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VCU Massey Cancer Center researcher Anthony Faber, Ph.D., was awarded a $46,000 grant from the Rally Foundation for Childhood Cancer Research and The Truth 365 to support his research to develop a new therapy for neuroblastoma, a rare type of cancer that develops in infants and young children.

The award will be shared with his research collaborator Yael Mossé, M.D., an attending physician at The Children’s Hospital of Philadelphia Cancer Center and an assistant professor at the Perelman School of Medicine at the University of Pennsylvania, for their research project, “A novel and potent therapy for MYCN-amplified neuroblastoma.” The aim of the project is to further develop the preclinical evidence needed to bring their novel therapy to clinical trials. Their therapy involves a combination of the developmental and orally available drug agents ABT-199 and MLN8237. ABT-199 is known as a selective BCL-2 inhibitor because it selectively binds to and inhibits the BCL-2 protein, which regulates cell death. Also known as alisertib, MLN8237 is currently being tested in clinical trials and is an inhibitor of the Aurora A kinase, whose expression occurs in a wide variety of cancers.  

Preliminary, preclinical data by Faber demonstrate the combination of ABT-199 and MLN8237 to be very effective in treating MYCN-amplified neuroblastoma. In multiple mouse models of MYCN-amplified neuroblastoma, the combination was shown to induce widespread cell death, thereby shrinking cancerous tumors and, in some cases, curing the mice.

MYCN-amplified neuroblastoma is a subset of neuroblastoma in which the expression of the MYCN protein, a member of the MYC family of oncogenes, is amplified. MYCN-amplified neuroblastoma is a particularly difficult to treat and aggressive pediatric cancer. Using a pharmacogenomic approach by which the drug candidates are sought that work specifically in MYCN-amplified cancers, Faber and Mossé have discovered what appears to be a successful, targeted therapy for this form of cancer. 

“We are honored to receive this funding from the Rally Foundation for Childhood Cancer Research and The Truth 365, as we share their passion and commitment to fighting this often deadly pediatric cancer,” said Faber, a member of the Developmental Therapeutics research program at VCU Massey Cancer Center, Blick Scholar and assistant professor at the Philips Institute for Oral Health Research at the VCU School of Dentistry. “We also are excited about the preclinical data and the prospects of bringing this therapy closer to clinical trial testing.”

Funding of this grant is provided by the Rally Foundation for Childhood Cancer Research (Rally), a national non-profit organization that raises awareness and funds for childhood cancer research to find better treatments with fewer long-term side effects and, ultimately, cures. This grant is co-funded with The Truth 365, a non-profit organization that raises awareness and funding for childhood cancer research. Rally and its Medical Advisory Board, consisting of leading childhood cancer researchers from across the nation, award grants through a competitive peer review process. All grants are made in honor or memory of a Rally Kid, a child who has fought or is currently fighting cancer.

“We are absolutely thrilled to support cutting-edge pediatric cancer research,” said Dean Crowe, founder and CEO of Rally. “It is important to fund promising scientists like Drs. Faber and Mossé and research projects like theirs as we work toward closing the childhood cancer research funding gap so that no parent ever has to hear that their child has cancer.”

This grant is part of the $1.7 million Rally will be awarding to 44 hospitals this year. Since its founding in 2005, Rally has distributed more than $7 million to more than 120 childhood cancer research projects nationwide, including basic science, fellowships and clinical trials. 

Written by: Jenny Owen