Looking Back on the Year of Precision Medicine

In 2016, one million or more volunteers may begin enrolling in one of the largest long-term medical research studies ever planned. It aims to inform future therapies targeted to the molecular, environmental, and behavioral factors that contribute to diverse diseases.

While this ambitious national cohort program, a key component of the Precision Medicine Initiative (PMI), looms large in the new year ahead, it is clear from a look back that precision medicine was already one of the biggest, most-discussed trends in medical research in 2015. The Children’s Hospital of Philadelphia remains committed to this approach, and several projects exemplify many of the year’s achievements in precision medicine.

An article in the year-end issue of the CHOP Research Institute newsletter Bench to Bedside highlights molecular targeted therapies in cancer, an area where precision medicine is most advanced. The successes and the remaining challenges map onto a few key themes:

• Making genomic discoveries is a vital first step toward identifying targets for precision treatments — and CHOP researchers are doing so at a rapid pace.
• Tracking and responding to the evolution of disease offers new insights into targets for treating relapsed disease. CHOP investigators published major studies this year showing genetic changes in leukemia and neuroblastoma as the cancer develops resistance to treatment and ultimately relapses. They anticipate more such studies in the future.
• Identifying existing drugs that target a disease-associated pathway is one of the fastest potential approaches to finding precision treatments. This approach showed preliminary success in a CHOP-involved study of patients in a molecular subgroup of medulloblastoma (brain cancer).
• Accelerating targeted drug discovery through preclinical testing is essential to translate genomic discoveries into targeted therapies. CHOP’s Pediatric Preclinical Testing Consortium launched by the National Cancer Institute will help researchers identify drug candidates for pediatric clinical trials.
• Creating and implementing innovative models for clinical trials is key to testing precision treatments in patients who are increasingly subdivided into smaller populations by disease types. Trials launching in 2016, Project:EveryChild Pediatric MATCH (for multiple advanced cancers) and NEPENTHE (for relapsed neuroblastoma) will target investigational treatments to the genetic abnormalities in individual patients’ cancers.