Huang Lab Publishes JCI Study Showing Luspatercept Improves Anemia in a Sickle Cell Disease Model

SAN ANTONIO, Texas — The Huang Lab at the Mays Cancer Center at UT Health San Antonio announces a new research letter in the Journal of Clinical Investigation: "Luspatercept ameliorates disease phenotype and complications in the Townes mouse model of sickle cell disease." The article was published February 2, 2026, and appears in Volume 136, Issue 3. Authors are Maiko Sezaki, Tian Li, Mingzhe Pan, Zhihong Wang, Jie Bai, Justin G. Horowitz, Julia Z. Xu, and Gang Huang.

Sickle cell disease causes chronic anemia and painful vaso-occlusive crises, and current options for the anemia carry real drawbacks. Transfusions can lead to iron overload and immune reactions, and erythroid-stimulating agents have shown inconsistent results. The study tested luspatercept, a fusion protein that traps TGF-beta ligands and is already approved for beta-thalassemia and some myelodysplastic syndromes, as an alternative approach.

In the Townes mouse model of sickle cell disease, luspatercept extended survival and improved red blood cell count, hemoglobin, and hematocrit. Treated mice showed fewer sickled and abnormal cells, fewer circulating reticulocytes, and longer-lived, better-quality red cells with lower mitochondrial content. The benefits did not depend on raising fetal hemoglobin.

The authors also traced effects upstream in the bone marrow and spleen, including relief from anemia-driven stress on blood-forming stem cells and reduced reliance on extramedullary hematopoiesis. A shift toward patrolling monocytes, which clear endothelium-adherent sickle cells, points to protection against vaso-occlusion. Together the results build a rationale for clinical trials of luspatercept in patients with sickle cell disease.

"Managing anemia in sickle cell disease is a balancing act, and luspatercept improved the blood on several fronts at once," said Dr. Gang Huang, principal investigator. "These preclinical results make a strong case for testing the drug in patients."

DOI: https://doi.org/10.1172/JCI197706

About the Huang Lab

The Huang Lab at UT Health San Antonio is a basic and translational cancer research group within the MD Anderson Mays Cancer Center at UT Health San Antonio. Our team integrates hematology and solid tumor biology with immunology and metabolism to uncover mechanisms and design interventions. We advance cell and biologic therapies, including CAR T-cell approaches for solid tumors. We also conduct research in trauma and military health. We move discoveries toward clinical impact through product development and collaborations with clinicians and industry. Education and mentorship are core to our mission. We train students, postdoctoral fellows, and early-career scientists in rigorous, multidisciplinary research. Together, we aim to deliver therapies and technologies that improve patient outcomes.