Adopting a functional precision medicine approach to reduce cancer disparities in Hispanic and Black children of Miami

Florida International University, Miami FL

Investigators

Linked publications, trials & patents

Abstract

Modified Abstract Section: FIU-RCMI Research Project #1 ABSTRACT Cancer remains the leading cause of disease-related mortality among children in the United States. Outcomes are particularly poor for pediatric patients with relapsed, refractory, or rare cancers, where five-year survival rates remain dismal and treatment options are limited. Most available therapies are repurposed from adult oncology and lack pediatric-specific efficacy and safety data. These challenges are further compounded by persistent disparities in clinical trial participation and access to advanced therapies: African American children with cancer are significantly underrepresented in clinical research, limiting their access to innovative treatments and contributing to disparities in outcomes. To address these urgent clinical and equity-related needs, we propose to expand a clinically validated Functional Precision Medicine (FPM) program integrated with explainable artificial intelligence (xAI) to optimize individualized, low-toxicity therapies for pediatric patients with relapsed, refractory, or rare cancers who have exhausted standard-of-care options. FPM combines comprehensive genomic and transcriptomic profiling with high-throughput ex vivo drug sensitivity testing (DST) on live patient-derived tumor cultures to directly assess treatment response. Our DST scoring system generates quantitative, actionable data to guide the selection of effective and less toxic therapies that might otherwise be overlooked by genomics alone. Specific Aim 1 will evaluate the clinical utility of FPM in improving outcomes for children with advanced cancers. We hypothesize that integrating FPM with xAI will improve response rates and progression-free survival while reducing treatment-related toxicity. Aim 1A will expand access to FPM-guided therapy through Nicklaus Children’s Hospital in Miami, a clinical site that serves a diverse pediatric population, including African American children. Aim 1B will compare outcomes in children receiving FPM-guided therapy versus those receiving conventional treatment. Specific Aim 2 will elucidate correlations between tumor molecular alterations and ex vivo drug response. We hypothesize that distinct genomic, transcriptomic, and phenotypic properties influence drug sensitivity and resistance. Using multi-omics molecular profiling and an xAI platform, we will identify novel biomarkers predictive of therapeutic response and toxicity. Preliminary studies demonstrate >80% DST success rates and >65% actionable findings, with strong correlation to clinical benefit. This integrative FPM-xAI platform represents a scalable, logistically feasible strategy to personalize therapy, reduce treatment-related toxicities, and improve outcomes in pediatric oncology. Importantly, it offers a pathway to broaden access to innovative cancer care for African American children and others historically excluded from precision oncology research.