Versiti Blood Research Institute Articles
Targeting Leukemia with Precision and Global Collaboration
Twins Lily and Bailey Dove faced leukemia diagnoses just 18 months apart. Their journey through treatment was challenging, requiring multiple blood and platelet transfusions, but their bond helped them support each other through it all. Today, both are cancer-free and living normal lives, a testament to the advancements in cancer care that inspire the work at Versiti Blood Research Institute (VBRI).
"It felt amazing to finally be able to move on," Bailey recalls. "To be physically healthy again, it felt amazing."
The Search for Treatments that Target Cancer Precisely
Leukemia affects more than 500,000 people in the United States, with over 60,000 new diagnoses each year. Despite treatment advances, this blood and bone marrow cancer still claims 20,000 to 25,000 lives annually in the U.S., with only two-thirds of patients surviving longer than five years. The outlook is even bleaker for acute myeloid leukemia, where fewer than one-third survive beyond five years.
Current treatments often damage healthy cells alongside cancerous ones, causing debilitating side effects. But there's growing hope in a different approach: targeted therapies that selectively attack cancer cells based on an individual's unique genetic and molecular profile. These treatments retrain the body's existing defense systems to recognize and destroy cancer, potentially offering fewer side effects and more favorable outcomes.At VBRI, we're working to uncover the molecular drivers of leukemia that can unlock these precision treatments.
Advancing Therapies and Anticipating Resistance
John Pulikkan, PhD, develops targeted therapies for AML while investigating the genetic mechanisms that drive this aggressive cancer. Using advanced humanized mouse models, his team tests potential treatments for two high-risk subtypes of AML. For one subtype caused by CEBPA gene mutations, Pulikkan's team has identified a promising treatment target. For another, caused by inversion 16 — one of the most frequent AML mutations — they've developed a targeted inhibitor that has been patented, licensed, and is moving toward clinical trials.
In addition to developing treatments, the Pulikkan Lab is also addressing treatment resistance, a phenomenon where patients who initially respond well to therapy gradually become less responsive over time.
"One drawback of targeted therapy is that patients will develop resistance over time," Pulikkan explains. "We are running projects to know what the potential mechanisms of resistance are so we can prepare in advance."
This work exemplifies global collaboration at its finest. The lab's research draws on patient samples from hospitals worldwide, while partnerships with pharmaceutical companies and clinicians enable rapid testing of potential treatments. Through this network, Pulikkan advances therapies that could transform outcomes not only for leukemia patients, but for all cancer patients.
VBRI's unique focus on hematologic diseases positions us to develop targeted therapies that bring science beyond the lab and into patients' lives, transforming stories like Lily and Bailey's from rare successes into expected outcomes. Together, we research cures.