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Targeted Therapy: Advancing Oncology Care with Precision and Purpose

Chumi Khurana, Senior Vice President, Oncology Franchise Head

Precision science is changing what is possible in oncology, and nowhere is that more meaningful than in rare cancers, where patients have historically had the fewest options and the stakes are highest.

For too long, patients with rare cancers have faced a difficult reality of too few treatment options, too little scientific attention, and too much uncertainty. That is changing, and the shift is rooted in something fundamental, a move away from treating tumors by where they originate and toward understanding the biology driving them.

This evolution is not new. Targeted approaches such as HER2-directed therapies have been shaping oncology for over 20 years but the field is continuing to move toward ever greater precision. Researchers are increasingly able to characterize tumors at a much finer molecular level, identifying specific drivers, resistance mechanisms and distinct patient subpopulations. This continued progress is creating new possibilities in some of oncology’s most challenging areas – from HER2-positive gastric cancers, where new therapeutic approaches are expanding what targeted treatment can achieve, to rare brain tumors such as diffuse midline glioma, where progress is beginning to reshape what is possible for patients. The science is advancing, and so is the commitment behind it.

In this video from the 2026 ASCO Annual Meeting where I sat down with STAT Brand Studio, we discuss the potential therapies have to improve patient outcomes, the encouraging breakthroughs happening in rare cancers and what I'm excited about for the future of cancer care.

In oncology, progress is rarely linear. But from my perspective, we’re in a period of genuine scientific momentum, one that is reshaping how we understand and approach cancer care, particularly for patients with rare cancers who have historically had the fewest options.

A Shift Toward Treating the Biology

Targeted therapies have fundamentally changed how we think about cancer care. In the past decades, treatment was largely defined by where a tumor originates, but today we’re increasingly guided by the underlying biology of a tumor, its molecular characteristics, its genetic drivers, and what is actually happening at the cellular level. For many patients, that shift toward precision has translated into better outcomes .

The evolution of HER2-targeted therapies is just one example that illustrates how far the field has come. What began as a single approach to targeting one protein has grown into a landscape of multiple therapies, each with distinct mechanisms of action. That evolution reflects something important: deeper scientific understanding, applied with focus, can continue to move the field forward in meaningful ways. And it points to why rare cancers, where that kind of focused scientific attention has historically been limited, represent such an important frontier.

A New Era in Neuro-Oncology

Neuro-oncology has long been one of the most challenging areas in cancer care. Tumors in or near the brain are often difficult to access surgically, driven by complex biology, and historically resistant to conventional drug delivery approaches. The unmet need here has been significant and persistent.

What is encouraging now is that the science is advancing. We have a much deeper understanding of the molecular and genetic drivers of brain tumors than we did even a decade ago. Biomarker-driven approaches are helping identify which patients may benefit from specific therapies, and new approaches to drug design are improving our ability to reach these tumors in ways that weren’t previously possible.

Why Rare Cancer Matters

This is precisely where Jazz chooses to focus. Our commitment to rare cancer is not incidental to our strategy. It is central to who we are. Our purpose is to innovate to transform the lives of patients and their families. We are particularly focused on people living with rare diseases who often have few or no therapeutic options, and oncology fits squarely within this commitment.

As tumor types are defined with greater molecular precision, rare cancer has become an increasingly serious area of scientific focus across the field. We’re deepening our expertise here, building on a track record in rare disease that we believe positions us well to contribute meaningfully, across areas including rare gastric cancers and diffuse midline glioma, where patients have historically had few options and where focused scientific attention can make a real difference.

The Work Ahead

Progress in cancer care is built through persistent research and a clear focus on what matters most to patients. The momentum we are seeing across oncology, in targeted therapies, in neuro-oncology, in rare disease, reflects what becomes possible when science and purpose are aligned. At Jazz, that alignment is what drives us.