One-size-fits-all drugs are giving way to treatments tailored to individuals’ genetic makeups.
When the Food and Drug Administration recently a promising new lung cancer drug named Zykadia four months ahead of schedule, it heralded the medication as a “breakthrough” therapy.
The drug isn’t meant for everyone with the devastating disease, which kills an estimated 160,000 Americans each year. Or even for the majority of patients with its most common form, non-small-cell lung cancer.
Rather, Zykadia is designed for a sliver of patients — about 5 percent — who have advanced non-small-cell lung cancer and have a specific gene mutation that causes tumors to become resistant to existing treatment. For them, and only them, the drug has the proven potential to shrink tumors and extend lives.
The FDA’s speedy approval of Zykadia offered the latest evidence that the age of “personalized medicine,” while long predicted, is increasingly becoming reality. For reasons scientific and economic, one-size-fits-all blockbuster drugs are giving way to treatments tailored to individuals’ genetic makeups and aimed at narrow subsets of broader diseases.
“It’s a new world,” said Wendy Selig, president of the Melanoma Research Alliance, the largest private funder of research on the disease, which this year is expected to kill nearly 10,000 Americans. “We’re segmenting what we thought of as large diseases into smaller populations of patients that are defined by genetic distinctions. . . . The goal is to match the right therapy to the right patient, and to do it with minimal collateral damage.”
Since 2011, the FDA has approved numerous new treatments for melanoma patients with certain types of genetic mutations. The agency also has given the green light to many drugs for other specific cancers, and to a revolutionary treatment for a small proportion of people with cystic fibrosis. Companion diagnostic tests often help identify which patients might benefit from the targeted treatmen