VHL UK Ireland has granted £41,000 to Crick Insitute Researchers – Dr. Scott Shepherd, Prof. Samra Turajlic and Prof. William Drake
Background
Belzutifan is transforming the management of individuals with von Hippel-Lindau (VHL)
disease. However, its real-world efficacy and safety outside clinical trials remain poorly
characterised. The pivotal trial that led to its approval included only 61 patients, limiting
our understanding of how patient-specific factors, such as VHL mutation type, age, sex,
and co-morbidity, affect treatment response and toxicity.
Additionally, no robust quality-of-life (QoL) data exist to quantify how Belzutifan improves
patient well-being or how side effects, such as anaemia, impact daily life. Moreover,
mechanisms of response and resistance remain undefined; a more detailed
understanding is crucial for optimising treatment strategies and may have broader
relevance to targeting the VHL-HIF axis in other solid cancers beyond the VHL disease
setting.
UK VHL Systemic Therapy Forum and TRACERx Renal Study
We have established a UK VHL Systemic Therapy Forum, a national network of VHL
specialists and medical oncologists who prescribe Belzutifan. This group aims to:
• Share clinical experiences and develop treatment guidelines.
• Facilitate collaborative research into real-world outcomes and safety
Study Design and Data Collection
Within the TRACERx Renal protocol at The Royal Marsden Hospital, in collaboration
with Imperial College Hospitals, St. George’s, Guy’s & St Thomas’, and Barts Health
NHS Trust,
This research will provide critical real-world insights into Belzutifan’s effectiveness and
toxicity while advancing our understanding of resistance mechanisms in VHL disease with wider relevance to patients with cancer.
