Sheffield CAR T MS Patient: Best Birthday Gift

Sheffield Teaching Hospitals NHS

A Rotherham man has become the first patient at Sheffield Teaching Hospitals NHS Foundation Trust and the seventh participant in a global trial to receive CAR T-cell therapy as a potential "one-and-done" treatment for multiple sclerosis (MS).

Numan Unees was diagnosed with MS six years ago and rapidly lost his ability to walk. He received the CAR T-cell therapy obecabtagene autoleucel, also known as obe-cel, at the Royal Hallamshire Hospital as part of a landmark clinical trial just two days before his 40th birthday, saying it was the "best birthday gift ever".

The phase I AUTO1-MS1 trial, which is running in four UK sites including Sheffield, is one of the first to test the safety of CAR T-cell therapy as a treatment for MS.

CAR T-cell therapy is a novel therapy where a patient's white cells are genetically engineered to seek and destroy abnormal immune cells causing disease, leading to a resetting of the immune system.

It has shown huge promise in the treatment of some blood cancers, with patients achieving long-term remission after having failed multiple standard treatments.

Researchers are now looking to see if this mechanism can be replicated in patients with autoimmune conditions such as MS and lupus.

If successful, it offers hope of a one-off, immediate treatment for MS which could slow, or even halt disease progression.

Sheffield leads the way

| Numan Unees receiving the obe-cel CAR T treatment for his MS at the Royal Hallamshire Hospital

Numaan, who has two young children, said:

"MS has affected my life severely. I went from being fit and healthy to having to use crutches within three to six months of diagnosis. The deterioration was rapid and my mobility was gone. It was a bad situation, being in a wheelchair with the pressure of having two young children, but we've always tried to stay positive.

"The hope of this treatment for me is that it stops the multiple sclerosis within my body and from there I'm hoping to do physiotherapy and get some mobility back because I'm stuck at home and I can't work or drive anymore. If I can get some mobility back, if not 100% at least walk, I can get some purpose back and set an example to the kids and be working.

"I was apprehensive at first as it's a new treatment for MS and I was expecting to have to go to London, but the knowledge base and the expertise of everyone here has been above and beyond what I expected. The whole team in Sheffield have been amazing, they've been legends. I feel very blessed."

A significant 'moment' for MS patients

| A specialist CAR T nurse gets Numan's reenigneered cells ready for his CAR T infusion

In MS, CAR T aims to reset the immune system by depleting B cells which are believed to be driving the autoimmune attack causing the disease.

Once infused back into the body, the patient's own modified T cells - which are given new instructions in the lab - act as a living drug to target and eliminate B cells.

The trial, which is currently open to patients aged between 18 and 60 years diagnosed with the progressive form of MS who are not responding well to the best medications already available and whose disability is worsening, is set to recruit up to 18 patients globally.

Professor Basil Sharrack, Consultant Neurologist at Sheffield Teaching Hospitals NHS Foundation Trust, said: "We are delighted to have delivered CAR T to our first MS patient as part of this breakthrough research. This represents a significant moment for MS patients, as by targeting and eliminating disease-causing cells in the tissues of the nervous system, CAR T offers a one-off immune system reset that could stop disability progression in this disease.

"As the only UK site to have pioneered stem cell transplantation for MS, it's fantastic to once again be at the forefront of cutting-edge research that could give hope to thousands in the MS community."

Professor John Snowden, Honorary Consultant Haematologist at Sheffield Teaching Hospitals NHS Foundation Trust and Professor of Haematology at the University of Sheffield, said: "CAR T is a rapidly evolving treatment which has already transformed the lives of thousands of cancer patients previously considered untreatable. The type of CAR T therapy used in this trial was also recently approved on the NHS for use in certain blood cancers. We have significant expertise in delivering this complex, specialist treatment here in Sheffield, so it's exciting to be at the helm of this new chapter of research in advanced therapy medicinal products, or ATMPs. On behalf of the whole team, I would also like to acknowledge the phenomenal work of our pharmacy, nursing and research teams and the fantastic collaboration between our haematology and neurology teams which has made this possible."

MS is a condition where the immune system mistakenly attacks nerves in the brain and spinal cord.

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