UCI Health launches clinical trial to test investigational cell therapy for lupus

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.”

Dr. Sheetal Desai, a UCI Health rheumatologist and principal investigator at the UCI Alpha Clinic

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.