The first successful use of CAR-T cells to treat severe muscle inflammation
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The first successful use of CAR-T cells to treat severe muscle inflammation
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“The Lancet”: The first successful use of CAR-T cells to treat severe muscle inflammation
Idiopathic inflammatory myopathies are a group of rare immune-mediated disorders that primarily affect skeletal muscle but can also involve other organs such as the lungs, skin, and joints.
Recently, the University of Erlangen showed in a document published in The Lancet that they were the first in the world to successfully treat patients with severe muscle inflammation (myositis) using CAR-T cells . Successful treatment has now been published in a case report.
What is myositis?
Idiopathic inflammatory myopathies are a group of rare immune-mediated disorders that primarily affect skeletal muscle but can also involve other organs such as the lungs, skin, and joints.
What are the clinical manifestations of myositis?
Acute phase : Patients experience muscle aches, weakness, and fatigue, which can even lead to paralysis in severe cases.
Chronic phase : The patient will have obvious symptoms such as muscle swelling, pain and atrophy for a period of time, but these symptoms will disappear after a period of time.
Remission period : When myositis gradually improves, these symptoms will return to the previous state.
In The Lancet, a patient with idiopathic inflammatory myopathy was successfully treated with CD19 CAR T cells .
A 41-year-old man presented with refractory antistatase syndrome manifested by elevated creatinine kinase concentration, MRI findings of muscle inflammation, interstitial lung disease, Raynaud syndrome, perioral edema, and anti-Jo- 1 Autoantibodies.
Muscle biopsy showed signs of anti-synthetic peptidase syndrome, surrounding muscle atrophy and necrotic muscle fibers.
Reengineering a patient’s own immune cells brings hope for the future
” Autoimmune inflammatory muscle disease is a serious condition that can be fatal if diagnosed too late, or if the patient responds inadequately to drugs that suppress the immune system, ” explains Professor Georg Schett, Head of the Rheumatology and Immunology Unit .
In the patient’s case, all conventional treatments to suppress the immune system failed. He was rescued by CAR-T cells : ” CAR-T cells are immune cells taken from a patient’s blood and genetically engineered to carry a chimeric antigen receptor (CAR), and once these cells are returned to the patient, the CAR This allows the modified immune cells to specifically target the disease-causing cells,” explains Prof. Andreas Mackensen.
Fully recovered after six months
After receiving the CAR-T cell infusion, the patient’s health improved dramatically : Inflammation in the muscles, lungs and joints almost completely recurred. His strength, performance and endurance are all back.
“It was particularly surprising that Mr. S was able to stop taking all immunosuppressive drugs, especially corticosteroids, without having another attack,” said Dr. Fabian Müller, who cared for the patient. After receiving CAR-t cell therapy for 6 months, Mr. S has fully recovered from his autoimmune disease.
“It was like hitting a reset button! Before the treatment, I couldn’t do anything, and now I’m functioning normally again,” the patient said.
This is the second autoimmune disease that doctors and researchers at the German Center for Immunotherapy (DZI) at the University of Erlangen have successfully used CAR-t cells to treat.
Taken together, this case demonstrates the feasibility, tolerability, and efficacy of CAR T-cell therapy in the treatment of idiopathic inflammatory myopathy . Despite discontinuation of all immunosuppressive drugs, the researchers observed complete resolution of the patient’s antisynthetase syndrome, and this resolution was maintained even after reconstitution of B cells.
Although short-term effects cannot be completely ruled out, all haematopoietic systems recovered rapidly and completely within 1 week, no response to previous cyclophosphamide treatment, and no problems were noted during treatment.
The first successful use of CAR-T cells to treat severe muscle inflammation
(source:internet, reference only)
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