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A patient with multiple sclerosis has become the first person in the UK to receive CAR T cell therapy to treat her condition. Emily Henders, 37, from Bushey received her infusion earlier this month at University College London Hospitals NHS Foundation Trust (UCLH) as part of a global trial.
The AUTO1-MS1 trial was supported by the NIHR UCLH Biomedical Research Centre (BRC), which has been supporting the CAR-T cell programme at UCLH/UCL for more than 10 years.
“This is truly groundbreaking work, reflecting the combined strengths of UCLH and UCL, and made possible by NIHR support through our Biomedical Research Centre,” said Professor Karl Peggs, director of the NIHR UCLH BRC.
Multiple sclerosis (MS) is a condition that affects the central nervous system, including the brain and spinal cord. When a person has MS, the coating (myelin) that protects the nerves is damaged. This causes a range of symptoms such as blurred vision, and problems with movement, memory and thinking.
There are more than 150,000 people with MS in the UK and nearly 7,100 people are newly diagnosed each year.
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.
CAR T cell therapy has been proven to be effective in treating blood cancer, so researchers are now testing the treatment in autoimmune conditions such as lupus and MS.
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.
Principal investigator Dr Wallace Brownlee, consultant neurologist at UCLH, 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.”
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. It has delivered promising results in treating patients with an aggressive blood cancer.
The second-generation CAR T cell therapy is known as obecabtagene autoleucel (obe-cel). It 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 US Food and Drug Administration (FDA) and UK Medicines and Healthcare products Regulatory Agency (MHRA) approval for acute lymphoblastic leukaemia. However it remains an experimental treatment for autoimmune conditions.
UCLH consultant haematologist Dr 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 and Dr Brownlee are supported by the NIHR UCLH BRC. The BRC has been crucial to the development of CAR T 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 delivery of the trial in close collaboration with the research team.
Professor Karl Peggs, 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.”
The first UK participant in the trial, Ms Henders, said: “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.
“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.”
NIHR Biomedical Research Centres (BRCs) facilitate early stage experimental medicine research and support the translation of scientific discoveries.
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