Clinical trial tests promising CAR T-cell therapy for lupus
First trial participant shows significant reversal of symptoms
UCI Health rheumatologist Dr. Sheetal Desai says a new cellular therapy being tested in a clinical trial has the potential to offer lupus patient long-term relief.
Orange, Calif. — UCI Health has opened a clinical trial to test an investigational cell therapy for lupus with the potential to give patients long-term remission of symptoms.
One of the first California participants, a 41-year-old Irvine woman, experienced a striking decrease in symptoms and an improved quality of life, said Dr. Sheetal Desai, a UCI Health rheumatologist and principal investigator at the UCI Alpha Clinic, one of 10 U.S. clinical trial sites.
Desai said the CAR T-cell therapy has the potential to help patients with moderate to severe systemic lupus erythematosus who do not respond to current treatments. She is a professor and chief of the UC Irvine School of Medicine's Division of Rheumatology and executive vice chair of the Department of Medicine.
Developed by Fate Therapeutics, a clinical-stage biopharmaceutical company, FT819 is a first-in-kind, enhanced CAR T-cell product created in a lab from a line of induced pluripotent stem cells rather than a human donor.
The bioengineered immune system cells work by attacking and eliminating autoreactive B cells that drive the disease. In April, the U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to the product for the phase 1 trial after the company reported positive results in initial clinical studies.
Unlike many CAR T cell therapies that involve collecting and reprogramming a patient’s own T cells, FT819 does not require extensive genetic preparation or prolonged hospital stays in isolation. It is also being evaluated as a treatment for idiopathic inflammatory myositis, systemic sclerosis and a type of vasculitis.
Debilitating disease
Lupus, which affects nearly 1.5 million Americans, is a chronic autoimmune disease that can cause permanent damage to joints and major organs throughout the body. The most common form, systemic lupus erythematosus (SLE), is nine times more prevalent in women than men. It often emerges between ages 15 to 45 and is thought to be caused by a combination of genetics, hormones and environmental factors.
Until now, rheumatologists could offer only a handful of medications that do not fully control symptoms or disease progression. But within a few weeks after an infusion of FT819 cells, Desai's trial participant showed the first consequential reversal of symptoms in nearly two decades, becoming fully functional and able to live a more normal life.
“I am truly humbled and elated to witness the transformation in a patient I have cared for over the past 18 years, a woman who had long battled relentless fevers, joint pain, lupus nephritis and fatigue so profound she slept 18 to 20 hours a day despite trying nearly every available therapy,” she said.
Participants in the early phase clinical trial will be followed closely for two years to determine the product's safety and pharmacokinetics. The FT819 study is funded by the California Institute for Regenerative Medicine, which was created by voters in 2004 to accelerate the development of innovative cell and gene therapies. It is being conducted by the Alpha Clinic in coordination with the UCI Health Hematopoietic Stem Cell and Cellular Therapy Program.
Learn more about the FT819 clinical trial ›
You may also email alphaclinic@hs.uci.edu or call 949-824-3990 for additional information about this and dozens of other leading-edge clinical trials offered by the UCI Alpha Clinic.
About the Alpha Clinic
The UCI Alpha Clinic is one of nine California Institute for Regenerative Medicine clinical research centers in the state. The clinical trial arm of the UC Irvine Sue & Bill Gross Stem Cell Research Center, it.specializes in leading-edge stem cell clinical trials and gene therapies. It also seeks to accelerate the development of new treatments through partnerships with patients, medical providers and clinical trial sponsors. Visit stemcell.uci.edu to learn more about regenerative medicine research and stem-cell clinical trials.
About UCI Health
UCI Health, one of California’s largest academic health systems, is the clinical enterprise of the University of California, Irvine. The 1,317-bed system comprises its main campus UCI Medical Center, its flagship hospital in Orange, Calif., the UCI Health — Irvine medical campus, four hospitals and affiliated physicians of the UCI Health Community Network in Orange and Los Angeles counties and a network of ambulatory care centers across the region. UCI Medical Center provides tertiary and quaternary care and is home to the only Orange County-based National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center, gold level 1 geriatric emergency department and regional burn center. Powered by UC Irvine, UCI Health serves 5.6 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us on Facebook, Instagram, LinkedIn and X (formerly Twitter).