In recent years, the U.S. Food and Drug Administration has adopted public policies designed to reinforce cancer care’s shift toward a more effective and efficient era of personalized medicine. In 2020, these policies helped drive the approval of nine personalized cancer treatments that provide new hope for patients and more efficiency for health systems.
Personalized medicine is based on scientific discoveries showing that every patient’s cancer — like every person — is genetically unique. Guided by these findings, physicians can use diagnostic tests to determine which treatments will work best for each patient.
A gene is the basic physical unit of inheritance. Genes are passed from parents to offspring and contain the information needed to specify traits.
Most personalized cancer treatments are targeted therapies that disrupt the function of proteins fueling the growth of cancer cells. By inhibiting the activities of genes known to drive cancer, these therapies are designed to destroy cancerous cells without affecting healthy ones.
The eight targeted therapies the FDA approved in 2020 provide new treatment options for genetically defined subsets of patients with breast, lung, and thyroid cancers, as well as cancers of the gastrointestinal tract and the bile ducts, which connect the liver to the small intestine.
CAR T-cell therapies
Scientists are also developing ways to genetically re-engineer a patient’s immune cells so they can recognize and attack blood cancers. Chimeric antigen receptor (CAR) T-cell therapy is a form of immunotherapy that uses specially altered T-cells to kill cancers. In 2017, the FDA approved CAR T-cell treatments for acute lymphoblastic leukemia and diffuse large B-cell lymphoma. In 2020, the agency approved another CAR T-cell therapy for mantle cell lymphoma. The new approval expands the horizons of CAR T-cell therapies, which scientists hope will have a tremendous impact on care for patients with many types of cancer.
Scientists are also developing ways to genetically re-engineer a patient’s immune cells so they can recognize and attack blood cancers.
A new era
These newly approved treatments add to the growing list of available personalized medicines, which now account for one of every four drugs approved since 2014. Personalized treatments help physicians improve care for patients and health systems by targeting the right drug to the right patient at the right time.