UCLH clinicians are playing a leading role in a new UK clinical trial testing whether an experimental drug can make cutting-edge blood cancer treatments safer and more accessible for thousands of patients with multiple myeloma.
Professor Rakesh Popat, consultant haematologist at UCLH, is co-chief investigator on the trial, called TOPICAL, which is testing an experimental anti-inflammatory treatment known as POLB 001. The drug aims to prevent a common side effect called Cytokine Release Syndrome (CRS), a potentially dangerous immune reaction that can occur following powerful cancer immunotherapy treatments.
Professor Popat said: “This study addresses an important question, whether patients can safely receive bispecific antibodies, particularly when they are dosed outside of hospital. While CRS in the most part is manageable, it leads to increased use of hospital resources and a higher burden of treatment on patients by the need for close monitoring and administration of treatment once it occurs. Having an oral preventative treatment can help reduce this and make the treatment easier for patients.”
New immunotherapy treatments are transforming survival prospects for patients with blood cancers such as multiple myeloma, lymphoma and leukaemia. However, these treatments can trigger CRS, causing severe flu-like symptoms including high fever, dangerously low blood pressure and breathing difficulties, which can in some cases be life-threatening.
Nearly a quarter of patients who develop severe CRS require intensive care, and studies show that more than 70% of patients receiving some cancer immunotherapies are affected by CRS, highlighting an unmet need for proactive, preventative treatments.
TOPICAL is led by The Christie NHS Foundation Trust and is taking place at UCLH at the National Institute for Health and Care Research (NIHR) Clinical Research Facility: UCLH, and four other NHS hospitals across the UK. The trial plans to recruit around 30 patients with relapsed or treatment-resistant multiple myeloma, who will receive POLB 001 alongside an approved bispecific antibody therapy. The trial is supported by the NIHR Biomedical Research Centre: UCLH.
Dr Emma Searle, consultant haematologist at The Christie and co-chief investigator on the TOPICAL trial, said: “Immunotherapy treatments are transforming survival rates for patients with difficult-to-treat blood cancers, but their use will continue to be restricted until we find a way to deliver them safely outside of specialist hospitals.
“Because of the risk of CRS, which is unpleasant and can be dangerous, many patients can’t be treated near to home, which is a real problem. Many patients have to stay in hospital to receive immunotherapy, and some must travel long distances for treatment in specialist centres.
“If this new therapy can safely reduce the risk of CRS, it could help give these treatments near to or in people’s homes. Ultimately, our aim is to help more patients benefit from these life-saving therapies while reducing the burden of treatment on patients, families and the NHS.”
POLB 001 is being developed by London-headquartered biotechnology company Poolbeg Pharma (London Stock Exchange Listed, Ticker: POLB). The oral treatment works by targeting inflammatory pathways involved in CRS. Researchers will assess whether it can prevent or reduce CRS following teclistamab treatment.
The investigator-led study is sponsored by the DIDACT Foundation, a partner organisation of Accelerating Clinical Trials Ltd (ACT), which is dedicated to improving outcomes for people with blood cancers. Early results are expected later this year.
