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Precision Medicine for Pediatric Cancer

Friday, March 15th 2019 at 6:57pm UTC

Considering the implications for diagnosis and treatment

LOS ANGELES–(BUSINESS WIRE)– Research performed over the last several decades has led to an increased
understanding of the genetics of cancer. The clinical application of
this knowledge for pediatric cancer has lagged behind studies performed
for adults. In a perspectives article published in the prestigious
journal Science,
Dr. Jaclyn Biegel, from Children’s Hospital Los Angeles, and Dr.
Alejandro Sweet-Cordero, of the University of California, San Francisco,
survey the landscape of this young field and present opportunities for
using genomic information to advance a new era of care for children with
cancer.

Cancer arises from genetic changes, including DNA mutations, that are
either present at birth, or are acquired over time. Many adult cancers
are initiated by mutations acquired through exposure to substances like
smoking and radiation or simply from aging. The tumors may contain
hundreds of sequence alterations, and identifying which changes drive
the growth of the tumors and impact treatment response can be
challenging. In contrast, pediatric malignancies often develop from a
very small number of mutations, only some of which overlap with the
types of mutations seen in adult cancers. Furthermore, an estimated 20%
of pediatric cancers arise in children who have a genetic predisposition
to malignancy. For this reason, the clinical genetic assays developed to
inform prognosis and treatment decisions for adult cancers have not been
as useful in pediatrics.

OncoKids®
was one of the first next-generation sequencing panels to detect DNA and
RNA changes that characterize pediatric cancers. The panel was developed
at Children’s Hospital Los Angeles under the guidance of author Jaclyn
Biegel, PhD, FACMG
, Director of CHLA’s Center
for Personalized Medicine
. The OncoKids® panel provides a molecular
diagnosis, informs prognosis, and highlights novel therapeutic targets
across the broad spectrum of cancers in children, including leukemias,
brain tumors and other solid tumors.

“To truly achieve personalized medicine in pediatric oncology, we need
to be able to determine if a child is genetically predisposed to develop
cancer,” said Dr. Biegel. In addition to tumor testing, germline
testing
that uses a sample of a patient’s blood, is critical
for identifying those children who have a genetic risk for developing
cancer in the future. Besides benefiting the patient, this information
has implications for the entire family, since an abnormality that is
passed down from parent to child can also raise the risk of developing
cancer in siblings.

Although tremendous progress has been made in pediatric cancer care,
treatment resistant disease and relapse continue to negatively impact
patient outcomes. Genetic profiling of pediatric cancers is typically
done at the time of diagnosis or at the time of relapse to help
determine treatment planning. According to Dr. Biegel, future studies
that may be performed over the course of treatment and at remission have
the potential to provide critical information about the mechanisms of
tumor progression, treatment resistance and metastasis.

Tremendous opportunity exists for changing outcomes in children with
cancer by using an integrated approach to evaluating children and their
families that includes genomic medicine as a central component in their
care.

About Children’s Hospital Los Angeles

Children’s Hospital Los Angeles has been ranked the top children’s
hospital in California and sixth in the nation for clinical excellence
by the prestigious U.S. News & World Report Honor Roll. The
Saban Research Institute at CHLA is one of the largest and most
productive pediatric research facilities in the United States. CHLA also
is 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, visit CHLA.org,
the child
health blog
and the research
blog
.

Contacts

Media contact:
Ellin Kavanagh
ekavanagh@chla.usc.edu
(323)
361-8505

Source: Children’s Hospital Los Angeles


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