Despite many efforts to find better, more effective ways to treat cancer, it remains a leading cause of death by disease among children in the U.S.
Cancer patients are also getting younger. Cancer diagnoses among those under 50 has risen by about 80% worldwide over the past 30 years. As of 2023, cancer is the second-leading cause of death both in the U.S. and around the world. While death rates from cancer have decreased over the past few decades, about 1 in 3 patients in the U.S. and 1 in 2 patients worldwide still die from cancer.
Despite advances in standard cancer treatments, many cancer patients still face uncertain outcomes when these treatments prove ineffective. Depending on the stage and location of the cancer and the patient’s medical history, most cancer types are treated with a mix of radiation, surgery and drugs. But if those standard treatments fail, patients and doctors enter a trial-and-error maze where effective treatments become difficult to predict because of limited information on the patient’s cancer.
My mission as a cancer researcher is to build a personalized guide of the most effective drugs for every cancer patient. My team and I do this by testing different medications on a patient’s own cancer cells before administering treatment, tailoring therapies that are most likely to selectively kill tumors while minimizing toxic effects.
In our newly published results of the first clinical trial combining drug sensitivity testing with DNA testing to identify effective treatments in children with cancer, an approach called functional precision medicine, we found this approach can help match patients with more FDA-approved treatment options and significantly improve outcomes.
What is functional precision medicine?
Even though two people with the same cancer might get the same medicine, they can have very different outcomes. Because each patient’s tumor is unique, it can be challenging to know which treatment works best.
To solve this problem, doctors analyze DNA mutations in the patient’s tumor, blood or saliva to match cancer medicines to patients. This approach is called precision medicine. However, the relationship between cancer DNA and how effective medicines will be against them is very complex. Matching medications to patients based on a single mutation overlooks other genetic and nongenetic mechanisms that influence how cells respond to drugs.
Functional precision medicine involves testing drugs on tumor samples to see which ones work best.
How to best match medicines to patients through DNA is still a major challenge. Overall, only 10% of cancer patients experience a clinical benefit from treatments matched to tumor DNA mutations.
Functional precision medicine takes a different approach to personalizing treatments. My team and I take a sample of a patient’s cancer cells from a biopsy, grow the cells in the lab and…
