New Precision Medicine Trial Opens for 20 High-Risk Neuroblastoma Patients

Spectrum Health Helen DeVos Children’s Hospital announces a new, multicenter clinical trial through the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC) in partnership with the Translational Genomics Research Institute (TGen). Made possible by a grant from Dell, this study will enroll twenty newly diagnosed children with high-risk neuroblastoma.

This groundbreaking trial is the first study to rely on massive computing resources to help integrate comprehensive genomic information at diagnosis into targeted therapy identification early in the patient’s course of treatment. In addition, the targeted investigational drug DFMO will then be used following treatment to prevent relapse.

Neuroblastoma is an aggressive childhood cancer that occurs in children less than 6 years of age. The first challenge is that 20 percent of children are unable to attain remission. The primary goal of this study is to evaluate the feasibility of using molecular guided therapy in combination with standard chemotherapy to increase response. The second challenge in neuroblastoma is that approximately 40 percent of children relapse. The second goal is to follow upfront chemotherapy with maintenance therapy using DFMO to prevent relapse in patients with newly diagnosed high-risk neuroblastoma. If this pilot of 20 patients is successful, the NMTRC will further explore this as a new way of treating children with neuroblastoma with the hope of increasing overall survival.

“We are proud to partner with Dell and TGen to bring forward a deeper understanding of each patients tumor at diagnosis as we further develop our personalized medicine program”, said “Dr. Giselle Sholler, chair of the NMTRC. “Our study aims to identify the pathways driving each patient’s tumor. This understanding can help us match a targeted therapy to a patient’s tumor. We have shown in previous studies that DFMO targets important cancer stem cell pathways and therefore we have designed this new trial to add this drug as maintenance therapy to try to prevent children from relapsing.”

“The power of clinical trials such as this one allows potentially groundbreaking questions to be answered in a clinically relevant setting,” said Dr. Jeffrey Trent, TGen President and Research Director. “This trial hopefully will set the path for additional studies that look to positively impact the field of Neuroblastoma.”

“The work that Dr. Sholler and her colleagues are doing to treat pediatric cancer at the NMTRC is absolutely inspiring, “said Trisa Thompson, Vice President and Chief Responsibility Officer at Dell. “We are proud that our technology and charitable grants are fueling the team’s genomic research and helping address some of the toughest cancer battles facing children around the world.”

The NMTRC is a group of 25 universities and children’s hospitals headquartered at the Helen Devos Children’s Hospital offering a nationwide network of childhood cancer clinical trials. The following is a list of the NMTRC enrolling sites for this specific trial:

Helen DeVos Children’s Hospital in Grand Rapids, MI
Cardinal Glennon Children’s Medical Center in St. Louis, MO
Rady Children’s Hospital in San Diego, CA
Arnold Palmer Hospital for Children, Orlando, FL
Medical University of South Carolina, Charleston, SC
Levine Children’s Hospital, Charlotte, NC
Connecticut Children’s Medical Center, Hartford, CT
Dell Children’s Blood and Cancer Center, Austin, TX
Children’s Hospitals and Clinics of Minnesota, Minneapolis, MN
Kapi’olani Medical Center, Honolulu, HI
Penn State Milton S. Hershey Medical Center & Children’s Hospital, Hershey, PA
Arkansas Children’s Hospital, Little Rock, AR

To participate in this study, please contact:

Study Chair
Giselle Sholler, MD
(o): 616-267-0332
Giselle.sholler@helendevoschildrens.org

Program Manager
Genevieve Bergendahl, RN
Genevieve.bergendahl@helendevoschildrens.org
(o): 616-267-0335

About Spectrum Health
Spectrum Health is a not-for-profit health system, based in West Michigan, offering a full continuum of care through the Spectrum Health Hospital Group, which is comprised of 12 hospitals, including Helen DeVos Children’s Hospital; 183 ambulatory and service sites; more than 3,100 physicians and advanced practice providers, including 1,200 members of the Spectrum Health Medical Group; and Priority Health, a health plan with about 654,000 members. Spectrum Health is West Michigan’s largest employer, with 23,400 employees. The organization provided $283 million in community benefit during its 2015 fiscal year. Spectrum Health is the only health system in Michigan to be named one of the nation’s 15 Top Health Systems® by Truven Health Analytics for 2015. This is the fourth time the organization has received this recognition.

About NMTRC
The Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC) is a group of 25 universities and children’s hospitals headquartered at the Helen Devos Children’s Hospital that offer a nationwide network of childhood cancer clinical trials. These trials are based on the research from a group of closely collaborating investigators who are linked with laboratory programs developing novel therapies for high-risk neuroblastoma and medulloblastoma. The NMTRC’s mission is to create a national collaborative effort of researchers, oncologists and family advocates to bring forward new therapies for children with relapsed neuroblastoma and medulloblastoma with the goal of improving the quality of life and survival of children with neuroblastoma and medulloblastoma. For more information, visit: www.nmtrc.org. Follow NMTRC on Facebook and Twitter @NMTRC.

About TGen
Translational Genomics Research Institute (TGen) is a Phoenix, Arizona-based non-profit organization dedicated to conducting groundbreaking research with life changing results. TGen is focused on helping patients with neurological disorders, cancer, and diabetes, through cutting edge translational research (the process of rapidly moving research towards patient benefit). TGen physicians and scientists work to unravel the genetic components of both common and rare complex diseases in adults and children. Working with collaborators in the scientific and medical communities literally worldwide, TGen makes a substantial contribution to help our patients through efficiency and effectiveness of the translational process. For more information, visit: www.tgen.org. Follow TGen on Facebook, LinkedIn and Twitter @TGen.