The very young treated have no more symptoms and no longer take drugs months after the only infusion. Treatment could also be studied for other autoimmune diseases such as multiple sclerosis. New studies on larger samples are needed
A young woman dropped out of school because she suffered from a form of lupus that was so severe it caused her heart muscle to swell. Last July she went back to class. Another patient started riding again. Still another is back and DJs in nightclubs. For everyone chronic fatigue disappeared thanks to the innovative care with the cellule CAR-T used in this experimental case as “compassionate care”. For now there are only five patients, caution is also required in evaluating the possible side effects of the therapy, but the premises are encouraging.
The application of CAR-T to lupus
All study participants suffered from a severe form of systemic lupus erythematosus (SLE), autoimmune rheumatic disease which can lead to severe damage to the organs of the body. Now I’m in remission from
pathology after being treated with CAR-T cells, a technique born as a therapy against blood cancers, which consists in taking the patient’s immune cells (T lymphocytes) and modifying them in the laboratory so as to make them capable of killing the cells responsible for the disease. The study describing the radical treatment against lupus, published in Nature by a German working group, aroused the enthusiasm of doctors and patients. According to reports from the scientists, the patients have stopped the conventional drugs and after a monitoring period of 17 months they are still free from the disease: the symptoms have disappeared, therapies are no longer necessary, the antibodies that are at the basis of the disease have no longer been detected.
The spread of the disease
Circa 5 million people suffer from some form of lupus around the world (about 60 thousand in Italy). The disease is more common in females than in males, with a ratio of 9 to 1. The age group in which it occurs most often is between 15 and 50 years. It can cause different symptoms from patient to patient, but often the first signs are fatigue, low-grade fever, skin rash and joint pain. The most common form is systemic lupus erythematosus which, although not widespread, is more common than
multiple sclerosis (MS)
. They are both autoimmune diseases in which the immune system attacks the body instead of fighting the pathogens that attack it. However, while MS is an autoimmune disease in which the immune system attacks nerve tissue, with lupus all organs can be a target and affects the skin, joints, kidneys, brain, heart.
The therapies
Most patients are treated with glucocorticoids and targeted therapies which they have like target the T lymphocytes or B lymphocytes that produce the antibodies. However, these treatments are often not effective in keeping lupus in severe form at bay disease remains today orphan of a definitive cure. Lupus treatments are still so limited that even the rich and famous with the disease have struggled to cure themselves. The pop star Serena Gomez, experienced organ failure and required a kidney transplant. Precisely the variability of the disease that affects patients in different ways has made the search for a therapy complex.
The technique
German researchers from the University of Nuremberg Immunotherapy Center with colleagues from the Institute of Human Genetics selected a group of patients affected in a disabling way by the disease. Five patients (four women and a man with an average age of 22) have undergone asingle infusion of CAR-T. The technique is based on the engineering of a patient’s immune system cells which are “trained” to hunt down enemy cells. Particularly in lupus le Altered B cells produce autoantibodies that attack organs and tissues causing joint pain, rash, fatigue and other symptoms. Thanks to CAR-T, scientists trained patients’ immune cells to attack those cells responsible for producing autoantibodies. From 3 months up to 17 months after a single infusion, all patients showed an improvement in symptoms, the organs affected by the disease were cured and the autoantibodies disappeared so much so that steroid-based therapies, potentially harmful, have been suspended. In fact the disease is almost completely eradicated.
The surprise of the scientists
It is still too early to say that the patients have recovered, but the results are encouraging. The same main author of the work George Schett he said he was surprised: «It seems miraculous to me that CAR-T can solve serious cases of lupus and we are were shocked“. One hundred days after the therapy, the B lymphocytes actually reappeared, causing the team of researchers to fear that lupus flare-ups could make themselves felt again. Instead the new B lymphocytes acted normally, working in the immune system, but without exaggerating, so much so that the autoantibodies never came back. Furthermore, the function of the immune system has not been destroyed, as is often the case in cancer patients undergoing CAR-T (other cancer treatments, such as chemotherapy, make patients deeply immunocompromised before they even start CAR-T). In this case, the therapy eliminated the antibodies that cause lupus preserving immunity to the pathogens against which patients had been vaccinated, such as measles, mumps, rubella, chicken pox and hepatitis B. The researchers reported no serious infections or toxic effects in up to five patients. “It is quite exciting to be able to give such young people the opportunity to change their lives. Before the treatment, lupus dominated these kids’ conversations and dictated their plans, ”Georg Schett told Stat.
The future
We will now need to conduct a larger clinical trial and longer follow-up to definitively determine safety and efficacy of CAR-T therapy against this disease, but if CAR-T therapy proves effective and safe even on a larger number of patients, it could also be extended to many other diseases involving B lymphocytes such as systemic sclerosis, multiple sclerosis and severe autoimmune diseases. And if toxicity and other health risks are confirmed low, the therapy could be performed in the clinic, in just one day. So far the good news. There therapy is however very expensive today (approximately 400 thousand euros) and it requires a lot of work because the care is personalized. For these reasons, even if the therapy can actually be used in the future, it will still be destined for the most severe cases of lupus, resistant to treatment, with patients with a severely compromised quality of life.
September 21, 2022 (change September 21, 2022 | 15:52)
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