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Sarcomas – cancers of the body's connective tissue – account for 10-15 percent of all cancers in children. For Carlos Rodriguez-Galindo, MD, and his colleagues at Dana-Farber/Children's Hospital Cancer Center, the key to better treatments for childhood sarcomas lies in the connective relationships of basic and clinical scientists around the world.
Research that bridges multiple investigators and institutions is a necessity for progress against uncommon cancers. In cancers that arise in only a few hundred Americans a year, clinical trials to explore new therapies must be national in scope, so enough patients can participate to produce useful results.
This approach is particularly helpful for pediatric sarcomas, which annually strike about 1,500 children and teens under age 18 in the U.S.
Within those 1,500 cases are more than 30 different types of sarcoma, each with its own characteristics and treatment guidelines. Some of the rarest varieties occur in only a handful of children annually.
For many years, physician-scientists at Dana-Farber and Boston Children's Hospital have collaborated on clinical trials of new pediatric sarcoma treatments through the Children's Oncology Group (COG), a National Cancer Institute-supported consortium of childhood cancer experts across North America, Australia, New Zealand, and Europe. Now, investigators at the two hospitals are developing a series of COG trials that they will lead.
"In the past few years, we've built a corps of young scientists interested in genetic abnormalities that drive and sustain sarcomas," says Rodriguez-Galindo, who heads Dana-Farber's pediatric clinical trials program.
"They have begun to identify specific molecular abnormalities in these cancers that may be targeted by drugs. This has created the opportunity for us to organize and direct trials of novel therapies."
The research program focuses on the three most common categories of sarcomas in children: rhabdomyosarcomas and other soft-tissue sarcomas, which arise in tissues such as muscles, tendons, fat, blood vessels, and nerves; osteosarcomas, which begin in bones; and Ewing's sarcomas, which grow in bones or the soft tissue that surrounds them.
Unlike the organizers of most clinical trials, the Dana-Farber/Children's Hospital Cancer Center team leading new-drug trials for these sarcomas will include the basic scientists who identified the drugs' molecular targets.
"For conditions in which the number of patients is small, you need to maximize your scientific resources," Rodriguez-Galindo says.
"By bringing such a diverse team together, we hope to learn as much as we can from this research."
Learn more about pediatric sarcoma research at Dana-Farber/Children's Hospital Cancer Center.
Paths of Progress Spring/Summer 2012 Table of Contents