New Phase 1 clinical trial to test Durvalumab in pediatric patients


In an innovative, first-in-pediatrics study, available only at Children's Hospital Los Angeles (CHLA), researchers will be enrolling children between 1 and 18 years of age who have certain types of relapsed or treatment-resistant cancer.

The aim of this investigator-initiated Phase 1 trial is to test the safety, tolerability and metabolism of the drug Durvalumab in pediatric patients with solid tumors, lymphoma and central nervous system tumors.

Durvalumab, which is also being investigated in several types of adult cancer, is what is known as a checkpoint inhibitor. A "checkpoint" is a molecule that is activated or inactivated to allow the body's immune system to attack foreign cells while leaving normal cells alone. Cancer cells often find ways to evade these checkpoints to avoid being attacked by the immune system.

Drugs like Durvalumab can target such checkpoints and boost the immune response against cancer cells.

PD-1 is a checkpoint protein found on T cells which normally signals the T cell to turn off its immune response. When PD-1 attached to another protein – PD-L1 – that is found on cancer cells, it allows the cancer to evade immune attack.

"By shutting off the signal by inhibiting PDL-1, Durvalumab in essence allows the body's own T cells to recognize the cancerous cells as 'foreign' and attack them," said Leo Mascarenhas, MD, MS, deputy director and head of oncology at CHLA's Children's Center for Cancer and Blood Diseases. Mascarenhas and Josephine HaDuong, MD, co-lead investigators of the study, seek to enroll between 18 and 24 young patients with cancer.

"This is an example of how Children's Hospital Los Angeles offers the newest, cutting-edge treatments to our patients," says Mascarenhas.


This research is being conducted with support from AstraZeneca Pharmaceuticals LP.

The Children's Center for Cancer and Blood Diseases (CCCBD) at Children's Hospital Los Angeles has been ranked by U.S. News and World Report among the top 10 pediatric cancer centers in the country since these rankings began in 2008. It has one of the largest programs of clinical trials for children with cancer and blood disorders in the U.S., enrolling nearly 1,400 children in more than 140 different trials each year. The CCCBD is part of the 20-member Pediatric Phase 1 Pilot Consortium, funded by the National Cancer Institute to improve therapy outcomes for children with cancer.

About Children's Hospital Los Angeles

Children's Hospital Los Angeles has been named the best children's hospital in California and among the top 10 in the nation for clinical excellence with its selection to the prestigious U.S. News & World Report Honor Roll. Children's Hospital is home to The Saban Research Institute, one of the largest and most productive pediatric research facilities in the United States. Children's Hospital is also one of America's premier teaching hospitals through its affiliation since 1932 with the Keck School of Medicine of the University of Southern California. For more information, visit Follow us on Twitter, Facebook, YouTube and LinkedIn, or visit our blog at

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Debra Kain
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