Perspectives in Bone Marrow Transplantation: The Promise and Premise of Cell Therapy for Children

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By: Laurence Cooper, M.D., Ph.D., associate professor, Department of Pediatrics

Significant advances have been made in the treatment of pediatric cancer through clinical trials that compare chemotherapy regimens in patients to determine which one works best against a particular cancer. While this strategy has worked for the past three decades to improve the chances of children surviving cancer to 80%, this approach is reaching a point of diminishing returns.

Why is this?
For one, pediatric oncologists rely on chemotherapy drugs that were mostly developed decades ago. For children whose cancer relapses, their options are limited due to the lack of new agents on the market. In addition, relapsed cancers tend to be resistant to chemotherapy.

Secondly, for childhood cancer survivors, the drugs that have cured them also have made a long-term impact on their bodies. According to the Childhood Cancer Survivor Study, nearly 75% of childhood cancer survivors will develop chronic health problems or secondary cancers within 30 years of diagnosis. If a child is diagnosed between the ages of 5 and 10, that means that 35- to 45-year-olds are having major problems. New agents are needed to improve the chances of surviving and reduce the toxicities of treatment.

What are the barriers?
The problem pediatric oncologists face is a lack of new drugs designed for pediatric cancers. The bottom line for biopharmaceutical companies is that they typically cannot recoup the financial cost of developing new therapies for rare cancers, such as those in children. So, pediatric oncologists often rely on medicines that were developed for adults and then project their impact for children. This "hand-me-down" approach has inherent problems as no cancer therapy for adults is invented anticipating greater than a 30-year survival from disease.

What's the solution?
Cell-based therapies can provide an answer to these barriers. Many people think of cell therapy as simply bone marrow transplants, but the world of cell therapy encompasses even more therapeutic approaches to treating cancer.

Cell therapy is an approach being pioneered at a few centers to harness the power of a child's immune system to target their tumor. These therapies can be made specifically to kill cancer cells based on an entirely different principal than disrupting cell division like chemotherapy does. You and I are born with an immune system that has an exquisite ability to separate invading organisms from our own cells. Thus, for most of us, we can fight off an infection without fighting off ourselves.

Investigators at M. D. Anderson and elsewhere have developed tools to turn immune cells into cancer-fighting cells. Clinical trials within the Children's Cancer Hospital at M. D. Anderson are now establishing the safety and feasibility of this approach. Investigators are infusing two types of immune cells, T cells and NK cells, as an approach to safely attacking tumors that are resistant to chemotherapy. Just as your naturally occurring immune cells provide ongoing surveillance against recurrence of infection, so the expectation is that these cancer-specific immune cells, provided through immunotherapy (a type of cell therapy), will patrol the child's body and be able to respond and possibly prevent recurrence.

We live at the beginning of the golden age of cell therapy. Clinical trials are investigating the promise of this approach.

1 Comment

It is a very useful article for those who ill. I'll put this article to my term paper to know more about it!

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