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In a 1st, scientists use designer immune cells to send an autoimmune disease into remission

illustration of blob-like b cells releasing tiny, y-shaped antibodies

In lupus, B cells release “autoantibodies” that latch onto your body’s cells, triggering a damaging immune response.(Image credit: NANOCLUSTERING/SCIENCE PHOTO LIBRARY via Getty Images)

Five patients with hard-to-treat lupus entered remission after scientists tweaked their immune cells utilizing a technique normally used to take care of cancer. Following the one-time therapy, all five patients with the autoimmune disease stopped their standard treatments and haven’t had a relapse.

This treatment, referred to as chimeric antigen receptor (CAR) T-cell therapy, must be tested in larger sets of lupus patients before it could be approved for widespread use. If the results endure in larger trials, the treatment could someday offer relief to people who have moderate to severe lupus.

“For them, that is a really breakthrough,” said Dr. Georg Schett, director of rheumatology and immunology at Friedrich Alexander University Erlangen-Nuremberg in Germany. Schett may be the senior writer of a fresh report describing the tiny trial, that was published Thursday (Sept. 15) in the journal Nature Medicine (opens in new tab).

“It is a single shot of CAR T cells and patients stop all treatments,” Schett told Live Science. “We were really surprised [at] how good this effect is.”

Related: Scientists finally have proof mysterious immune cell in humans

Rebooting the disease fighting capability

Lupus is really a chronic disease where the disease fighting capability inadvertently attacks your body’s own cells, leading to inflammation, injury, pain and fatigue. The outward symptoms, starting from mild alive threatening, can arrive in “flares” and patients often take multiple drugs to lessen their frequency and severity.

In lupus, dysfunctional B cells, a kind of immune cell, generate “autoantibodies” that glom onto your body’s cells and summon other cells to destroy them. Several medications target these harmful B cells, however they don’t work with all lupus patients.

“There exists a group that is really very severe plus they cycle through several therapies rather than enter into remission,” Schett said.

Schett’s group theorized that such treatment-resistant lupus patients may potentially reap the benefits of CAR T-cell therapy, which includes previously been used to take care of cancer patients. During CAR T-cell therapy, doctors extract immune cells, called T cells, from the patient’s blood, genetically tweak those T cells in the lab and inject them back to the patient’s body, based on the NIH’s National Cancer Institute (opens in new tab) (NCI). In every the approved cancer therapies, these engineered T cells target B cells with specific molecules on the surfaces, wiping out both problem cells and healthy B cells.

Without these B cells, patients could be more susceptible to infections, and CAR T-cell therapy also posesses threat of triggering “cytokine release syndrome,” where T cells suddenly unleash a flood of inflammatory molecules in to the bloodstream. So, despite its potential benefits, the procedure isn’t befitting anyone who has only mild disease.

For his or her trial, Schett and his colleagues recruited treatment-resistant patients with common type of lupus, called systemic lupus erythematosus (SLE). All of the trial participants showed damage in multiple organs, like the kidneys, heart, lungs and joints.

Related: New tool estimates your immune ‘age,’ predicts threat of disease

Following treatment, all five participants’ B cell counts plummeted, as did their autoantibody levels. Their lupus symptoms abated plus they all stopped taking their prior medications, therefore far, no patients have relapsed. The 1st patient treated whose case was described in the the New England Journal of Medicine (opens in new tab) has experienced drug-free remission for 17 months.

“She lives a totally normal life,” Schett said.

Notably, five months post-treatment, the patient’s B cell count begun to rise but her symptoms didn’t return. Because the horde of dysfunctional B cells have been annihilated from your body, the bone marrow began making new “baby” B cells that don’t generate exactly the same autoantibodies as their predecessors did, Schett said.

Another four patients’ also started making new B cells within months of treatment, without relapsing. It appears as if rebooting the B cell system in this manner may avoid the disease from returning but they’ll have to keep monitoring the patients to be certain, Schett said.

“The mean follow-up of 8 months is prematurily . to find out whether that is complete remission,” said Dr. Jean Yean-jin Lin, an instructor of medicine (rheumatology) at the Northwestern University Feinberg School of Medicine, who was simply not mixed up in trial. “It is possible these naive B cells as time passes might re-encounter self-antigens and be autoreactive,” Lin told Live Science within an email.

None of the patients developed cytokine release syndrome or other serious unwanted effects, but this might not hold true for several lupus patients, she noted. “The tolerability seemed ok however when more patients are treated more side-effects will probably come to the top,” Dr. Ronald van Vollenhoven, a professor of rheumatology at Amsterdam University Medical Centers who was simply not mixed up in trial, told Live Science within an email.

Schett and his team are organizing a more substantial trial of CAR T-cell therapy for lupus, and also the autoimmune diseases systemic sclerosis and myositis. Later on, the therapy may be tested as cure for arthritis rheumatoid and multiple sclerosis, among other autoimmune disorders, Schett said.

If eventually approved, CAR T for lupus “will be a choice for patients who’ve very severe SLE and who’ve failed available treatments,” van Vollenhoven said. “More long-term, the question is if this novel therapy could achieve long-lasting remission as well as ‘cure.'”

“The prospect of CAR T to reshape the disease fighting capability and result in durable treatment free remission is exciting,” Lin said.

Originally published on Live Science.

Nicoletta Lanese is really a staff writer for Live Science covering health insurance and medicine, alongside a variety of biology, animal, environment and climate stories. She holds degrees in neuroscience and dance from the University of Florida and a graduate certificate in science communication from the University of California, Santa Cruz. Her work has appeared in The Scientist Magazine, Science News, The San Jose Mercury News and Mongabay, among other outlets.

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