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Researchers Win Grants to Explore New Drug Targets for Childhood Cancers

Published on September 29, 2017 in Cornerstone Blog · Last updated 11 months 1 week ago


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National Childhood Cancer Awareness Month is coming to a close, but new research endeavors are continuing the momentum and driving toward better treatment options. Hyundai Hope On Wheels awarded two pediatric cancer investigators at Children’s Hospital of Philadelphia research grants during a Celebration of Life Gala held Sept. 27 in Philadelphia.

Pediatric oncologist Kathrin Bernt, MD, received a Hyundai Scholar Hope Grant, and pediatric oncologist Kristopher Bosse, MD, received a Hyundai Young Investigator grant. Actress Jennifer Freeman hosted the awards event, and recording artist Luke Wade entertained the audience of pediatric cancer experts, young cancer survivors and their families, and Hyundai executives and dealership owners.

Dr. Bernt’s research project focuses on a common subtype of the blood cancer acute myeloid leukemia (AML) called inv(16). Long-term survival of inv(16) AML, which is considered a “good-risk subtype,” is still inferior to acute lymphoblastic leukemia (ALL), the other common subtype of leukemia in children. New therapeutic approaches are urgently needed, but designing a drug that inhibits inv(16), which is an inversion of chromosome 16, is extremely difficult. So Dr. Bernt is looking at two neighboring “helper” proteins, MN1 and DOT1 L, that inv(16) may rely on to cause leukemia. An inhibitor of DOT1 L is already in clinical trials, and if researchers can confirm that DOT1 L plays a major role in inv(16) AML, that drug could be a valuable new DOT1 L directed epigenetic therapy.

“Because inv(16) is considered to have a lower risk of relapse than other subtypes, it is not as intensely studied as other types of leukemia,” Dr. Bernt said. “Yet outcomes are still nowhere near as good as for ALL. I am very excited to be working on a project that will help us to better understand this subtype and find new therapeutic approaches.”

Dr. Bosse is investigating the development of new targeted immunotherapeutics in high-risk neuroblastoma, a cancer of the developing peripheral nervous system that is the most common cancer in infants. In a cover story that recently appeared in the journal Cancer Cell, Dr. Bosse described how he and colleagues working in the lab of John Maris, MD, at CHOP pinpointed a cell-surface protein called glypican-2, or GPC2, that is selectively present on neuroblastoma cells and is necessary for a neuroblastoma tumor to grow aggressively. Their findings suggest that GPC2 is a promising target for immunotherapy. With the help of his young investigator grant, Dr. Bosse plans to continue to engineer proteins that seek out GPC2 on neuroblastoma cells and deliver potent drugs to kill cancer cells and spare healthy cells.

“The development of new safe and effective targeted immunotherapies for childhood cancers is urgently needed, and with this HHOW YI funding, we are working towards this goal for children with neuroblastoma,” Dr. Bosse said.

In September, Hyundai Hope On Wheels awarded 40 research grants, totaling $8.5 million, to children’s hospitals across the country participating in the Children’s Oncology Group (COG), which is coordinated by COG Chairman Peter Adamson, MD, a CHOP pediatric oncologist. COG member institutions care for more than 90 percent of the 14,000 children and adolescents who are diagnosed with cancer each year in the U.S. For more information on Hyundai Hope On Wheels’ highly competitive grant program and how to apply, visit here.