Five people with severe autoimmune disease are the first in the world to be treated with a therapy that uses genetically altered cells.
Five people, four women and a man, aged 18 to 24, received transplants of modified immune cells to treat a disease that can cause life threatening damage to the heart, lungs, brain and kidneys.
All five patients who were treated have been off their medication for between three and 17 months. According to doctors, the apparent success raises hopes for tackling other autoimmune diseases.
The immune system can mistakenly attack healthy tissues and organs. The causes are not well understood, but researchers think it may be caused by viral infections and changes in the body around puberty.
More women than men are affected by the condition. It is difficult to diagnose because the symptoms can flare up and overlap with other diseases. Extreme fatigue, organ damage, and pain in the joints and muscles can be caused by lupus. There is a skin rash on the nose and cheeks.
Five severely ill patients were treated with CAR T-cell therapy by doctors in Germany. In 2015, the approach was used in a leukaemia patient. CAR T-cell therapy involves collecting the patient's T-cells and modifying them so that they attack new targets, such as cancer cells.
The modified T-cells were used to attack the patients' B cells. B cells attack healthy tissues instead of defending the body against invaders.
The therapy wiped out the patients' B cells and improved their condition according to the study. Severe symptoms of the disease, including arthritis, fatigue, and fibrosis of the heart valves, were all gone after therapy.
The patients' B cells were found to have recovered about four months after the treatment, but they no longer produced the antibodies that helped them fight disease. The authors theorize that the therapy resulted in a regrowth of the immune system.
Prof Schett is a rheumatologist who led the work at Friedrich-Alexander University. A number of diseases dependent on B cells may respond to this treatment. Rheumatoid arthritis and myositis are included. Multiplesclerosis may respond to CAR T-cell treatment.
Schett's team wanted to make sure the therapy didn't impair the patients' immune systems and make them more susceptible to infections. Before and after the therapy, they assessed the patients' responses to a number of vaccines. The immune responses of the patients were not different after the treatment.
The study promises to extend the scope of CAR T-cell therapy, which has thus far seen its major impact in the treatment of blood cancers, to autoimmune diseases which are in some patients poorly controlled with other medicines. I'm very excited about the possibilities for this form of living therapy.