrow is then injected directly into the bloodstream in a procedure similar to a blood transfusion. The bone marrow migrates to and takes root in the recipient's bones, and the cells begin to divide. It generally takes about three weeks and sometimes longer for the transplanted bone marrow to start producing white blood cells to protect against infections, making the procedure quite risky. Healthy marrow may have been supplied either by the patient in the early stage of the disease, or by a donor. Only someone who has a compatible tissue type — ideally a close relative — can be a donor.

Both of Kochanowicz's brothers were perfect blood matches, giving him excellent odds for a full recovery. As the result of a successful transplant, Kochanowicz is now entering his sixth year of remission. Keller wasn't as fortunate.

"We were told that brothers and sisters were the best matches," says Kathy Keller. "But since Neil was an only child, we tested the kids, me, and his parents." Keller's father ended up donating the needed bone marrow.

A parent or child is able to donate needed bone marrow only about 1 percent of the time, says Edwin P. Alyea, a medical oncologist with the Dana-Farber Cancer Institute in Boston. As in Keller's rare situation, Alyea says, "When they can, that's very lucky."

He also says that unrelated donor registries make it possible for people to have blood matches outside their families. These registries increase the chances of finding donors based on ethnic background and other specific qualifications. But Alyea says that "more minorities need to contribute to the registries" and greater outreach is needed in these communities.

A major complication of bone marrow transplants is graft-versus-host disease (GVHD) in which the transplanted marrow cells react against the patient's tissues; primarily the liver, the skin, and the digestive tract.

Despite surviving leukemia, Kochanowicz's last six years haven't been easy. His transplant experience included severe GVHD — for nearly two years he was unable to swallow, and a condition called scleroderma hardened the connective tissue in his skin.

Alyea explains that although a bone marrow transplant plays a key role in the treatment for certain types of leukemia, as a whole "it is basically an exchange of one disease for another. You're trading the disease itself, for which the treatment options and the chances of cure may be limited, for the possible complications of the transplant treatment," he says.

Alyea also says doctors have learned that patients who develop acute or chronic GVHD have a lower risk of the disease returning after the bone marrow transplant. "This demonstrates that the donor's immune system may play a role in cure," says Alyea. After a long and frustrating battle with the unpleasant effects of GVHD, Kochanowicz now manages his symptoms with medications, and admits that he feels better than he has "in a very long time."

Biologic Therapy

A relatively new addition to the family of cancer treatments is biological therapy (sometimes called immunotherapy). Biological therapy uses the body's immune system, either directly or indirectly, to fight cancer or to lessen the side effects that may be caused by some cancer treatments.

Patricia Keegan, M.D., a deputy division director in the FDA's Center for Biologics Evaluation and Research, explains that the immune system is a complex network of cells and organs that work together to defend the body against foreign invaders. This network is one of the body's main defenses against cancer. For example, the immune system may recognize the difference between healthy cells and cancerous cells in the body, and work to eliminate those that become cancerous.

Biological therapies, says Keegan, are designed to repair, stimulate, or enhance the immune system's responses when cancer prevents it from functioning adequately. Some immune system substances can be produced in the laboratory for use in cancer treatments. But Keegan says that, although biological drugs can be effective in treating different types of leukemia, there are so few available because "they are labor-intensive — the development of blood products is a relatively young field that is still developing."

The Future

Scientists are finding better ways to treat leukemia, and the chances of recovery keep improving. A physician who specializes in the treatment of leukemia is in the best position to discuss a person's prognosis and to offer the best course of treatment for a particular type of leukemia.

Survival rates may indicate how long groups of people may live. However, it's important to remember that statistics are averages based on large numbers of people. These numbers cannot be used to predict what will happen to an individual because no two people are identical, and treatments and responses vary.

About the Author

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