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CureSearch Announces $450,000 in New Research Funding to Drive Innovative Treatments for Pediatric Neuroblastoma and MPNST

Tessa Gatz, Director of Communications, CureSearch for Children’s Cancer
Phone: (240) 235-2204  |  tessa.gatz@curesearch.org

Bethesda, MD – October 28, 2019 – CureSearch for Children’s Cancer is proud to announce funding for two CureSearch Young Investigator Awards supporting promising new therapies for pediatric neuroblastoma and malignant peripheral nerve sheath tumor (MPNST). Each project will receive $225,000 over three years with the intent to move new therapies quickly into clinical trials within 2-5 years.

Adam Durbin, MD, PhD of the Dana-Farber Cancer Institute will investigate druggable targets in neuroblastoma, a pediatric tumor of the peripheral sympathetic nervous system that impacts nearly 700 children in the U.S. each year. Current neuroblastoma therapies contribute to a survival rate of only 50% and have significant long-term toxicities, including deafness, cardiac insufficiency, infertility and increased risks of additional malignancies.

Dr. Durbin identified a protein, EP300, that is necessary for neuroblastoma growth and worked to develop a novel, bioavailable compound that is able to selectively destroy EP300. If successful, this project will not only offer an innovative treatment strategy for high-risk neuroblastoma, but has the potential to expand into other EP300-dependent pediatric tumors, including myeloid leukemia and rhabdomyosarcoma.

“As both a scientist and pediatric oncologist, I am focused on developing new drugs for children with challenging-to-treat cancers,” said Dr. Durbin. “Funding from CureSearch for Children’s Cancer is critical to the early steps of our work to try to deal with these issues by developing less toxic, more effective therapies for children with cancer.”

Kathryn Lemberg, MD of Johns Hopkins University is pursuing a new treatment strategy for MPNST, an aggressive cancer that is typically seen in adolescence and young adulthood. These tumors can arise in patients with the cancer predisposition syndrome neurofibromatosis type I, without a known cause or as secondary cancers in response to radiation therapy. MPNSTs are incredibly challenging to treat as they often metastasize and do not respond well to chemotherapy or radiation therapy. When incompletely removed at diagnosis, the 4-year event-free survival rate is only 30% and the median overall survival rate in the pediatric population is 30 months.

“Pediatric and young adult patients deserve more effective treatments with fewer side effects than have been classically available for these tumors,” stated Dr. Lemberg. “As a Young Investigator I look forward to collaborating with others in the CureSearch community to successfully develop new medicines for sarcoma.”

Dr. Lemberg’s research aims to exploit a common characteristic of tumor cells to trigger their destruction. It will attempt to block the ability of cells to use glutamine, an essential nutrient needed to survive. If successful in MPNST, the findings of this study have the potential to expand to more than 1,700 kids who are diagnosed in the U.S. each year with pediatric soft tissue sarcomas.

“CureSearch Young Investigators are held to incredibly high standards. They are expected to move potential therapies out of the lab and into the clinic in an accelerated timeframe,” stated Caitlyn Barrett, CureSearch National Director of Research and Programs. “These two researchers are focused on promising therapeutics for deadly pediatric cancers where treatments are currently lacking. Their work over the next three years will propel the therapeutics toward clinical trials and more importantly to where they can reach children”.


About CureSearch for Children’s Cancer

CureSearch for Children’s Cancer, a national nonprofit organization based in Bethesda, MD, works to end childhood cancer by driving targeted and innovative research with measurable results in an accelerated time frame. CureSearch focuses on advancing the strongest research out of the laboratory and into clinical trials and development, where better, less-toxic treatments can quickly help children.

CureSearch Young Investigator Awards support researchers early in their careers to drive transformational science and deliver the next generation of cancer treatments.

For more information, visit curesearch.org, follow CureSearch on Twitter @curesearch or join the conversation on Facebook at facebook.com/curesearch.

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