On May 12, 2026 Researchers at Rice University, in collaboration with The University of Texas MD Anderson Cancer Center and clinical partners, reported results from a first-in-human trial evaluating a novel cell-based platform for localized delivery of interleukin-2 (IL-2) in patients with advanced ovarian cancer.
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The study, titled "First-In-Human Trial of Encapsulated Cell-Based Protein Producers for Localized IL-2 in Patients with High-Grade Serous Ovarian Carcinoma," published in Clinical Cancer Research, tested AVB-001, an investigational therapy composed of encapsulated, engineered cells that continuously produce IL-2 within the abdominal cavity. The approach is designed to overcome longstanding challenges associated with systemic IL-2 therapy, including toxicity and short drug half-life.
"Traditional IL-2 therapy has shown potent antitumor activity, but its clinical use has been limited by severe side effects and delivery challenges," said Omid Veiseh, a professor of bioengineering at Rice and a senior author on the study. "This platform allows us to localize and sustain cytokine exposure directly where tumors reside while minimizing systemic toxicity."
High-grade serous ovarian cancer frequently spreads throughout the peritoneal cavity, making it an ideal candidate for localized treatment strategies. In this Phase I dose-escalation trial, 14 patients with platinum-resistant disease received a single intraperitoneal administration of AVB-001 via a minimally invasive laparoscopic procedure.
The therapy was generally well tolerated, with no life-threatening treatment-related adverse events observed and no maximum tolerated dose reached. Half of the patients experienced disease stabilization, including several with prolonged periods of clinical benefit.
"These patients have very limited treatment options, so even achieving disease stability is encouraging at this stage," said Dr. Shannon Westin, a gynecologic oncologist at MD Anderson and co-lead investigator of the trial. "Importantly, we are seeing clear biological activity that supports continued development."
Immune analyses revealed that the therapy successfully activated key antitumor immune cells, including CD8+ T cells and natural killer cells, without expanding regulatory T cells that can suppress immune responses, a known limitation of conventional IL-2 therapy. The treatment also triggered increases in inflammatory cytokines and markers of immune activation, confirming its intended mechanism of action.
Notably, the study observed dose-dependent upregulation of the immune checkpoint protein CTLA-4, suggesting that combining the therapy with checkpoint inhibitors could further enhance antitumor activity.
"What is exciting is that we are not just delivering a drug, we are programming a microenvironment," said Dr. Amir Jazaeri, professor of gynecologic oncology at UT MD Anderson, member of the Rice Biotech Launch Pad’s clinical advisory board and senior author on the study. "This opens the door to combination strategies that could amplify immune responses in ways that have not been feasible before."
The implanted cell capsules are designed to release IL-2 over approximately one week, after which activity declines. Based on these findings, researchers believe that repeat dosing or higher exposure levels may be necessary to achieve stronger clinical responses.
To explore this, the team also conducted preclinical studies in nonhuman primates, demonstrating that repeat administration of the therapy was well tolerated and produced consistent pharmacological effects without added toxicity.
"This is a foundational step," added Veiseh, a Cancer Prevention and Research Institute of Texas Scholar and director of the Rice Biotech Launch Pad. "We now have evidence that the platform is safe, biologically active and potentially scalable. The next phase is optimizing dosing and exploring combination therapies to unlock its full clinical potential."
The research builds on ongoing efforts at the Rice Biotech Launch Pad, an accelerator focused on translating academic discoveries into clinical applications, and highlights the strength of the Houston biotechnology ecosystem in advancing next-generation therapies.
Future studies will evaluate higher doses, repeat administration strategies and combination approaches with immune checkpoint inhibitors to enhance efficacy.
The study was supported by the Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services, through the Targeted Hybrid Oncotherapeutic Regulation (THOR) project. The views and conclusions contained in this document are those of the authors and should not be interpreted as representing the official policies, either expressed or implied, of the United States government.
(Press release, Rice University, MAY 12, 2026, View Source [SID1234665569])