Rare Procedure to Fight Childhood Cancer
So, early next week, the 12-year-old boy from Pecatonica, Illinois will undergo a rare procedure at American Family Children's Hospital in an attempt to save his life. The experimental treatment relies heavily on "natural-born killers", specialized cells that can seek out and destroy the cancer cells in his body.
Ben has neuroblastoma, a type of cancer in which solid tumors arise from cells that look like primitive nerve cells. Standard treatments – surgery, chemotherapy and radiation – have not cured his disease and several experimental therapies also haven't worked.
So his medical team will try a novel approach that has not yet been tested in children with cancer.
Next Monday, Ben will receive a stem-cell transplant from his mother, Peggy. Worldwide, fewer than 50 children with a solid tumor have been treated with a stem-cell transplant from a parent.
But Ken DeSantes, MD, Ben's pediatric oncologist, is adding another twist: after his mother's stem cells are infused, Ben will get a second infusion of "natural killer" (NK) cells. The research team wants to find out if the killer cells in her immune system can recognize her son's tumor cells and destroy them.
DeSantes, associate professor of pediatrics at the UW School of Medicine and Public Health, says this second part of the transplant makes the procedure unique.
"The concept is that the immune system of the donor will eradicate cancer cells resistant to chemotherapy and radiation," he said. "We are taking the approach one step further by isolating additional NK cells from the donor and infusing these into the patient post-transplant. We believe these NK cells may be able to mediate a potent anti-tumor reaction."
According to Peiman Hematti, MD, director of the cell-processing laboratory at UW, a few hundred milliliters of blood will be taken from Peggy Johnson and processed in a lab before certain cells from it are given to her son.
"The laboratory had to receive approval from the Food and Drug Administration (FDA) before being allowed to proceed with this operation," says Hematti. "This usually involved performing several test samples to convince the FDA that the lab is capable of such procedures."
Forty years ago, UW Hospital started using bone marrow transplants to treat childhood cancers such as leukemia and neuroblastoma by using the child's own stem cells.
DeSantes said this approach expands on the work of Dr. Hans Grettinger, who researched the use of NK cells to treat childhood cancer at hospitals in the United States and Germany.
Funding for the research is provided by the Midwest Athletes against Childhood Cancer (MACC) Fund, Miltenyi Biotec, a global biomedical firm, and Viracor who will be monitoring patients for the development of viral infections. DeSantes says the research could provide an exciting new strategy to treat children with cancer, but one of the biggest roadblocks is cost.
"Money from the MACC fund seeded this research and really got it off the ground. I am very grateful to them," said DeSantes. "We are currently seeking additional funds so that the treatment can be offered to other patients who might benefit from this approach."
Ben entered the hospital earlier this month to prepare for the stem-cell and NK cell transplants. The success of the procedure will not be known for several months when Ben undergoes testing to determine if his tumors are regressing.
If the procedure is successful among patients in the trial, it will suggest the new approach could open up a way to treat neuroblastoma, and possibly other solid tumors, that currently cannot be cured with standard therapies.
Date Published: 07/08/2009