Kidney Cancer Experts Develop New Strategies to Advance Clinical Trials for Rare Subtypes
Key Insights
Researchers at the 2025 Kidney Cancer (search) Research Summit discussed innovative approaches to overcome clinical trial challenges in rare kidney cancer subtypes, including remote principal investigator models for multicenter studies.
Experts emphasized the need for pharmaceutical industry partnerships and larger consortia to compile research data and translate laboratory discoveries into clinical trials for rare kidney cancer (search) variants.
The kidney cancer (search) community is adopting strategies from other cancer types to conduct trials for molecularly defined therapeutic vulnerabilities that occur in smaller patient populations.
Kidney cancer (search) researchers are developing innovative clinical trial strategies to address the unique challenges of studying rare cancer subtypes, according to discussions at the 2025 Kidney Cancer Research Summit. The conference featured sessions focused on overcoming barriers to advancing care for patients with uncommon kidney cancer variants including papillary, chromophobe, translocation, and unclassified subtypes.
Remote Trial Management Shows Promise
A. Ari Hakimi, MD, a urologic surgeon at Memorial Sloan Kettering Cancer Center who moderated key sessions at the summit, highlighted an innovative approach proposed by W. Kimryn Rathmell, MD, PhD, chief executive officer of The Ohio State University Comprehensive Cancer Center (search). The strategy involves conducting multicenter trials linked by a principal investigator who meets with patients remotely.
"That was a cool strategy, and there's some logistics about that, but that is a good strategy that we could try to employ in cancer," Hakimi noted during his discussion with CancerNetwork at the conference.
Balancing Trial Size and Statistical Power
The Clinical Trial Design and Execution session addressed a fundamental challenge in rare cancer research: determining optimal trial design. Researchers debated the merits of larger phase 3 trials, which take longer to accrue patients, versus smaller trials that may lack statistical power to detect meaningful differences.
"There was a debate between doing larger phase 3 trials, even though it takes longer to accrue, vs doing smaller trials that may be underpowered. We heard the pros and cons of both of those," Hakimi explained.
Industry Partnerships and Consortia Critical for Progress
Translating laboratory discoveries into clinical applications requires collaboration with pharmaceutical companies and larger research consortia, according to summit discussions. The Department of Defense (search) and other funding sources have enabled significant progress in understanding genetic and immunologic drivers of different kidney cancer (search) subtypes.
"The major goals are going to be translating the discoveries that we're making in the lab to clinical trials," Hakimi said. "There's a strong understanding—we had a premeeting session on working with industry and working with larger consortia to help bring together information that we're learning from individual patients, from small studies to a broader trial setting."
Cross-Cancer Strategies Offer Solutions
Experts emphasized that challenges in rare kidney cancer (search) subtypes are not unique to this disease area. Successful strategies from other cancer types can be adapted for kidney cancer research, particularly for molecularly defined therapeutic vulnerabilities that occur in smaller patient populations.
"Rare events or therapeutic vulnerabilities that occur in smaller numbers of patients are common across cancers. There are strategies that you can use to conduct trials in those types of scenarios, and we can develop molecularly defined strategies that may transcend specific types of cancers," Hakimi noted.
Community Engagement Essential for Patient Advocacy
Researchers stressed the importance of staying current with available clinical trials and treatment options to effectively advocate for patients with rare kidney cancers. This includes monitoring social media, company websites, and foundation resources to identify opportunities for patient enrollment.
"As a community, if we're looking to help enroll and advocate for patients with rare diseases, we need to be aware of what is out there," Hakimi emphasized. "Just being part of a bigger community and looking to enroll patients with rare events and rare diseases, into these bigger things [can help] because if we don't do it, no one else will."
The summit concluded with optimism about overcoming current challenges through strategic prioritization of promising drug targets linked to cancer biology and continued collaboration across the broader cancer research community.