What is precision medicine?
Precision medicine is a tailored approach to treatments for human diseases based on the characteristics of each individual. For cancer patients, this means using treatments tailored to the molecular abnormalities and characteristics of a tumor. Tumors can stem from the same genetic mutations, and be susceptible to the same drugs, even if they affect different sites in the body.
Therapies developed to target a specific molecular abnormality, called targeted therapies, are more precise and often less toxic than traditional treatments such as chemotherapy and radiotherapy. The terms “molecularly targeted drugs,” “precision medicines,” or “personalized medicine” are sometimes used interchangeably.
What is genomic sequencing?
Sequencing means determining the order of the four chemical building blocks that make up the DNA molecule. When a tumor is sequenced, the DNA in tumor cells will show a mutation in a gene or a group of genes. Targeted therapies work against these mutations, fighting the diseased cells. Sequencing is a step needed for determining targeted therapies.
How can children benefit from precision medicine?
Research shows that patients whose treatment was selected based on the molecular characteristics of their tumor had significantly better outcomes. (See a recent study presented in June 2016 at the annual meeting of American Society of Clinical Oncology).
Every year about 16,000 children under the age of 19 are diagnosed with cancer in the U.S., and nearly 2,000 kids die. The precision medicine approach in pediatrics tries to identify targeted therapy options for the 20% of patients who are not cured using standard treatments. It also has the potential to improve outcomes for cancer survivors, since two-thirds of all survivors suffer from long-term toxicities.
Genomic sequencing can also indicate which therapies will not work or are inappropriate for particular patients.
Can the knowledge gained from using precision medicine in adult cancers benefit kids with cancers?
The number of FDA-approved molecularly targeted cancer therapies is approaching 100, and there are about 900 cancer drugs in development, mostly for adult cancers. In many cases, the abnormalities that cause pediatric cancers have similar counterparts in adult cancers. This makes it possible in some cases to redirect therapies that were developed for adult indications to pediatric uses.
See infographics explaining precision medicine.
Based on AACR interview with Andrew Kung, MD, PhD, professor of pediatrics and chief of the Division of Pediatric Hematology, Oncology, and Stem Cell Transplantation at New York-Presbyterian/Morgan Stanley Children’s Hospital and Columbia University Medical Center. Read the full interview.
Not enrolling yet:
Pediatric MATCH (Molecular Analysis for Therapy Choice)
Sponsor: Children’s Oncology Group
Acute Lymphoblastic Leukemia Therapies Informed by Genomic Analyses
Investigator: Jodi Mayfield
Sponsor: New Mexico Cancer Care Alliance
Matched Targeted Therapy For High-Risk Leukemias
Investigator: Yana Pikman
Sponsor: Dana-Farber Cancer Institute
X01 Projects for the Gabriella Miller Kids First Program: An Integrated Clinical and Genomic Analysis of Treatment Failure in Pediatric Osteosarcoma
Investigator: Kenan Onel
Sponsor: University of Chicago
X01 Projects for the Gabriella Miller Kids First Program: Genetic Contribution to Ewing Sarcoma in 330 parent-Offspring Trios
Investigator: Joshua D. Schiffman
Sponsor: University of Utah
Ages not specified:
Exploring Precision Cancer Medicine for Sarcoma and Rare Cancers (2013-2017), MI-ONCOSEQ
Investigator: Arul Chinnaiyan, Scott Roberts
Sponsor: University of Michigan
Tumor Genomic Profiling in Patients Evaluated for Targeted Cancer Therapy
Investigator: David Hyman
Sponsor: Memorial Sloan Kettering Cancer Center
UNC Chapel Hill
Rutgers Cancer Institute of New Jersey (UMDNJ)
Cincinnati Children’s Hospital Medical Center
Children’s Medical Center (Dallas TX)
Memorial Sloan-Kettering Cancer Center
Rady Children’s Hospital of San Diego
Children’s Hospital of Philadelphia – Center for Childhood Cancer Research
Children’s Hospital of Alabama
University of Iowa Hospitals and Clinic Cancer Center