Systematic Lupus Erythematosus (SLE) is a rare chronic autoimmune disease that usually occurs in young women. Unlike its sister disease Cutaneous Lupus Erythematosus, which only affects the skin, SLE causes the body’s immune system to attack its skin and organs, which can become life-threatening if unchecked.
Such is the case of 16-year-old Thu-Thao V who was diagnosed with SLE by doctors at Universitätsklinikum Erlangen. By the time she came in, her kidneys, heart, skin, and joints had all been affected and she “had to take almost 20 tablets every day” just to keep her symptoms at bay, according to the University’s press release.
“The joint pain was also accompanied by water retention due to my renal insufficiency, strong palpitations, and hair loss. After an acute episode, the symptoms were particularly severe,” she said.
What was wrong?
It was her body’s B cells. Usually, these cells are a part of the immune system’s lines of defense against foreign objects, creating antibodies against them. However, Thu-Thao V’s, B cells had gone haywire, producing autoantibodies against her own cells.
Previous B cell depletion therapies had failed, but the doctors could not give up. So they decided to turn to CAR-T cells.
-Prof. Dr. Andreas Mackensen, Director of the Department of Medicine 5 – Haematology and Oncology, Universitätsklinikum Erlangen.
‘CAR stands for chimeric antigen receptor which is an artificial receptor. Immune cells, or T cells, from the patient are genetically engineered in the laboratory to add the CAR. The CAR recognizes special antigens on the surface of the target cells and destroys them. Cell therapy with CAR-T cells is already being successfully used to treat leukemia and lymphoma.’
The doctors stopped all of Thu-Thao V’s treatments before starting CAR-T therapy, only supplementing it with low-dose prednisolone. 3 days after infusion, they saw a marked increase in T-cells in vivo and saw no related side effects.
Within 6 months of infusion, Thu-Thao V has completely recovered and completely stopped taking any medications. The treatment significantly destroyed her affected B cells and she hasn’t suffered a relapse since.
Having no joint pain, she can finally do sports. She’s now also breathing well, and her heart rate is normal.
Seeing this success, the researchers are looking forward to recreating the results for other patients with autoimmune diseases in a clinical trial. They published their findings in the New England Journal of Medicine.
Source: Friedrich-Alexander-Universität Erlangen-Nürnberg (FAU)