What is ASCO GU?
Every year, clinicians and researchers from around the world gather to share the newest discoveries in cancer research. One of the most important meetings for kidney cancer research is the American Society of Clinical Oncology (ASCO) Genitourinary (GU) Cancers Symposium, held each year in San Francisco, California. The conference focuses on cancers that affect the urinary and reproductive systems: kidney, bladder, prostate, and testicular cancers. For the VHL community, this meeting is especially meaningful because many recent advancements in the most common type of kidney cancer, clear cell renal cell carcinoma (ccRCC), are tied directly to research into the VHL gene and HIF pathway.
Because mutations in the VHL gene are present in the majority of clear cell kidney cancers, discoveries made through VHL research continue to influence treatment strategies for kidney cancer worldwide.
This year, the central theme of ASCO GU was “Patient-Centered Care: From Discovery to Delivery”, emphasizing the importance of translating the latest research breakthroughs into improved clinical practice and patient outcomes.
Here are some key highlights from this year’s meeting:
New HIF-2α inhibitors are under development
One of the most exciting areas of research for the VHL community is the emerging development of a new class of medicines called “HIF-2α inhibitors”. When the VHL gene is not working properly, HIF-2α builds up inside cells and activates genes that help tumors grow and form new blood vessels. These drugs work by binding directly to HIF-2α and preventing it from activating genes that promote tumor growth. The very first HIF-2α inhibitor to earn FDA approval was belzutifan, and now similar experimental drugs are making it to clinical trials.
One example is casdatifan, which is showing promise for patients with metastatic kidney cancer who have already received several prior treatments.
Researchers found that patients whose erythropoietin (EPO) levels dropped the most were more likely to benefit from casdatifan treatment. Because HIF-2α directly controls EPO production, lower EPO levels suggest the drug is successfully blocking (inhibiting) HIF-2α.
Why it matters: Although research on these new drug candidates is still underway, if successful in ongoing studies, they could provide an alternative treatment option for individuals with VHL who either cannot tolerate the side effects of belzutifan or whose tumors do not respond to it.
Kidney cancer treatment is becoming more personalized
Doctors now understand that not all kidney tumors behave in the same way. Even tumors that look similar under the microscope can differ in the genetic changes that drive them, the immune cells that surround them, and how likely they are to respond to a given therapy. Because of this, clinicians and researchers are learning how to tailor treatments to the specific biology of a person’s tumor. The presentations at ASCO GU led to productive discussions about how to more efficiently detect and treat tumors, which treatments to use, and how to balance treatment benefits with side effects.
Why it matters: VHL patients can develop multiple kidney tumors over time, and a more personalized approach may help doctors better distinguish which tumors need intervention, which can be safely monitored, and which are most likely to work based on the unique characteristics of each individual tumor.
Belzutifan is highly effective in ccRCC
New data presented at the meeting showed that belzutifan is highly effective in treating ccRCC (and VHL–associated tumors), both alone and in combination with other therapies. Researchers are also exploring the benefits of using belzutifan in earlier stages of kidney cancer, various factors that might increase the severity of side effects, and why belzutifan doesn’t work on some tumors.
Why it matters: Belzutifan is the first therapy developed directly from decades of research into VHL, and ongoing research will help doctors understand how to use this drug most effectively and build upon its success.
Artificial Intelligence (AI) is reshaping oncology
AI is becoming a routine part of research and clinical practice, and is helping doctors identify tumors earlier, analyze medical images more quickly, and identify hidden patterns in patient data. These technologies have the potential to reduce the time and costs of developing new therapies and simplify clinical decision-making.
Why it matters: Individuals with VHL require lifelong monitoring and imaging to detect tumors. AI tools may eventually help doctors detect tumors earlier, identify subtle changes in imaging scans, and predict which tumors are likely to grow.
Highlights from our community
In addition to broader advances in kidney cancer research, several members of the VHL research community presented new findings at the meeting:
Eric Jonasch, MD (The University of Texas MD Anderson Cancer Center)
- Kidney cancer treatment combination studies and clinical trials
- Phase 1B/2 study of combination 177Lu girentuximab plus cabozantinib and nivolumab in treatment-naïve patients with advanced clear cell RCC
- A randomized phase 2 trial of nivolumab, relatlimab plus ipilimumab vs. nivolumab plus ipilimumab in first-line advanced renal cell carcinoma
- A multi-institution analysis of outcomes with first-line ipilimumab and nivolumab for 514 patients with metastatic clear cell renal cell carcinoma by number of doses
- Clinical outcomes and tolerability of ipilimumab/nivolumab in older (≥70 years) versus younger patients with metastatic clear cell RCC: A multi-institutional analysis of 514 patients
- Sarcomatoid versus rhabdoid dedifferentiation and histologic grade-specific outcomes in metastatic clear cell renal cell carcinoma (mccRCC): A multi-institutional analysis of 514 patients
- A phase II trial of nivolumab, ipilimumab and cabozantinib in patients with brain metastases from renal cell carcinoma
- Metastasis-directed therapy with or without pembrolizumab for oligometastatic clear cell renal cell carcinoma: Pooled analysis of two prospective single-arm phase II trials
- Circulating KIM-1 and ctDNA as prognostic markers in oligometastatic clear cell renal cell carcinoma (ccRCC): The K-COMPASS model
- TANKeR-70: TGF-β-receptor type 2 knockout allogeneic NK cells for renal cell carcinoma expressing CD70
- Ivonescimab in metastatic clear cell renal cell carcinoma (mccRCC) after immune checkpoint inhibitor therapy: The phase II IVORY trial
- A phase II study bolstering outcomes by optimizing immunotherapy strategies with evolocumab and nivolumab in patients with metastatic renal cell carcinoma (BOOST-RCC)
- Patient wellness and support services
James Brugarolas, MD, PhD (The University of Texas Southwestern Medical Center)
- Kidney cancer treatment strategies
- Pathologic findings and clinical outcomes after immune checkpoint blockade in renal cell carcinoma patients undergoing deferred consolidative nephrectomy
- Biomarker analysis from the phase 2 LITESPARK-013 study of 2 different belzutifan (bel) doses in participants (pts) with advanced clear cell renal cell carcinoma (ccRCC)
- A phase II study bolstering outcomes by optimizing immunotherapy strategies with evolocumab and nivolumab in patients with metastatic renal cell carcinoma (BOOST-RCC)
Kan Gong, MD, PhD (Peking University First Hospital)
- Belzutifan clinical trial
Looking Forward
The biggest takeaway from ASCO GU 2026 was that the pace of kidney cancer is accelerating and becoming more precise. Effective therapies like belzutifan are becoming mainstays in ccRCC treatment, doctors are learning how to better use and combine treatments, and artificial intelligence is helping amplify these efforts.
The progress we are seeing today is a reminder that discoveries made through VHL research continue to shape the future of kidney cancer treatment. As the field continues to evolve, the VHL Alliance remains committed to ensuring that the voice of the VHL community is part of the conversation. We look forward to carrying this momentum into the ASCO Annual Meeting later this year, where the VHL Alliance will host a booth to engage with researchers, clinicians, and partners across the oncology community.
These photos were pulled from a public gallery and are owned by ASCO
This post is brought to you with support from:
