Ranjit Bindra, MD, PhD
Project: Identification of Novel Targeted Agents for Alveolar Rhabdomyosarcoma
Rhabdomyosarcoma is the most common soft tissue sarcoma in children and adolescents. One subtype of rhabdomyosarcoma, called alveolar rhabdomyosarcoma (ARMS), has a particularly poor prognosis. Many children have metastatic disease (cancer that has spread throughout the body) at diagnosis and only 50% of children diagnosed with ARMS will survive five years. Because rhabdomyosarcoma is a rare cancer, there are obstacles to new drug discovery. One of these obstacles is lack of a thorough understanding of the genetic changes in rhabdomyosarcoma tumors.
Ranjit Bindra, MD, PhD, at Yale University, was awarded a CureSearch Young Investigator grant for his work identifying novel targeted drugs for ARMS. Dr. Bindra’s team aims to perform a large-scale screen for drugs that target a particular gene fusion (where genes on two different chromosomes combine to create a new hybrid gene) that leads to rhabdomyosarcoma. Upon completion of the screen, he then confirms the “drug hits” by examining the entire genome of ARMS in order to identify how the drugs impact activity of new targets. Finally, he validates these “hits” in a cell line panel, ensuring that multiple lines are impacted by the drugs in the same way. Dr. Bindra’s research promises to stimulate further drug discovery with the ultimate goal of delivering new medications that can be used to treat ARMS and other rare cancers.
As the result of this project, Dr. Bindra has made significant contributions to the understanding of ARMS:
- 3 novel ARMS mouse models developed
- 1 novel live-cell screening platform to search for selective inhibitors
- 100,000 compounds screened