Three patients in the UK have received a groundbreaking CAR T-cell therapy for severe lupus, offering hope for a potential cure and eliminating the need for lifelong medication. The treatment, previously used only in cancer patients, is now being explored for autoimmune diseases like lupus and multiple sclerosis.
The NHS trial, led by University College London Hospitals NHS Foundation Trust (UCLH) and University College London (UCL), involves genetically modifying cells to enable the body's immune system to recognize and attack problem cells. Early results suggest that this one-time therapy could revolutionize lupus treatment.
CAR T-Cell Therapy Offers New Hope for Lupus Patients
Lupus, affecting approximately 69,000 people in the UK, primarily women, is a chronic autoimmune disease with no known cure. Current treatments involve lifelong medication, ranging from ibuprofen to steroids and immunosuppressants, to manage symptoms such as joint pain, skin problems, fatigue, and organ inflammation.
Katie Tinkler, a 50-year-old mother of three from Guildford, who has battled lupus for 30 years, expressed her excitement about participating in the trial. "If this works, it's life-changing," she said. "Up 'til now, there's never been anything for lupus that is a possible cure. The dream is to be lupus-free – that would be phenomenal."
Katherine from Manchester, the first British patient to receive the treatment, shared similar sentiments. Diagnosed with lupus in her late 20s, she had to give up her job in banking due to the debilitating effects of the disease. "When you speak about lupus, you don't talk about the possibility of coming off medication," she said. "I cried happy tears going home from that (trial) appointment, thinking that there was a chance I could wake up every morning without pain."
Promising Results from Early Trials
In Germany, patients who underwent CAR T-cell therapy more than a year ago are now in remission and no longer require lupus medication. These findings have fueled optimism about the potential of CAR T-cell therapy to provide long-term benefits for lupus patients.
Professor Ben Parker, consultant rheumatologist and study lead at the Manchester Royal Infirmary, stated, "We are delighted to be the first to deliver this fantastic research in the UK, which we hope will revolutionize treatment for patients with high-risk lupus, that could potentially lead to a cure for the disease."
He further explained that the CAR T-cell treatment offers a radical alternative to current therapies that suppress the immune system. "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," Prof. Parker noted.
Trial Details and Future Outlook
The CAR T-cell therapy being used in the study is obecabtagene autoleucel (obe-cel), and the clinical trial is sponsored by Autolus Limited. Patients in the study will be monitored for 15 years to fully assess the long-term effects of the treatment.
UCLH consultant rheumatologist Dr Maria Leandro added, "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. 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."
While CAR T-cell therapy requires a 10-day hospital stay and carries potential side effects, such as increased susceptibility to infection, many patients with severe lupus, including those at risk of kidney failure, consider the potential benefits to outweigh the risks.