Brits suffering from severe autoimmune conditions such as lupus could benefit from a ‘groundbreaking’ drug as a trial is now underway. It could cure those with the most serious form of the disease
A “groundbreaking” drug is being trialled in the first Brits to cure autoimmune conditions such as lupus and multiple sclerosis.
Until now CAR T-cell therapy had been used only in the UK for cancer patients but now three patients have received it for lupus. Medics hope the treatment, which genetically modifies the immune system to recognise and attack problem cells, could prevent life-threatening damage to the heart, lungs, brain or kidneys.
Autoimmune conditions are when the immune system malfunctions and attacks healthy tissue. In lupus B cells become overactive and produce autoantibodies that attack healthy cells. It causes painful and swollen joints, fevers, hair loss, stomach pain and can lead to kidney failure.
CAR-T therapy works by genetically modifying T cells to target and eliminate B cells that cause inflammation in the body. One of the first three patients to receive CAR-T for lupus on the trial led by University College London is mother of three Katie Tinkler from Guildford.
Katie, 50, who received the treatment on Wednesday, said: “If this works, it’s life changing. Up until now, there’s never been anything for lupus that is a possible cure. 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, a 32-year-old mother called Katherine, was treated at Manchester Royal Infirmary in July. “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. CAR T-cell therapy could correct the underlying defect in lupus and potentially cure those with the most serious form of the disease. However there are risks of side effects such as confusion, agitation, and delirium while in severe cases CAR-T therapy can cause seizures.
Around 69,000 people in the UK are thought to have lupus which mostly affects women. People often take drugs for life, ranging from ibuprofen to steroid tablets and injections, or other immunosuppressant or biological medicines. Early indications are that the therapy – which only needs to be given once – could remove the need for lifelong medication.
Study lead Professor Ben Parker, a consultant rheumatologist at Manchester Royal Infirmary, said: “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.”
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. Co-author Dr Maria Leandro, of University College London Hospitals NHS Foundation Trust (UCLH) 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.”
