The American Society of Clinical Oncology (ASCO) wrapped up its annual meeting earlier this month. I was fortunate to attend alongside thousands of scientists, clinicians, and other professionals from around the world. I always return from the conference energized by scientific breakthroughs in oncology research, but this year was particularly exhilarating for the field of precision medicine. Here are my top takeaways from the conference and some insights on how the latest oncology research demonstrates the increasingly important role of genomics in improving patient outcomes.
Wider Application of Commercially Available Therapies for Tumor-Agnostic Use Poised to Extend the Benefits of Precision Medicine to More Patients
Since 2020, the FDA has approved several novel precision medicines targeting specific cancer-causing mutations, regardless of where in the body they originate. This year’s ASCO meeting showed this trend in full swing with the announcement of more validation studies targeting diverse genomic markers across multiple tumor types.
One study confirmed the efficacy of Balversa (erdafitinib), an oral therapy that blocks the activity of fibroblast growth factor receptor (FGFR) signaling proteins in heavily pretreated patients with advanced FGFR-altered solid tumors across 16 distinct cancer types, including glioma, pancreatic, and breast cancer (Link to Abstract). The Phase II RAGNAR trial demonstrated that Balversa (Janssen) resulted in a clinically meaningful response at a median follow-up of 17.9 months. Balversa is currently approved for bladder cancer patients with susceptible FGFR genetic alterations. The potential extended use of this drug is exciting, but even more importantly, the RAGNAR trial represents the largest tumor-agnostic trial of a targeted therapy to date.
Researchers also presented promising treatment results for a range of HER2-expressing solid tumors (Link to Abstract). The ongoing Phase II DESTINY-PanTumor02 trial submitted interim findings that Enhertu (trastuzumab deruxtecan) resulted in encouraging responses and long-lasting clinical benefit in several cancer types, including cervical cancer, endometrial cancer, ovarian cancer, biliary tract cancer, and bladder cancer. Enhertu (AstraZeneca) is currently approved for the treatment of HER-2 expressing breast, lung, and gastric cancers. The new applications for this therapy could help meet a substantial clinical need: many patients with hard-to-treat or advanced disease HER2-positive cancers currently have few or no options.
From these results for Balversa and Enhertu, it’s clear that tumor-agnostic therapies will continue to be a key focus area for drug development. The potential expansion of these treatments demonstrates how more patients benefit by directing precision medicines to all individuals whose tumors express specific biomarkers, regardless of where those tumors are located in the body. In years to come, we’ll see an increase in research on tumor-agnostic applications for commercially available targeted therapies.
Positive Results for NSCLC Treatment Marks a Potential Paradigm Shift for Early-Stage Use of Targeted Therapies
Groundbreaking data from the Phase 3 ADAURA trial presented at ASCO demonstrated that Tagrisso (osimertinib) could cut the death rate in half for early-stage epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) patients who have had their tumors removed. The study showed an estimated 85% of patients treated with Tagrisso (AstraZeneca) were alive at five years compared to 73% on placebo. Tagrisso is currently approved by the FDA and has been used in the clinical setting for years; however, this is the first time positive overall survival data has been presented.
Using precision medicine to treat disease early in its course can mean preventing the disease from spreading to other parts of the body, and fewer side effects for patients. These strong data will change how NSCLC is treated and likely influence an increase in research supporting the early-stage use of targeted therapies across different genomic mutations and cancer types.
The Promising Role of OncRNAs in Early-Stage Disease Detection
Exai Bio announced new data demonstrating the successful blood-based detection of stage I colorectal cancer (Link to Abstract). The next-generation liquid biopsy company uses RNA sequencing to identify orphan non-coding RNAs (oncRNAs) in blood samples. They then run this data through their proprietary machine learning model to accurately predict the cancer tissue of origin. These data added to the growing body of research demonstrating oncRNAs’ critical biological functions.
We look forward to more research interest and studies focused on the role and function of oncRNAs as suitable biomarkers for cancer diagnosis. The practical clinical utility is clear. Compared to traditional biopsy, sampling blood to analyze tumor materials is minimally invasive and may allow for earlier diagnosis.
As sequencing and machine learning technologies continue to advance, there’s a strong potential for even more oncRNAs applications, such as real-time monitoring of disease progression, treatment response, and relapse. We eagerly anticipate the clinical use of new liquid biopsy tests and, more generally, additional research to further identify the biological role of “orphan” RNAs.
Encouraging Results for Low-Grade Glioma Treatment Demonstrate Hope for Diseases with Stagnant Innovation
The Phase 3 INDIGO study presented promising results that targeting isocitrate dehydrogenase (IDH) mutations with vorasidenib significantly delayed tumor growth and the need for toxic chemotherapy for patients with grade 2 gliomas (Link to Abstract). The drugmaker, Servier Pharmaceuticals, announced that they’re working to determine timelines for submission of a New Drug Application (NDA) for vorasidenib to the FDA.
Therapeutic progress for low-grade glioma has been largely stagnant for two decades, making this breakthrough particularly heartening. The announcement was exciting for the thousands of patients who suffer from this disease and for anyone diagnosed with an illness that’s been historically difficult to target with precision medicine. These findings demonstrate that the industry should continue to invest in identifying novel genomic-based treatments, even in diseases for which a scientific breakthrough seems like a long shot. New technological advancements have allowed for great strides in omics biomarker discovery. We’re better poised than ever to increase our understanding of complex disease mechanisms, even for therapeutic areas that haven’t witnessed new treatment options in many years.
The Road Ahead
These findings presented at ASCO signify broad applications for precision medicine research that will impact patient care now and in the future. From a shift towards tumor-agnostic therapies and early-stage targeted treatments to advancements in disease detection and innovative solutions for hard-to-treat disease areas, the potential is immense.
Researchers armed with omics data today are poised to make these types of groundbreaking advancements that will be presented at ASCO in years to come. I can’t wait to return next year and see how additional understanding of genomics and other omics alterations have led to new paradigms in disease diagnosis and treatment, sometimes with previously unimaginable benefits.
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