Lupus patients in England in remission after pioneering NHS trial of GM therapy

Five Lupus patients in England are in remission after being treated with a revolutionary therapy that genetically modifies their own cells, in a medical breakthrough that could offer people a cure, doctors have said.CAR (chimeric antigen receptor) T-cell therapy involves removing a type of white blood cell also called T lymphocytes, which are crucial for hunting out infected or damaged cells, and engineering them to spot and destroy disease. The T-cells are then fed back into the patient via an infusion to reset their immune system.The therapy is already revolutionising cancer treatment. Now medics in London have successfully used the technique to effectively cure five NHS patients with severe Lupus aged between 19 and 50.CAR T-cell therapy, which patients only need to have once, could transform Lupus treatment and remove the need for lifelong medication, doctors said.One of the five patients said she no longer had any of the main symptoms of the disease more than three decades after her diagnosis. Katie Tinkler has also skied for the first time in a decade and was able to dance at her daughter’s wedding.In an NHS trial led by London-hospitals-foundation-trust" class="entity-link entity-organization" data-entity-id="144765" data-entity-type="organization">London" class="entity-link entity-location" data-entity-id="2807" data-entity-type="location">University College London hospitals foundation trust (UCLH) and London" class="entity-link entity-location" data-entity-id="2807" data-entity-type="location">University College London (UCL), nine patients with severe Lupus were recruited. They had not previously responded to any treatments. Most had Lupus nephritis, a serious complication involving the kidneys.Six patients received a lower dose of CAR-T and three got a higher dose. Five of those on the lower dose, who were followed up for an average of 11 months, went into remission within just a few months.The three on the higher dose have only been followed up for three months so far, but doctors believe they too could achieve remission.Tests showed patients experienced rapid improvements in markers of the disease, including a stabilisation or improvement in their kidney function, which had been damaged by Lupus.Lupus is a chronic autoimmune disease in which the immune system attacks healthy tissues, causing widespread inflammation and damage to organs such as the kidneys, lungs and heart.It can range from mild to severe, with symptoms including joint pain, skin problems, fatigue and inflammation of major organs. About 5 million people worldwide are thought to have Lupus, which mostly affects women.Prof Karl Peggs, the director of UCLH’s biomedical research centre, said that while larger studies were needed, CAR T-cell therapy could offer people the chance of a cure. “These findings are truly groundbreaking and offer fresh hope to people living with Lupus,” he said.“While more research is needed, the possibility that CAR T-cell therapy could deliver an immune reset and potentially free patients from the cycle of chronic autoimmune disease marks a remarkable step forward.“If these results are confirmed in larger studies, the prospect of a cure for Lupus may no longer be out of reach.”Tinkler had to give up her job as a fitness instructor because of pain and fatigue caused by the severe Lupus she had had since she was 20.Lupus caused her to develop swollen and painful joints, kidney damage, lung and heart disease, and a tendency to develop blood clots. Tinkler also experienced sepsis and multi-organ failure, and had to be put in a coma in intensive care.Now the 52-year-old from Guildford, Surrey, who is married and has three adult children, is in remission. “Thinking about how life was for beforehand and how it is now, it’s like night and day,” she said.“My whole system was affected by Lupus and my joints were so painful that sometimes I couldn’t walk. I couldn’t use my hands to pick up mugs.“My kidneys, heart and lungs were all getting worse. But now I don’t have these problems any more. It’s miraculous for me. My life two years ago versus now, it’s unrecognisable. I feel blessed.”