31 August 2015
Abu Dhabi - Children's National Medical Center, home of the Sheikh Zayed Institute for Pediatric Surgical Innovation on the Sheikh Zayed Campus for Advanced Pediatric Medicine in Washington, D.C., has discovered a method to cure neuroblastoma tumors in mice. This discovery opens the door for future clinical studies for a therapeutic patient-specific vaccine to cure one of the most common childhood cancers.
Neuroblastomais the third most common tumor in childhood, and the most common cancer in babies younger than one year old.
Anthony Sandler, MD, the Principal Investigator of the Immunology initiative of the Sheikh Zayed Institute for Pediatric Surgical Innovation led the study. Dr. Sandler said: "The work is part of a scientific effort to advance cancer immunotherapy by understanding how cancer cells change their behavior and activate the immune system."
"Historically, tumor vaccines held much promise, but demonstrated little clinical success," Dr. Sandler and his team wrote. "Thus, the task of establishing an effective anti-tumor response in neuroblastoma has been daunting." However, with this most recent study finding, Dr. Sandler says this failed promise is changing.
Peter Kim, MD, CM, PhD, Vice President of the Sheikh Zayed Institute said: "Since the establishment of the Sheikh Zayed Institute,our partnership with thegovernment of Abu Dhabiis helping us reach new heights in the field of pediatric innovation. This research is an embodiment of how together, we can continue to harness the power of innovation to help children in the US, the UAE and around the world."
The study revealed that "knockdown'" of a DNA-protein inhibitor, known as ID-2, in aggressive high-risk solid tumors resulted in activation of T-cells, which are white blood cells that have figured significantly in immunity research. Gene knockdown refers to a technique in which the expression of one or more of a cell's genes is reduced.
The research also focused on using "checkpoint blockade," a therapy in clinical use that allows for expansion of the immune response against tumors. "The combination of selective gene knock-down in tumor cells and checkpoint blockade produced a novel, potent T-cell triggered tumor vaccine strategy," Dr. Sandler says.
As Children's researchers examined the impact of the knockdown of ID-2 protein on a tumor, they implanted N2a, a fast growing mouse neuroblastoma cell line, in the mice. Unexpectedly, Sandler said, "Most of the mice rejected the tumor cells and subsequently were protected against further tumor challenges."
The researchers also noted that a "massive influx" of T-cells infiltrated the shrinking tumor, indicating that T-cells are necessary for antitumor immunity in this vaccination protocol.
The researchers will be working toward potential clinical trials to make further progress in neuroblastoma research, with immunotherapy playing a key role, Dr. Sandler says. He and his team noted that "strategies to circumvent treatment failures are intensively studied and amongst these strategies, immunotherapy has recently been revitalized and is attracting much attention."
About Children's National Medical Center
Children's National Medical Center, based in Washington, DC, has been serving children since 1870. Children's National is Magnet® designated, and is consistently ranked among the top pediatric hospitals by U.S.News & World Report. Home to the Children's Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children's National is one of the nation's top NIH-funded pediatric institutions. With a community-based pediatric network, seven regional outpatient centers, an ambulatory surgery center, two emergency rooms, an acute care hospital, and collaborations throughout the region, Children's National is recognized for its expertise and innovation in pediatric care and as an advocate for all children. For more information, visit ChildrensNational.org, or follow us onFacebookandTwitter.
About the Sheikh Zayed Institute for Pediatric Surgical Innovation
The creation of the Sheikh Zayed Institute for Pediatric Surgical Innovation was made possible by a $150 million (AED 355 million) gift from the government of Abu Dhabi, representing a shared vision to significantly and measurably advance medical innovation to create healthier and safer surgical outcomes for children worldwide. The government of Abu Dhabi made the gift in honor of the late Sheikh Zayed bin Sultan Al Nahyan, the founder of the United Arab Emirates and the country's president from 1971 until his death in 2004.
Launched in September 2009, the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children's National Medical Center is redefining what is possible in surgery through innovative, integrated research. The Institute's physician-scientists apply their expertise in their specialized fields to pursue the common goal to make pediatric surgery more precise, less invasive, and pain-free. By combining research and clinical work in this area, the Institute is developing knowledge, tools, and procedures that will benefit children in the Washington region, across the country, and around the world. For more information about the Sheikh Zayed Institute, please visit http://innovationinstitute.childrensnational.org/
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© Press Release 2015