Nav: Home

Dual stem-cell transplant improves outlook for children with high-risk neuroblastoma

June 05, 2016

CHICAGO (June 5, 2016)- Children with high-risk neuroblastoma whose treatment included two autologous stem-cell transplants were more likely to be free of cancer three years later than patients who underwent a single transplant, a Phase 3 clinical trial has found. The results of the Children's Oncology Group trial, led by investigators at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Seattle Children's Hospital, were presented today at a plenary session at the annual meeting of the American Society of Clinical Oncology. (Abstract LBA3).

The tandem transplant technique, initially developed by researchers at Dana-Farber/Children's in the 1990s, produced even better results when followed by treatment with immunotherapy agents, investigators found.

Three years after completing treatment, 61.8 percent of the participating patients who received two autologous transplants -- transplants using their own stem cells rather than a donor's -- were alive and cancer-free, compared to 48.8 percent of those who underwent a single transplant.

"Our ability to treat children with neuroblastoma has improved significantly over the past 25 years, particularly with the introduction of high-intensity chemotherapy regimens and stem-cell transplantation," says Lisa Diller, MD, chief medical officer of Dana-Farber/Boston Children's and senior author of the study. "The findings of this study define a new standard for the treatment of this disease."

Julie R. Park, MD, of Seattle Children's Hospital, is the study's lead author.

Neuroblastoma is a tumor that begins in nerve cells outside the brain and usually occurs in children under 6 years old. Though rare - with about 700 new cases annually in the United States - it is the second most common pediatric solid tumor and the most common cancer in infancy.

The trial enrolled 652 patients newly diagnosed with high-risk neuroblastoma, the vast majority of whom had Stage 4 (metastatic) disease. The median age of the participants was 3.1 years.

All patients were initially treated with surgery and six cycles of high-dose chemotherapy with multiple drug agents. Blood-forming stem cells were collected after the first two cycles for use in transplantation.

After the last chemotherapy cycle, 355 patients deemed good candidates for transplantation were randomly assigned to receive a single autologous stem-cell transplant with three chemotherapy drugs or a double transplant with a different chemotherapy combination. For patients receiving two transplants, the second was begun about six weeks after completion of the first. Patients received radiation therapy at the site of the initial tumor and, in some cases, the sites of metastases as well.

A subset of patients received immunotherapy after transplant, as participants in a separate clinical trial of dinutuximab, an agent that the federal Food and Drug Administration ultimately approved for use in neuroblastoma in 2015. In those patients, double transplant was also associated with an improved outcome: Among patients who received immunotherapy, 73.7 percent of the double-transplant patients were alive and cancer-free at the three-year mark, compared with 55.5 percent of single-transplant patients.

The investigators found that both event-free survival - survival without recurrence of disease or development of a second cancer -- and overall survival were higher in the two-transplant group, although the increase in overall survival was not statistically significant. The rates of severe acute side effects were similar in both treatment groups.

"As encouraging as these results are, the fact remains that we are treating some of our youngest patients with the most toxic therapies in our arsenal," Diller says. "While we continue to improve survival, we must also seek ways to reduce the toxicity of our treatments and the late effects many of these children will suffer."
Dana-Farber/Boston Children's Cancer and Blood Disorders Center -- the nation's top pediatric cancer center, according to U.S. News & World Report 2015-16. - brings together two internationally known research and teaching institutions that have provided comprehensive care for pediatric oncology and hematology patients since 1947. The Harvard Medical School affiliates share a clinical staff that delivers inpatient care at Boston Children's Hospital and most outpatient care at Dana-Farber Cancer Institute.

Dana-Farber Cancer Institute

Related Stem Cells Articles:

Computer simulations visualize how DNA is recognized to convert cells into stem cells
Researchers of the Hubrecht Institute (KNAW - The Netherlands) and the Max Planck Institute in Münster (Germany) have revealed how an essential protein helps to activate genomic DNA during the conversion of regular adult human cells into stem cells.
First events in stem cells becoming specialized cells needed for organ development
Cell biologists at the University of Toronto shed light on the very first step stem cells go through to turn into the specialized cells that make up organs.
Surprising research result: All immature cells can develop into stem cells
New sensational study conducted at the University of Copenhagen disproves traditional knowledge of stem cell development.
The development of brain stem cells into new nerve cells and why this can lead to cancer
Stem cells are true Jacks-of-all-trades of our bodies, as they can turn into the many different cell types of all organs.
Healthy blood stem cells have as many DNA mutations as leukemic cells
Researchers from the Princess Máxima Center for Pediatric Oncology have shown that the number of mutations in healthy and leukemic blood stem cells does not differ.
New method grows brain cells from stem cells quickly and efficiently
Researchers at Lund University in Sweden have developed a faster method to generate functional brain cells, called astrocytes, from embryonic stem cells.
NUS researchers confine mature cells to turn them into stem cells
Recent research led by Professor G.V. Shivashankar of the Mechanobiology Institute at the National University of Singapore and the FIRC Institute of Molecular Oncology in Italy, has revealed that mature cells can be reprogrammed into re-deployable stem cells without direct genetic modification -- by confining them to a defined geometric space for an extended period of time.
Researchers develop a new method for turning skin cells into pluripotent stem cells
Researchers at the University of Helsinki, Finland, and Karolinska Institutet, Sweden, have for the first time succeeded in converting human skin cells into pluripotent stem cells by activating the cell's own genes.
In mice, stem cells seem to work in fighting obesity! What about stem cells in humans?
This release aims to summarize the available literature in regard to the effect of Mesenchymal Stem Cells transplantation on obesity and related comorbidities from the animal model.
TSRI researchers identify gene responsible for mesenchymal stem cells' stem-ness'
Researchers at The Scripps Research Institute recently published a study in the journal Cell Death and Differentiation identifying factors crucial to mesenchymal stem cell differentiation, providing insight into how these cells should be studied for clinical purposes.
More Stem Cells News and Stem Cells Current Events

Trending Science News

Current Coronavirus (COVID-19) News

Top Science Podcasts

We have hand picked the top science podcasts of 2020.
Now Playing: TED Radio Hour

Listen Again: Reinvention
Change is hard, but it's also an opportunity to discover and reimagine what you thought you knew. From our economy, to music, to even ourselves–this hour TED speakers explore the power of reinvention. Guests include OK Go lead singer Damian Kulash Jr., former college gymnastics coach Valorie Kondos Field, Stockton Mayor Michael Tubbs, and entrepreneur Nick Hanauer.
Now Playing: Science for the People

#562 Superbug to Bedside
By now we're all good and scared about antibiotic resistance, one of the many things coming to get us all. But there's good news, sort of. News antibiotics are coming out! How do they get tested? What does that kind of a trial look like and how does it happen? Host Bethany Brookeshire talks with Matt McCarthy, author of "Superbugs: The Race to Stop an Epidemic", about the ins and outs of testing a new antibiotic in the hospital.
Now Playing: Radiolab

Dispatch 6: Strange Times
Covid has disrupted the most basic routines of our days and nights. But in the middle of a conversation about how to fight the virus, we find a place impervious to the stalled plans and frenetic demands of the outside world. It's a very different kind of front line, where urgent work means moving slow, and time is marked out in tiny pre-planned steps. Then, on a walk through the woods, we consider how the tempo of our lives affects our minds and discover how the beats of biology shape our bodies. This episode was produced with help from Molly Webster and Tracie Hunte. Support Radiolab today at