Functional precision medicine, which uses direct analysis of tumor cells to guide personalized therapy, is showing promise in treating pediatric cancers. A recent study explored the feasibility of this approach in children with relapsed or refractory cancers, revealing encouraging results.
Study Details and Findings
Diana J. Azzam, PhD, and her team conducted a small, observational study to assess the feasibility of functional precision medicine in guiding treatment for pediatric patients with relapsed or refractory cancers. The study enrolled 25 patients with various solid and hematologic cancers who had exhausted standard-of-care options. The median age of the patients was 10 years, and they had undergone between two and six prior lines of therapy.
The researchers performed drug sensitivity testing on patient-derived tumor cells from 21 patients and genomic profiling on 20 patients. The drug sensitivity testing involved exposing the tumor cells to up to 125 FDA-approved drugs to determine which treatments were most effective. The median turnaround times for drug sensitivity testing and genomics testing were 10 days and 27 days, respectively.
Of the patients, 19 received treatment recommendations based on the functional precision medicine approach, and 14 subsequently received therapeutic interventions. The study found that 83% of patients who received functionally guided treatments experienced an improvement in progression-free survival greater than 1.3-fold compared to their previous treatment. Additionally, 83% of patients achieved a significant increase in objective response rate compared with their previous treatment regimen, versus 13% of patients in the physician’s choice treatment cohort.
The Role of Functional Precision Medicine
Functional precision medicine integrates genomics molecular profiling with drug sensitivity testing. It involves testing numerous FDA-approved drugs directly on a patient’s own living cancer cells to identify the most effective treatments. This approach contrasts with traditional precision medicine, which primarily relies on DNA analysis or genomics to identify tumor mutations and then seeks matched treatments for those mutations.
Overcoming Challenges and Future Directions
Despite the potential benefits, several barriers exist to the widespread adoption of functional precision medicine. One significant challenge is physician hesitancy in using this method for treatment decision-making. Additionally, obtaining insurance coverage for this testing is a hurdle. To address these challenges, more research is needed to demonstrate the clinical utility and cost-effectiveness of functional precision medicine.
The next steps in research involve conducting larger, randomized, multicenter interventional clinical trials to compare the clinical utility of functional precision medicine with other approaches. Researchers are also working on getting more functional precision medicine assays certified, which will pave the way for FDA approval.
Integrating AI for Personalized Cancer Care
Artificial intelligence (AI) is expected to play a crucial role in advancing functional precision medicine. By integrating functional precision medicine, genomics, and AI, researchers aim to accurately identify novel biomarkers and match them with effective drugs and predict combinations for individual patients based on their tumor’s genomics and DNA sequencing. The more data generated, the better AI algorithms can be trained to personalize cancer care.
