CAR T-cell Therapy Shows Promise in Treating Severe Lupus in UK Trial
核心洞察
Three patients in the UK have received CAR T-cell therapy for severe lupus (搜索), a potentially life-threatening autoimmune disease.
Early results from a German study showed that patients who underwent CAR T-cell therapy are now in remission and do not require lupus (搜索) medication.
The NHS trial, led by UCLH and UCL, modifies cells to enable the immune system to recognize and attack problem cells, offering a possible cure.
CAR T-cell therapy is being trialed in the UK as a potential breakthrough treatment for severe lupus (搜索), offering hope for a possible cure. Until now, CAR T-cell therapy in the UK has been used only in cancer patients, but researchers believe it could treat a range of diseases including lupus and multiple sclerosis (搜索).
CAR T-cell Therapy for Lupus
The therapy involves genetically modifying a patient's own cells to enable their immune system to recognize and attack the cells causing the disease. This approach offers the potential for a one-time treatment that could eliminate the need for lifelong medication.
In a new NHS trial led by University College London Hospitals NHS Foundation Trust (UCLH) and University College London (UCL), three patients have received CAR T-cell therapy for the most serious form of lupus (搜索), a condition that can be life-threatening and cause damage to the heart, lungs, brain, and kidneys. Early indications suggest the therapy could "revolutionise treatment".
International Precedent
In Germany, patients who underwent CAR T-cell therapy more than a year ago are now in remission and do not need to take lupus (搜索) drugs, providing a promising precedent for the UK trial.
Patient Experiences
The first British patient, a 32-year-old mother named Katherine from Manchester, was treated in July. She reported feeling more energetic and experiencing no joint swelling even after a few weeks. Two other UK patients have undergone treatment at UCLH, including 50-year-old Katie Tinkler, who has lived with lupus (搜索) for 30 years and suffers from debilitating symptoms including joint pain and kidney disease. Tinkler expressed her excitement about participating in the trial, stating, "If this works, it’s life changing...The dream is to be lupus-free – that would be phenomenal."
Lupus and Current Treatments
Lupus (搜索) can range from mild to severe, with symptoms including joint pain, skin problems, fatigue, and inflammation of major organs. Current treatments often involve lifelong medication, ranging from ibuprofen to steroids and immunosuppressants. Around 69,000 people in the UK are thought to have the condition, which mostly affects women.
Expert Perspectives
Professor Ben Parker, consultant rheumatologist and study lead at the Manchester Royal Infirmary, said, "We are delighted to be the first to deliver this fantastic research in the UK, which we hope will revolutionise treatment for patients with high-risk lupus (搜索), that could potentially lead to a cure for the disease...This groundbreaking new therapy marks a significant milestone in our research into lupus."
Prof Parker explained that the treatment involved programming the CAR T cells, adding: “Ultimately, it offers a very radical treatment for lupus (搜索) compared to what’s currently available...In those people who’ve received it, they have been able to stop their immunosuppression after receiving CAR T and remain in what’s called drug-free remission."
Trial Details
The CAR T in the study is obecabtagene autoleucel (obe-cel) and the clinical trial is sponsored by Autolus Limited. Patients undergoing CAR T require a 10-day hospital stay and are warned of possible side-effects, such as their immune system being very susceptible to infection for several weeks or another condition that causes the immune system to go into overdrive. Patients in the new study will be followed up for 15 years to fully assess the effects of the treatment.
UCLH consultant rheumatologist Dr Maria Leandro said, "We have seen the effectiveness of CAR T in cancer, particularly blood cancers, and hope we can replicate this in autoimmune diseases, starting with lupus (搜索)...Our early phase trial aims to recruit 12 people worldwide and we hope to replicate the results of a small German trial which showed an improvement in lupus patients treated with CAR T-cell therapy."