Bone Marrow Transplant - Children

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Pediatric Clinical Studies

If your child has cancer, your first reaction when asked to enroll your child in a clinical study may well be, “No way are you going to experiment on my child!” If this is your reaction, consider this: Clinical trials are proven to offer children with cancer the best chance of survival.

In fact, more than half of all American children with cancer do take part in clinical studies, which is one reason that cure rates for childhood cancers have improved so remarkably over the past few decades. In contrast, only about 5 percent of adult cancer patients enroll in clinical studies, also called clinical trials.

One reason so many children take part in clinical studies is that—again, in contrast to adults—most young cancer patients are treated at academic medical centers. These centers have always had the two-fold mission of caring for patients and doing research to improve treatment methods and survival rates.

“We believe passionately in clinical trials—because they have worked,” says Dr. Russell Geyer, MD, a pediatric oncologist with Seattle Children’s and SCCA. “The overall likelihood of being cured of a childhood malignancy is 80 to 85 percent. In the early days, it was only 20 to 25 percent, and most of that improvement has been the result of clinical trials.”

Children must be included in clinical research because for the most part they have different cancers than adults do. So any new treatment for children must be tested in children. In addition, children respond differently to drugs than adults do, and Dr. Geyer says that children can often tolerate higher doses (relative to their size) of chemotherapy drugs.

It is important for parents to realize that in clinical studies doctors and researchers are not “experimenting” with totally unproven drugs in clinical trials. Even when a clinical study is testing a new drug not previously used in children, the drug has already been tested in adults.

In a typical randomized clinical trial, Dr. Geyer says, the children in one group will receive the standard treatment (which is often the best result of the last clinical trial), while children in the investigational group will receive the new treatment. This new treatment is often very similar to the standard treatment, but with different doses or different combinations of chemotherapy drugs that previously proved to be effective.

Read Dr. Geyer’s advice to parents considering a clinical study.