Patients with von Hippel-Lindau (VHL) disease are prone to developing tumors — both cancerous and non-cancerous — throughout their life.

Traditionally, patients with VHL-related kidney cancer were treated with surgery or ablation (a process that involves sending heat or cold to destroy the tumor). However, last summer the Food and Drug Administration (FDA) approved Welireg (belzutifan), a targeted therapy for patients with VHL-associated kidney cancer.

VHL disease-associated kidney cancer can occur early, (such as when patients are in their) 20s to 30s and is often multifocal and in both kidneys, according to an expert at The University of Texas MD Anderson Cancer Center in Houston.

In an interview with CURE®, Dr. Eric Jonasch, a professor in the Department of Genitourinary Medical Oncology at The University of Texas MD Anderson Cancer Center in Houston, discussed the basics of VHL-related kidney cancer, and how Welireg is changing the treatment trajectory for the disease.

READ MORE: VHL-Related Kidney Cancer Survivor Reflects on Decades of Change for the Rare Condition

CURE®: Can you explain what VHL is, and the cancers associated with it?

Jonasch: VHL disease is an inherited autosomal dominant germline genetic disorder which affects around 10,000 people in the United States. People with VHL disease have benign and cancerous tumors in their eyes, brain, spine, pancreas, adrenal gland, kidneys, inner ear and reproductive organs.

VHL disease associated kidney cancer can occur early, (such as when patients are in their) 20s to 30s and is often multifocal and in both kidneys.

What is the current treatment standard(s) of care for VHL-associated kidney cancer, and who should be tested for VHL?

Prior to the approval of Welireg, individuals would undergo surgery for tumors that approached 3 centimeters in size or undergo ablation. Since August 2021, we have the option of treating patients with an oral drug called Welireg, which has been shown to result in significant shrinkage of kidney tumors in more than half of the patients treated with this drug.

If a person diagnosed with clear cell kidney cancer is younger than 46, has multiple kidney tumors, has hemangioblastomas (a type of non-cancerous tumor) in the brain, pancreatic cysts or pancreatic neuroendocrine tumors, or has a strong family history of VHL disease, germline testing for VHL disease is appropriate.

What is the grand takeaway that patients need to know about VHL-associated kidney cancer?

In 2022, although careful follow-up with regular scans and a strong treatment team experienced in VHL disease is still really important, with Welireg, we finally have a treatment that will likely decrease the number of surgeries and interventions patients need to undergo in their lifetimes.

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