First UK patient with multiple sclerosis trialling CAR T cell therapy at UCLH

A UCLH patient with multiple sclerosis is the first in the UK to receive CAR T cell therapy in a clinical trial testing whether this personalised treatment may slow or even halt the progression of the disease.

Emily Henders, 37, of Bushey received her infusion earlier this month and is looking forward to being discharged from hospital.

“I hope taking part in the trial means I will never have to experience another relapse and that my MS symptoms will not progress. I know it is still experimental but it offers a scientific rationale which, as a biology teacher, makes sense to me,” she said.

There is no cure for MS, and while there are several treatments available to help manage symptoms or slow the progression of MS, none are fully effective in preventing relapses or the long-term progression of the condition.

But with CAR T cell therapy proven to be really effective in blood cancer patients, researchers are turning to testing the treatment in autoimmune conditions such as lupus and now MS.

Multiple sclerosis is a condition that affects nerves in the central nervous system, ie the brain and spinal cord. In MS, the coating (myelin) that protects the nerves is damaged. This causes a range of symptoms like blurred vision and problems with movement, memory and thinking.

CAR T cell therapy aims to reset the immune system by depleting B cells, which are thought to drive the autoimmune attack in MS. The therapy modifies the patient’s own T cells to target and eliminate B cells, potentially leading to long-term remission and halting disease progression.

UCLH consultant haematologist Claire Roddie said: “It is fantastic to be involved in the development of Obe-cel for patients with MS and this Phase I clinical study will help us understand how safe and effective obe-cel is for MS. Our ultimate goal is to achieve long periods of disease remission with a single, one-time CAR T treatment.” Dr Roddie is also associate professor in haemato-oncology at UCL and has been instrumental to the UCL/UCLH collaboration on new treatments using CAR T cell therapy.

The principal investigator on the clinical trial, called AUTO1-MS1, is Wallace Brownlee, a consultant neurologist at UCLH and clinical lead for multiple sclerosis at the National Hospital for Neurology and Neurosurgery. Dr Brownlee said: “Although treatments for MS have dramatically improved in recent years, none of the available medication fully stops relapses or progression of the illness. CAR T cell therapy is an exciting new frontier in the treatment of autoimmune conditions, and this trial will be essential to understanding the feasibility and safety of CAR T in people with MS.”

Despite the uncertainty about whether the therapy will work for her, Emily remains focused and keen to tackle her condition head on.

She added: “Ultimately, I am doing my best to preserve my ability to live life fully, and to enjoy it with my family for many years to come, without the limitations that this disease can bring. My focus is on living fully despite MS - being a wife, a mother, a teacher, and someone who refuses to be defined by illness.

“My journey with MS is about choice and action. I choose to be proactive, to educate myself, and to confront uncertainty head-on. While the path is often unpredictable and daunting, I refuse to let MS dictate my life and I remain hopeful, not only for myself but for others who may one day benefit from advances in treatment.”

There are more than 150,000 people with MS in the UK and nearly 7,100 people are newly diagnosed each year.

“This trial is open to UK patients diagnosed with relapsing or progressive forms of MS who are not responding well to the best medications already available and whose disability is worsening. We encourage patients aged between 18 and 60 years to speak to their neurologist and explore whether they might be eligible for a referral to this trial. The trial aims to recruit up to 18 patients globally by early 2027,” UCL and UCLH neurology research fellow Frederick Vonberg said.

The CAR T cell therapy being tested in this clinical trial was invented by scientists from the UCL Cancer Institute, led by Dr Martin Pule, and has delivered promising results in treating patients with an aggressive blood cancer.

The second-generation CAR T cell therapy, known as obecabtagene autoleucel (obe-cel), has reduced immune toxicity and persists for longer in blood cancer patients, overcoming two common limitations of earlier CAR T cell therapies.

It was licensed by UCL spinout Autolus and recently obtained the US Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) approval for acute lymphoblastic leukaemia but remains an experimental treatment for autoimmune conditions.

Dr Roddie and Dr Brownlee are supported by the National Institute for Health and Care Research (NIHR) UCLH Biomedical Research Centre (BRC). The BRC has been crucial to the development of CAR T cell therapy at UCL and UCLH, which has helped to lay the groundwork for this trial, with CAR T now being trialled in MS. The Cancer Clinical Trials Unit at UCLH are leading on the research delivery for this trial in close collaboration with the research team.

Prof Karl Peggs, UCLH Director of Research and Director of the NIHR UCLH BRC, said: “This study is an example of how we are taking a promising new therapy, which has already been shown to work well in some cancers, and extending it into other disease areas. It is truly groundbreaking work reflecting the combined strengths of UCLH and UCL, and made possible by NIHR support through our Biomedical Research Centre. It is also a great example of our close working relationships with the biotech industry. Thanks to studies like this, many more patients could benefit from CAR T therapy in future.”