Human-Based Tumor Model Shows Promise for Personalized Cancer Treatment

More than 2 million people are diagnosed with cancer every year in the U.S. Even among patients with the same diagnosis, the disease can vary greatly in terms of symptoms, treatment responses, and outcomes. This variation reveals a significant need for personalized medicine, and addressing this need requires better research models that more accurately capture the complexity of the disease.

Species-specific differences limit the extent to which animal experiments can effectively model human biology. Key factors, like genetics, tumor environment, and tumor development, differ across mice, monkeys, and humans. These limitations become particularly pronounced when attempting to model the complexity of an individual patient’s cancer. Personalized, human-based research approaches are powerful innovations capable of overcoming these limitations to ultimately improve treatment success.

In a promising new study, researchers grew miniature clusters of tumor cells called organoids from patient tumor samples. To ensure their approach would be versatile enough to work across many different patients and cancers, researchers developed the organoids from 191 patients with 15 cancer types. Compared with the patients' original tumors across multiple biological characteristics, the organoids showed a high degree of similarity, suggesting that the personalized tumor-derived organoids reliably replicated patients’ cancers and could help predict individual treatment responses.

Researchers then demonstrated practical application of the model for precision oncology. They derived a subset of the organoids from patients deemed ineligible for treatment with a class of drugs called PARP inhibitors, which are typically only covered by insurance for a narrow set of FDA-approved indications, leaving many patients who fall outside this coverage criteria to pay tens of thousands of dollars out of pocket every month. In testing those organoids for sensitivity to a PARP inhibitor, more than half showed a positive response, suggesting that some patients could benefit from treatments they might not otherwise be eligible for, and opening the door for expanded clinical criteria that would help alleviate financial burdens.

Highlighting another potential use, Dr. Juan Miguel Mosquera, one of the study's authors, explained, “you can use these organoids as patient ‘avatars’ during clinical trials of experimental therapies, for example, to get an early picture of treatment effects and side effects.”

Another author, Dr. M. Laura Martin, added, “The main takeaway from these studies is that patient-derived tumor organoids are becoming very useful tools for precision oncology.” In addition to advancing personalized cancer treatment, patient-derived organoid models like this one also help reduce animal experimentation, improving outcomes for humans and animals alike.