CAR T Cells Cross Over to Successfully Treat Autoimmune Disease

German researchers reported success in treating patients with systemic lupus erythematosus (SLE) using chimeric antigen receptor (CAR) T-cell therapy. All five patients included in the compassionate-use study achieved disease remission after 3 months, and remission lasted a median of 8 months. The findings were published in Nature Medicine.

“We have been able to help… young patients who were suffering from a life-threatening form of SLE and cure them of the disease completely by treating them with CAR T cells,” principal investigator Georg Schett, MD, said in a press release from Friedrich Alexander University Erlangen-Nuremberg in Erlangen, Germany. Schett is vice president of research, chair of internal medicine, and director of the department of Medicine – Rheumatology and Immunology.

SLE is the most common type of lupus, a disease in which the body’s immune system attacks its own tissues, causing widespread inflammation and damage. The disease can affect joints, skin, brain, lungs, kidneys and blood vessels. SLE not only significantly impairs patient quality of life but can also be fatal, with the most patients dying from associated cardiovascular disease.

The patients (four women and one man), median age 22 years, had disease that was refractory to several immunosuppressive drug treatments.

T cells were collected from patients, genetically modified, expanded and reinfused. CAR T cells expanded in vivo led to deep depletion of B cells, improvement of clinical symptoms and normalization of laboratory parameters, including seroconversion of anti-double-stranded DNA antibodies.

Not only did the therapy deplete B cells — improving clinical symptoms and lab measurements — when new B cells were produced, they were naïve — unexposed to antigens.

Co-investigator Dimitros Mougiakakos, MD, from the University of Magdeburg in Germany, said in the press release, “It is astounding that 100 days after CAR T cell therapy, the B cells came back, but the disease continues to stay away.”

The investigators are planning a basket clinical trial, which will include patients with various forms of severe autoimmune disease, such as psoriasis, rheumatoid arthritis and alopecia areata.