Doctors hail a “groundbreaking” new treatment for lupus that genetically alters a patient’s cells to fight the autoimmune disease and may end the need for life-long medication.
Lupus can range from mild to severe, with symptoms including joint pain, skin problems, fatigue and inflammation of major organs. Patients often have to take drugs their whole lives, ranging from ibuprofen to steroid tablets and injections, or other immunosuppressive or biologic drugs.
Around 5 million people worldwide are thought to have the condition, which mostly affects women.
In the UK, three patients received CAR T-cell therapy for the most severe form of lupus, which can be life-threatening and cause damage to the heart, lungs, brain and kidneys. In Germanypatients who had the new treatment are now in remission and do not need to take lupus drugs.
Prof Ben Parker, a consultant rheumatologist at Manchester Royal Infirmary, said he believed the breakthrough could revolutionize treatment for lupus patients, and could lead to a cure for the disease.
“Lupus is a disease that requires lifelong medication, but this therapy has the potential to change that, which is incredibly exciting. This groundbreaking new therapy is an important milestone in our research into lupus.”
Until now, CAR T-cell therapy has mostly been used in cancer patients, but researchers believe it could treat a range of other diseases.
It works by genetically modifying cells to enable the body’s own immune system to recognize and attack problem cells, offering people a possible cure.
The first British patient, a 32-year-old woman named Katherine, is at the National Institute for Health and Care Research Manchester Clinical Research Facility at Manchester Royal Infirmary in July.
Two other British patients underwent treatment at University College Hospital, with one of them – 50-year-old Katie Tinkler – receiving CAR T-cell therapy on Wednesday this week.
Tinkler, from Guildford, Surrey, who has had lupus for 30 years with symptoms including joint pain and kidney disease, said she was “very excited” to take part in the trial.
“If it works, it’s life-changing,” she said. “Until now, there has never been anything for lupus that is a possible cure. The dream is to be lupus free – that would be phenomenal.”
Patients who have the new treatment will be followed up for 15 years to fully evaluate its effects.
Doctors hope CAR T-cell therapy can correct the underlying defect in lupus and potentially cure those with the most severe form of the disease.
Even after a few weeks, Katherine said she felt she had “more energy” and was no longer experiencing joint swelling, adding, “I feel hopeful for the future.”
People undergoing the new treatment require a 10-day hospital stay and are warned of possible side effects. This includes their immune system being very susceptible to infection or some other condition that causes the immune system to go into overdrive for a few weeks.
However, many patients whose lupus worsens and who may enter kidney failure, for example, may decide that the risks are worth it. The CAR T-cell therapy in the study is called obecabtagene autoleucel and the trial is sponsored by Autolus Limited.
Dr Eleni Tholouli, the director of the adult stem cell transplant unit at Manchester Royal Infirmary, said: “It is an incredibly exciting time to now be offering this form of treatment to lupus patients through this clinical trial.”
“We hope [it] will provide positive outcomes and revolutionize treatment options for patients with the disease.”
