“The treatment is relentless but essential” says Caroline. This is the harsh reality of battling Primary Central Nervous System Lymphoma (PCNSL), a rare and aggressive blood cancer affecting the brain. Yet, despite the gruelling treatment, Caroline’s story is one of hope, and the incredible impact of clinical trials.

Primary Central Nervous System Lymphoma (PCNSL) is an extremely rare and aggressive form of blood cancer that affects the brain. With only around 300 people diagnosed in the UK each year, PCNSL presents a challenging journey for those affected. One such person is Caroline, who was diagnosed in April 2023 after what initially seemed like a minor health concern.

A Routine Check-Up Turns Into a Diagnosis

Caroline first noticed something was wrong when she began experiencing persistent migraines. Concerned, she visited her optician for a routine check-up. “They noticed a small bleed behind my eye but said it was nothing to be concerned about,” she recalled. However, Caroline’s instincts told her otherwise, prompting her to visit her local GP. After initial assessments, doctors suspected she had a swollen optic nerve and referred her for an MRI at Leicester General Hospital.

It was during this MRI scan that her life took an unexpected turn. “I felt fine, just thought it was a bit of a headache,” she said. But at midnight that night, doctors delivered devastating news, she had a brain lesion. Caroline was immediately admitted to the hospital for further testing. After ten days of intensive scans and procedures, she was transferred to Nottingham University Hospital NHS Trust for a brain biopsy.

The Isolation and the Diagnosis

Following her biopsy, Caroline was moved to an isolation room to protect her from potential infections. It was in this setting that she received her official diagnosis of PCNSL surrounded by the amazing medical team and her husband. “I couldn’t see my son for 40 days, but we video chatted frequently, which helped keep my spirits up” she said, reflecting on one of the most difficult aspects of her journey.

The OptiMATe Trial

Caroline was given the opportunity to participate in The OptiMATe trial, an international clinical research study funded by Cancer Research UK aiming to improve treatments for PCNSL. , with the trial also running in Germany, Italy, and Austria.

“I just feel extremely grateful and lucky that I’ve got the opportunity to be on the trial,” she said. “This disease is horrible, and I want to do anything I can to make this easier for somebody else. Trials like this will hopefully mean people in the future have access to easier treatment than I’ve had.”

Clinical trials like OptiMATe are crucial for rare diseases like PCNSL, where treatment options are limited. Caroline knows firsthand how important these studies are, not just for herself but for future patients. “Without the funding and a clinical trial, my outcome could have been very different” she acknowledged. “People don’t realise how vital research is until they are in a position like mine.”

Led in the UK by Professor Chris Fox, Consultant Haematologist at Nottingham University Hospitals NHS Trust, and coordinated by the Southampton Clinical Trials Unit, The OptiMATe trial involves a combination of chemotherapy and targeted drugs. If the treatment proves effective, it is followed by high-dose chemotherapy and a stem cell transplant using the patient’s own cells.

While Caroline is grateful for the opportunity, she does not sugarcoat the gruelling nature of her treatment. Yet, despite the physical and emotional toll, she remains hopeful that her participation will pave the way for better therapies.

She recalls the support from the Clinical Team at Nottingham University Hospital as incredible. Her dedicated research nurse, Abigail Sutcliffe was approachable and consistently available to address any questions she had. Gaining a clear understanding of her treatment made her feel more at ease.

While Caroline’s experience highlights the impact of clinical trials for newly diagnosed patients, research is also critical for those who relapse or don’t respond to initial treatment. Another example of an innovative trial tackling this difficult to treat disease is the PRiZM+ trial—a TAP-funded study supported by Cure Leukaemia.

PRiZM+ focuses on patients with relapsed or refractory PCNSL, for whom treatment options are extremely limited and outcomes can be poor. This trial is investigating a promising new combination of therapies aimed at overcoming resistance to previous treatments. It represents a vital lifeline for patients who have already endured the toughest aspects of this disease and are in urgent need of alternatives.

Like OptiMATe, PRiZM+ is pushing the boundaries of what’s possible in PCNSL care. Together, these trials reflect the critical role of research in driving forward better treatments.

A Wish for Kinder Treatment

Caroline’s current care includes MRI scans and blood tests every three months, along with support from a clinical psychologist. She remains vocal about the importance of research and funding for rare blood cancers like PCNSL.

Her ultimate wish is that her experience contributes to progress. “I just want my clinical trial to help someone else,” she said. “I want the future treatment to be kinder.”