Mascarenhas Laboratory

Research Topics

  • Acute Lymphoblastic Leukemia
  • Bone and Soft Tissue Tumors
  • Hepatoblastoma
  • Ewing Sarcoma
  • Osteosarcoma

Research Overview

Leo Mascarenhas, MD is director of the Clinical Trials Office in the Cancer and Blood Disease Institute and Principal Investigator for the National Cancer Institute funded Children’s Oncology Group grant at Children’s Hospital Los Angeles. He is an associate professor of Clinical Pediatrics at the Keck School of Medicine of the University of Southern California.

Childhood cancer is the leading cause of disease-related death in pediatrics in the United States. Dr. Mascarenhas and his team's research focuses on developmental therapeutics for patients with bone and soft tissue sarcomas. Despite the remarkable progress made in the treatment of childhood cancer in the last four decades, improved therapies are still needed for our patients with a poor prognosis and less toxic therapies for those destined to survive. This can be accomplished only through basic, translational and clinical research.

Childhood Cancer Research 

Cancer can be caused by changes in our DNA. Scientists have come to understand that cancer may also result from epigenetic changes that are caused by an accumulation of chemical signals that can switch a gene on or off. Epigenetic alterations have been shown to be associated with the development of cancer, drug resistance and disease relapse. Research indicates that these alterations in cancer cells and drug resistance can be overcome using certain medications. The next step is to test these medications in patients.

Key Findings

  • Discovered first epigenetic drug combination used in pediatric AML patients. If effective, this may provide a new strategy to overcome resistance in children with other types of cancer.
  • Created Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL), that is focused on translational research in early phase clinical trials for childhood cancer. The operation center for TACL is located at Children’s Hospital Los Angeles. 


  • National Institute of Health (NIH)