Study: Precision Medicine Platform Aids 83% of Pediatric Cancer Cases

First Ascent Biomedical, in collaboration with Florida International University (FIU), has unveiled the outcomes of a recent prospective clinical investigation employing an innovative functional precision medicine (FPM) platform. This study has yielded significant findings, indicating that the FPM platform successfully pinpointed specific and efficacious therapeutic choices for 83% of the participants diagnosed with pediatric cancers that had relapsed or exhibited resistance to treatment.

First Ascent Biomedical has recently disclosed the outcomes of a prospective clinical study conducted in collaboration with Florida International University (FIU), showcasing a pioneering functional precision medicine (FPM) platform. This groundbreaking study, published in the April issue of the journal Nature Medicine, underscores the potential of the company’s FPM platform to revolutionize the treatment landscape for rare, aggressive, or recurrent cancers, particularly those with limited targeted therapy options. Notably, this study marks the first prospective FPM investigation targeting both liquid and solid tumors in pediatric and adolescent cancers. Additionally, it represents the debut of Nature Medicine publishing an FPM approach to guide treatments for relapsed/refractory patients.

The principal investigator of the study, Diana Azzam, PhD, and Co-Founder of First Ascent Biomedical, expressed the significance of these findings, emphasizing the study’s role in advancing personalized medicine for patients. The study, conducted from 2019 to 2022, enrolled 25 pediatric/adolescent patients with recurrent or refractory solid and liquid malignancies who had exhausted standard-of-care options. The primary objective was to generate actionable treatment recommendations from FPM data within a clinically actionable timeframe.

Results from the study demonstrated the feasibility of utilizing FPM data to inform subsequent treatment recommendations for relapsed and refractory pediatric patients. Specifically, the FPM platform provided actionable treatment options for 88% of enrolled patients, with drug sensitivity testing (DST) results available within a median of nine and ten days for hematological and solid tumors, respectively.

Jim Foote, CEO and Co-Founder of First Ascent Biomedical, highlighted the significance of the FPM platform in providing highly personalized and actionable treatment plans, citing his personal motivation derived from the loss of his son to cancer. The study also revealed that patients who received FPM-guided treatment experienced improved best overall response and progression-free survival compared to their previous regimens.

Furthermore, the study outcomes underscored the critical need for more refined treatment options for hard-to-treat refractory pediatric/adolescent cancers. Nicole de Lara Puente, CEO of Live Like Bella® Childhood Cancer Foundation, expressed their privilege in funding this innovative approach, emphasizing its potential to transform the treatment landscape for childhood cancer.

The integration of advanced drug screening technologies, molecular profiling, and proprietary AI models in the FPM platform represents a significant advancement in therapeutic precision and clinical benefit. First Ascent Biomedical aims to establish a commercial lab with greater capacity to provide nationwide access to this personalized treatment approach.

Dr. Noah Berlow, Co-Founder of First Ascent Biomedical, emphasized the platform’s potential to elevate personalized oncology by combining genomics with functional evidence on drug efficacy, thus enabling clinicians to tailor treatments to each patient’s unique cancer profile.

In summary, the FPM platform holds promise in addressing the unmet needs in cancer treatment, particularly for patients with high-risk, relapsed, or refractory solid tumors. Its integration of genomic profiling with functional drug testing represents a paradigm shift in personalized oncology, offering new hope for improved treatment outcomes and quality of life for cancer patients.

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