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First Ascent Biomedical Reveals: Precision Medicine Platform Pinpoints Effective Treatments for 83% of Children Facing Tough Cancers

Tuesday, April 23, 2024

First Ascent Biomedical revealed today the outcomes of a pioneering clinical investigation carried out in collaboration with Florida International University (FIU) using an innovative and groundbreaking functional precision medicine (FPM) platform. The study successfully pinpointed specific and effective therapeutic strategies for 83% of the participants suffering from pediatric relapsed or treatment-resistant cancers.

Published in the April edition of the journal Nature Medicine, the study showcased the potential of the company’s FPM platform to revolutionize the treatment landscape for rare, aggressive, or recurrent cancers where targeted therapies are scarce. It marked the first prospective FPM study targeting both liquid and solid tumors in pediatric and adolescent cancers, also representing Nature Medicine's premiere publication of an FPM approach to guiding treatments for relapsed/refractory patients.

Diana Azzam, PhD, Principal Study Investigator and Co-Founder of First Ascent Biomedical, commented, “These results represent a significant leap in personalized medicine for patients, as they expand the utility of FPM by encompassing both liquid and solid tumors, irrespective of cancer type. After a decade of research, this study validates the use of FPM data to guide the next line of therapy for children who have exhausted standard-of-care options. We now offer new hope for those battling difficult-to-treat malignancies.”

Running from 2019 to 2022, the clinical trial feasibility study enrolled 25 pediatric/adolescent patients with recurrent or refractory solid and liquid malignancies who had exhausted standard-of-care options, regardless of cancer type. The primary goal was to generate actionable treatment recommendations from FPM data, comprising drug sensitivity testing (DST) data and/or genomics data, for over 60% of patients enrolled within a clinically actionable time frame of less than two weeks.

The study confirmed the feasibility of furnishing clinicians with a blend of drug sensitivity profiles and molecular data (FPM) to inform subsequent treatment recommendations for relapsed and refractory pediatric patients. The data yielded actionable treatment options for 88% of enrolled patients, with DST results available within a median of nine and ten days for hematological and solid tumors, respectively, enabling clinicians to recommend treatments within a relevant time frame.

Jim Foote, CEO and Co-Founder of First Ascent Biomedical, stated, "The First Ascent FPM platform represents a sophisticated amalgamation of drug sensitivity testing and genomics, scrutinized by our innovative AI-driven technology. These findings signify a true breakthrough, as we managed to provide the vast majority of study participants with a highly personalized and actionable treatment plan. As a father who tragically lost his son to cancer, I yearned for something like this for my child and comprehend the urgency and significance of our work."

In the study, treatments identified were subsequently adjusted with a targeted drug if an actionable genomic mutation was detected. Furthermore, 83% of patients who received FPM-guided treatment exhibited an improved best overall response (Partial Response or better) and a median 8.5-fold increase in progression-free survival (PFS) compared to their previous regimens. Conversely, 13% of patients receiving treatment of physician's choice (TPC) achieved an objective response, consistent with anticipated outcomes for refractory pediatric/adolescent cancers after multiple lines of prior therapy, underscoring the necessity for more refined treatment options.

Nicole de Lara Puente, Chief Executive Officer of Live Like Bella® Childhood Cancer Foundation, remarked, “Childhood cancer stands as the primary disease-related cause of death for children, and Live Like Bella® is honored to have funded this innovative and groundbreaking approach to transform the treatment landscape for children battling cancer. We eagerly anticipate the continued advancement of this work for the betterment of generations of children in the years ahead.”

The First Ascent platform signifies a pioneering integration of advanced drug screening technologies, molecular profiling, and proprietary AI models to broaden therapeutic precision and clinical benefit. First Ascent Biomedical is constructing a commercial lab with expanded capacity, endowed with a Clinical Laboratory Improvement Amendment (CLIA) certification of the FPM system, to enable oncologists and patients nationwide to access this personalized treatment approach.

Noah Berlow, PhD, Study Co-Author and Co-Founder of First Ascent Biomedical, commented, "By melding genomics with functional evidence on the efficacy of drugs against each patient's cancer, we can elevate personalized oncology to unprecedented heights. This groundbreaking platform equips clinicians with the vital insights needed to expedite these children's access to more effective therapies.”

The AI-powered FPM platform could potentially address a substantial unmet need, given that cancer remains the leading cause of disease-related death for both adults and children in the U.S. While survival rates have improved for certain cancers like leukemia, progress has stagnated for high-risk, relapsed, and refractory solid tumors, which often lack actionable genomic drivers and targeted treatments.

First Ascent's innovative technology could pave new avenues for pharmaceutical companies by aiding in the identification of therapeutic biomarkers and combination regimens for expedited drug development against challenging cancers.

In conclusion, the study signifies a major advancement in personalized medicine for pediatric cancer patients, offering new hope and improved treatment outcomes through the innovative integration of functional precision medicine.



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