New First-in-Pediatrics Trial at Children’s Hospital Los Angeles to Test Durvalumab for Certain Cancers

LOS ANGELES–(BUSINESS WIRE)–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,
and LinkedIn,
or visit our blog at


Children’s Hospital Los Angeles
Debra Kain, 323-361-7628