Doctors hail “groundbreaking” new treatment for lupus that genetically modifies a patient’s cells to fight the autoimmune system disease and could end the need for lifelong medication.
Lupus can range from mild to severe, with symptoms including joint pain, skin problems, fatigue and inflammation of major organs. Patients often need to take medications throughout their lives, ranging from ibuprofen to steroid tablets and injections, or other immunosuppressants or biologic medications.
About 5 million people worldwide are believed to have the condition, which mainly affects women.
In Britain, three patients have 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 Germany, patients who received the new treatment are now in remission and do not need to take lupus drugs.
Prof. Ben Parker, a rheumatologist at Manchester Royal Infirmary, said he believed the breakthrough could revolutionize the treatment of lupus patients and 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 marks an important milestone in our lupus research.”
Until now, CAR T-cell therapy has mainly been used in cancer patients, but researchers think it could treat a range of other diseases.
It works by genetically modifying cells so that the body’s own immune system can recognize and attack problem cells, offering people a possible cure.
The first British patient, a 32-year-old woman called Katherine, was treated at the National Institute for Health and Care Research Manchester Clinical Research Facility at Manchester Royal Infirmary in July.
Two other British patients have undergone 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 “extremely excited” to take part in the trial.
“If this works, it will be 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 receiving the new treatment will be followed for 15 years to fully assess its effects.
Doctors hope that CAR T-cell therapy can correct the underlying defect in lupus and possibly cure people with the most severe form of the disease.
Even after a few weeks, Katherine said she felt like she had “more energy” and no longer had joint swelling. She added: ‘I have hope for the future.’
People who undergo the new treatment require a 10-day hospital stay and are warned about possible side effects. This includes making their immune system very susceptible to infections or some other condition that causes the immune system to go into overdrive for several weeks.
However, many patients whose lupus worsens and may develop 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 study is sponsored by Autolus Limited.
Dr. Eleni Tholuli, director of the Adult Stem Cell Transplant Unit at Manchester Royal Infirmary, said: “It is an incredibly exciting time to now be offering this treatment option to lupus patients through this clinical trial.”
“We hope it will yield positive results and revolutionize treatment options for patients with the disease.”