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A Look at The Future of Targeted Therapy

During my medical training 15 years ago, the oncology paradigm was a one-size-fits-all approach for cancer. Chemotherapy drugs were given based on where the cancer originated, without any consideration of the vast molecular diversity of each patient’s cancer. 

Over the past decade, I have seen the development and application of broad molecular testing of cancers, through analysis of DNA, RNA, and proteins to decode individual patient tumors. These tremendous improvements in categorizing and determining the driving forces in an individual’s cancer have coincided with the advancement of new categories of treatment for the millions of cancer patients that cannot be cured with surgery alone. 

“Precision medicine” targets molecular features in cancer cells that are not in normal cells and it is now a focus of drug development.

Modernizing clinical trials

To test precision medicines, the clinical trial system needed to be modernized; the one-trial-for-one-drug-in-one-disease model was inadequate. “Basket” and “umbrella” trial designs allow for multiple drugs to be tested simultaneously in multiple cancer types with unique molecular characteristics, such as DNA mutations, under one master protocol. 

The American Society of Clinical Oncology and the National Cancer Institute (NCI) have taken the lead with large trials (TAPUR and NCI-Match) that have enrolled thousands of cancer patients within the past five years.

I have seen the effects firsthand. This year, I witnessed a man with three prior surgeries and multiple types of chemotherapy respond for the last 14 months to a pill for a rare RET gene fusion in his colon cancer cells. Another man, whose life expectancy was six months in 2015, has been on three different drugs in the TAPUR trial and is thriving five years later. 

These experiences have heightened my gratitude for a new era in cancer care, one in which individualized treatment approaches are on the rise.

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