Stem cell transplant saves Meg from neuroblastoma
Morris
Kletzel, MD, director of the Stem Cell Transplantation Program at Children's
Memorial, can barely stifle his excitement as he talks about the impressive rates
of survival for high-risk neuroblastoma patients that he has seen in a
recently completed clinical trial conducted at Children's Memorial — the first of its kind in the
world. He is joined in his enthusiasm by Scott and Carrie Sanders, whose
daughter, Meg, is alive today because of it.
Neuroblastoma is a tumor of the nervous system that
manifests outside of the nervous system. It typically begins in collections of
nerve tissue, or ganglia, along the spinal cord or in the adrenal glands. Very
often neuroblastoma tumors are categorized as high-risk and resist even the best
treatments known. Typically less than 30 percent of the children with the
high-risk forms survive. Dr.
Kletzel
estimates that 150 to 200
children in the U.S. are diagnosed annually with neuroblastoma. On the average,
six to ten of those children are diagnosed at Children's.
Margaret “Meg”
Sanders was one of these children,
diagnosed when she was just two-and-a-half years old in 1996. That year, on the
morning of September 9, Carrie Sanders was waving good-bye to her two older
children as they boarded the bus for school. As she turned back to her youngest,
Meg, she found the child collapsed from an acute stomach pain. She called her
husband Scott at work, and Carrie rushed Meg to her doctor, Patrick
Gries, MD , a member of Children's Community Physician Association (CCPA) at
Glenbrook Pediatrics. An X-ray revealed what two hours of exams and tests could
not—Meg had a mass “the
size of an orange”
on her
back chest wall. Suspecting the seriousness of the condition, Dr. Gries
immediately scheduled an appointment for Meg with hematologist/oncologists at
Children's Memorial Hospital, where physicians confirmed that the mass was
neuroblastoma.
An innovative stem cell
transplant procedure developed by Dr. Kletzel at Children's is now providing
children with aggressive neuroblastoma, like Meg Sanders, with greatly improved
chances of survival and cure. In September of 1997 Meg was one of the first
children to undergo the triple tandem stem cell rescue that was part of a new
clinical trial. In this regimen, three transplants are performed in succession
rather than the single transplant in the conventional treatment. The new
protocol also involves an intensive treatment strategy that uses virtually every
known modality: four cycles of initial chemotherapy, surgery to remove the
tumor, another rounds of chemotherapy alternating with removal of a patient's
own stem cells for a series of three infusions or transplants. The term "rescue"
is used because the child's own stem cells are removed prior to chemotherapy,
preserved and re-infused during transplant to "rescue" the child's bone marrow
and immune system.
Results of the four-year
clinical trial recently completed by Dr. Kletzel and his team show a remarkable
improvement in a disease with formerly grim statistics. In a few months, all of
the children in the study will pass the statistically significant three-year
mark after therapy. At this time, the new three-year survival rate, which has
stagnated at approximately 25 percent for approximately 15-20 years, officially
reaches an astonishing 75 percent.
"But these success rates
don't come without a cost," says Dr. Kletzel. The toxicity of the intensive
therapy is very high, says Dr. Kletzel, and the children who undergo it become
very sick as a result of the extremely high doses of chemotherapy. Compared to
the conventional treatment, the treatment and recovery period from a triple
tandem procedure is much longer, up to as long as one year.
"Although the
transplantation process was extremely difficult for us, the Children's staff was
terrific," says Carrie Sanders. "They showed us charts and graphs of what Meg
would experience throughout her treatment. They also took our two older
children, Matthew and Anne on a tour of the clinic and explained that Meg would
feel yucky, she might throw-up, and that she would lose her hair, but it would
grow back."
The Sanders family is especially thankful for this new treatment
because today, Meg is disease free. She returns to Children's every four months for tests
to make sure the cancer is still in remission. A
healthy, bounding six-year-old, Meg enjoys playing basketball with her brother and sister,
and still adores the Polly Pockets and Beanie Babies that kept her company
while she was in the hospital.
Whether or not other
centers will be able to duplicate Children's astounding survival rate is unclear
at the moment, says Dr. Kletzel. "Other institutions don't have the kind of
advanced technology that we do, they don't have the same team of care givers,
the same state-of-the-art facilities. It's my hope, though, that as the word
spreads nationwide, overall survival rates will eventually improve."
For other families with
children like Meg, the Sanders family hopes so, too.
Children's Memorial Hospital seeks philanthropic funding to enhance its
programs and services. As a proud partner of the Children's Miracle Network
(CMN), all funds raised in the Chicago area through CMN also benefit Children's
Memorial. To find out how your support can help the hospital better serve
children and families, please contact the Children's Memorial Foundation at
773.880.4237 or Foundation@childrensmemorial.org.