A breakthrough for lupus treatment? Study explores CAR T-cell therapy for autoimmune disease – UC Davis Health

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People with lupus know theres no cure for their condition. Treatment options usually include medications to prevent flare-ups and manage symptoms, but nothing to effectively control the disease progression.

However, researchers at UC Davis Health want to change that, as they are striving to improve the quality of life for people living with lupus through a novel clinical trial evaluating CAR T-cell therapy as a treatment for the autoimmune disease.

Lupus, formally known as Systemic lupus erythematosus (SLE), is a chronic autoimmune disorder that occurs when the bodys own defenses, the immune system, attacks the body by mistake. In this case, the cells of the immune system that produce antibodies called B cells attack healthy tissue. This leads to inflammation and damage to multiple organs, including the skin, joints, kidneys, heart and lungs.

Another subtype of the condition is lupus nephritis (LN), when lupus affects the kidney. LN causes progressive damage to the kidneys and can lead to kidney failure if not properly treated.

Lupus can be treated with immunosuppressive medications, but the therapy options are limited with variable efficacy and disease control, explained Gaurav Gulati, medical director of Rheumatology, Allergy and Clinical Immunology and principal investigator of the study. There is no cure for lupus, so these drugs are taken over prolonged periods of time, which can cause additional side effects. There is a significant need for targeted and more effective treatment options for individuals with lupus. This is vital for improving long-term control of this disease and also improve patient outcomes.

About 1.5 million Americans, and at least five million people worldwide, have a form of lupus according to the Lupus Foundation of America. Anyone can develop lupus, but certain people are at higher risk, including:

Modified T cells, known as CAR T cells, are an FDA-approved treatment for different forms of cancer including acute lymphoblastic leukemia, non-Hodgkin lymphoma, and multiple myeloma. With cancer, the immune system often fails to deploy T cells right away or at all. When it does, the attack is ineffective. CAR T-cell immunotherapy changes these collected T cells to produce chimeric antigen receptors (or CARs) that adhere to tumors to destroy them.

A recent study published in the New England Journal of Medicine, suggests CAR T-cell therapy could become a highly effective treatment for SLE patients who do not respond to current lupus therapeutics.

The study included 15 patients eight with lupus, four with systemic sclerosis (scleroderma), and three with idiopathic inflammatory myositis, a rare muscle disease. Researchers were able to eliminate or reduce symptoms and disease biomarkers with a single infusion of CAR T cells designed to target B cells, the immune cells that are key to driving autoimmunity.

There were no relapses among the studys lupus patients, who were monitored for up to two years after the treatment.

The new, industry-funded clinical trial with Cabaletta Bio is evaluating CABA-201, an investigational therapy in patients with either SLE or LN who have active disease. UC Davis Health is one of just nine health care institutions worldwide and the only hospital in the Western United States to participate in the clinical trial.

CABA-201 is a chimeric antigen receptor CAR T-cell therapy. Participants in the study will have their blood drawn and filtered through a machine in a process called apheresis. The procedure separates part of the blood such as platelets or white blood cells, while returning the rest of the blood to the body.

The trial will isolate T cells in the blood and researchers will genetically modify, or change, the T cells by putting in a code to add the CAR. This way, they can recognize, attack, and destroy the B cells in the participants body. This includes the bad B cells that engage in the participants autoimmune disease.

Once the T-cells are modified, or changed, they are supported to increase in number to produce a dose of CABA-201 cells. The CABA-201 cells are infused into the participants body, where they attack the B cells, eliminating the bad B cells and potentially enabling healthy B cells to repopulate in the body.

Our primary goal for this phase of the trial is to evaluate the safety and effectiveness of the single dose infusion of the CAR T-cell therapy, noted Gulati. If successful, this one-time treatment will correct the underlying defect and improve the quality of life for these patients.

Additional UC Davis sub-investigators for the clinical trial include Mehrdad Abedi, professor of internal medicine in the division of hematology and oncology, and rheumatology fellows Stacey Guo and Ruchi Shah. The team aims to enroll 12 patients between the ages of 18 and 65, six of them with active LN and six with active non-renal SLE.

For more information about this trial, contact the UC Davis study team at (916) 237-8850 or the sponsors study team can be reached at (267) 759-3100 or clinicaltrials@cabalettabio.com.

You can also take a survey on UC Davis StudyPages to learn if you may qualify for enrollment.

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A breakthrough for lupus treatment? Study explores CAR T-cell therapy for autoimmune disease - UC Davis Health