Three patients in the UK have received a”groundbreaking” treatment for lupus on the NHS, which experts hope could eradicate the need for lifelong drugs.
Until now, CAR T-cell therapy in the UK has been used only in cancer patients, but researchers believe it could treat range of diseases including lupus and multiple sclerosis.
The therapy works by genetically modifying cells to enable the body’s own immune system to recognise and attack problem cells, offering people a possible cure.
In the 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, a condition that can be life-threatening and cause damage to the heart, lungs, brain and kidneys.
Early indications are that the therapy – which only needs to be given once – could “revolutionise treatment” and remove the need for lifelong medication.
The dream is to be lupus-free - that would be phenomenal
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 drugs.
The first British patient, a 32-year-old mother 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 UK patients have undergone treatment at UCLH, with one of them – 50-year-old Katie Tinkler – receiving CAR T on Wednesday this week.
Mrs Tinkler, a mother of three from Guildford, has had lupus for 30 years and suffers debilitating symptoms including joint pain and kidney disease.
She told the PA news agency she was “beyond excited” to take part 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.”
Lupus can range from mild to severe, with symptoms including joint pain, skin problems, fatigue and inflammation of major organs.
People often take drugs for life, ranging from ibuprofen to steroid tablets and injections, or other immunosuppressant or biological medicines.
My body's struggling with it all now, and it's definitely got worse over the last decade ... So I just feel any opportunity that I can have is great
Around 69,000 people in the UK are thought to have the condition, which mostly affects women.
Mrs Tinkler, whose lupus has got worse over the last decade, said: “When the professor phoned me and said ‘we’re bringing a trial here, would you be interested?’ oh my gosh, I jumped at it.
“I feel that my body’s getting tired, I’ve had this for 30 years, it’s being attacked from inside 24/7.
“My body’s struggling with it all now, and it’s definitely got worse over the last decade … So I just feel any opportunity that I can have is great.”
The first British patient treated – Katherine from Manchester – said she “couldn’t believe it” when she was told she could take part in the trial.
I had to give up my job in banking. I had difficulty just getting out of bed, climbing the stairs and even doing day-to day tasks. I was forced to adapt to living life in a different way
“When you speak about lupus, you don’t talk about the possibility of coming off medication,” she said.
“Nothing had worked permanently for me. All the drugs, even when taken at high doses, have just offered temporary relief and nothing has really helped with the pain. I thought ‘this could be a solution I never thought possible’.”
Katherine was diagnosed with lupus in her late 20s and it turned her life “upside down”.
She said: “I had to give up my job in banking. I had difficulty just getting out of bed, climbing the stairs and even doing day-to day tasks. I was forced to adapt to living life in a different way.
“I cried happy tears going home from that (trial) appointment, thinking that there was a chance I could wake up every morning without pain, being able to do things like play with my daughter, and imagining all the possibilities of living a somewhat normal life – it felt amazing.”
Patients in the new study will be followed up for 15 years to fully assess the effects of the treatment.
But medics are hoping CAR T-cell therapy could correct the underlying defect in lupus and potentially cure those with the most serious form of the disease.
Even after a few weeks, Katherine said she feels she has “more energy” and has no joint swelling, adding: “I’m feeling hopeful for the future.”
Professor Ben Parker, consultant rheumatologist and study lead at the Manchester Royal Infirmary, told PA: “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, that could potentially 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 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 compared to what’s currently available.
“What currently is available are treatments that suppress the immune system, that you have to continually take, that are partially effective in most people, very effective in a few people and ineffective in a substantial number of patients with lupus.
“The CAR T treatment offers something very different…
“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.
“So it’s potentially very radical, but it still needs to be proven that what we’ve seen in a small number of people is replicable in a trial setting and then in more routine use.”
People 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.
However, many patients whose lupus is getting worse and who may be entering kidney failure, for example, decide the risks are worth it.
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.
“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.”
The CAR T in the study is obecabtagene autoleucel (obe-cel) and the clinical trial is sponsored by Autolus Limited.
Dr Eleni Tholouli, director of the adult stem cell transplant unit at Manchester Royal Infirmary, said: “It is an incredibly exciting time to now offer this mode of treatment to lupus patients through this clinical trial, which we hope will provide positive outcomes and revolutionise treatment options for patients with the disease.”
Famous people with lupus include actress and singer Selena Gomez, who has spoken about how it affects her, and singer Paula Abdul.
