BY SILVIA CERNEA CLARK
Millions of Americans live with chronic and rare diseases tied to the lymphatic system ⎯ a critical but often overlooked network of vessels that helps regulate fluid balance and immune function. Lymphatic disorders are progressive and sometimes life-threatening. Current treatments focus on symptom management, but there are no approved therapies that repair damaged vessels or restore normal function.
A team of researchers led by Rice bioengineer Omid Veiseh has been awarded up to $18.2 million in funding from the Advanced Research Projects Agency for Health (ARPA-H) to develop a first-of-its-kind regenerative treatment aimed at restoring damaged lymphatic vessels and potentially curing lymphedema, a condition that affects more than 10 million Americans.
The award supports the agency’s Groundbreaking Lymphatic Interventions and Drug Exploration program, known as GLIDE, led by ARPA-H program manager Dr. Kimberley Steele. GLIDE aims to advance lymphatic medicine by improving our understanding of the role of lymphatic dysfunction in disease and building effective, affordable and accessible treatment options.
By targeting the structural damage that causes lymphedema, the therapy aims to restore lymphatic function.
“As a surgeon, I was trained to fix what I could see — but I was never taught about the one system that connects everything. As a rare disease parent, I’ve lived the heartbreak of watching someone you love suffer while medicine has no answers. And now, as an ARPA-H program manager, I get to help change that story for millions of families,” Steele said in an agency press release.
Lymphedema can develop because of inherited conditions or as a result of cancer treatment. Patients often experience chronic swelling, tissue thickening, infections and reduced mobility. Annual care costs run into the billions of dollars nationally.
“People affected by lymphedema have no other option than lifelong supportive management,” said Veiseh, professor of bioengineering at Rice, Cancer Prevention and Research Institute of Texas Scholar and director of the Rice Biotech Launch Pad. “Our goal is to change that by developing a one-time injectable treatment that gets to the root cause of the disease.”
Projected costs for the ELIXIR platform range between $5,000 and $10,000 per individual patient, which represents less than half the current annual cost of managing lymphedema.
The research project benefits from a unique translational infrastructure, including RBL LLC, a biotech venture creation studio dedicated to rapidly building companies based on breakthrough medical technologies, and the Rice Biotech Launch Pad, a biotech accelerator dedicated to bridging the gap between academic research and clinical application.
RBL portfolio company SteerBio Inc. is leading the development and commercialization effort. SteerBio is led by Martha Fowler, CEO and co-founder, alongside Veiseh and clinical collaborators Dr. Edward Chang, professor in the Department of Plastic Surgery at The University of Texas MD Anderson Cancer Center, and Dr. Ionela Iacobas, medical director of the Vascular Anomalies Center at Texas Children’s Hospital/Baylor College of Medicine.
Veiseh and his team will develop a programmable regenerative therapy called Eliminating Lymphatic Irregularities by Cross-disciplinary Intelligent Regulation, or ELIXIR, which is designed to rebuild broken lymphatic vessels.
The treatment will use a minimally invasive, subcutaneous injection to deliver engineered human retinal pigment epithelial cells ⎯ already approved by the U.S. Food and Drug Administration for treating degenerative eye disease ⎯ encased in a protective hydrogel. The hydrogel shields the cells from immune attack while allowing them to function. Inside the cells are engineered genetic circuits that produce therapeutic proteins to stimulate vessel repair.
The genetic circuits can only be activated by small-molecule regulators, giving physicians control over when and how much therapeutic protein is produced. The approach enables sustained, localized treatment and is designed as a one-time outpatient procedure.
Initial preclinical testing has shown 100% vessel regrowth toward healthy lymph nodes and 80% edema reduction with testing currently underway in large animal models.
“By targeting the structural damage that causes lymphedema, the therapy aims to restore lymphatic function,” Veiseh said. “We are building a scalable, affordable platform that could redefine the standard of care for lymphedema and open the door to a new era in lymphatic medicine. Beyond lymphedema, the technology could establish proof of concept for programmable living therapies that treat other structural and immune-related diseases.”
Over five years, the research team will advance the therapy from preclinical testing in animal models to a first in-human feasibility trial. Success will be measured by restored lymphatic function and clearance for an Investigational New Drug application with the FDA.