A study led by a team of German scientists provides evidence of the safety and efficacy of CD19 CAR T cell therapy and its impact on autoimmune diseases. The study investigating CAR T for autoimmune diseases was hailed as “a most significant study [on the subject] to date”.
An estimated one in every ten people worldwide have been diagnosed with an autoimmune disease. As reported in the February 22nd issue of the New England Journal of Medicine and recently published in Inside Precision Medicine, for the last several years the use of CAR T therapy to treat autoimmune diseases has been gaining traction.
The current treatments for autoimmune disorders are mostly immunosuppressive agents or blocking antibodies which manage, but do not cure, the disorders.
Several clinical trials of chimeric antigen receptor (CD19 CAR T) in systemic lupus erythematosus (SLE) are ongoing, while others are underway for multiple sclerosis. Other autoimmune disorders such as scleroderma are being tested.
CAR T cells are being called upon for cancer treatment with particular emphasis on hematologic neoplasms that target malignant B cells. The most common types of neoplasms are various forms of lymphoma and leukemia and the progressive, often life-threatening forms of myelodysplastic syndromes.
In addition, autoreactive B cells are often the cause of autoimmune diseases — namely idiopathic inflammatory myositis, systemic sclerosis, and lupus erythematosus. Each of the aforementioned diseases can result in damage to various organs, inflammation and increased mortality.
Autoimmune treatments often require lengthy immune suppression as the immune system produces self-targeting antibodies and goes on the attack against its own cells.
Although antibody focused targeting of B-cells has brought improvements in the treatment of autoimmune diseases, it has yet to bring about complete drug-free remission which was one of the team’s expectations for this CD19 CAR T study.
About the Study
Fifteen recipients were given one infusion of CD19 CAR T cells after having been preconditioned with cyclophosphamide and fludarabine. Their progress was then followed for two years.
Among the represented immune disorders were SLE, idiopathic inflammatory myositis, and systemic sclerosis. The team reported that every patient experienced remarkable improvement. As a result, immunosuppressive therapy was discontinued for all patients.
The treatment has been deemed to be relatively safe. Among adverse effects, 10 patients experienced mild cytokine release syndrome response-Grade 1, with one patient having a Grade 2 response. Lastly, one patient developed pneumonia requiring hospitalization.
The authors of the study wrote that the data support the long term and short term efficacy and safety of CD19 CAR T cell therapy, while also noting that there is a need for additional clinical trials.
You can read more about the study over at Inside Precision Medicine, or read the full study results at the New England Journal of Medicine.
