Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

KIDNEY CANCER

Belzutifan: a novel therapy for von Hippel–Lindau disease

Nature Reviews Nephrology volume 18, pages 205–206 (2022)Cite this article

Subjects

A recent clinical trial reports promising efficacy and safety data for belzutifan in patients with von Hippel–Lindau (VHL) disease–associated renal cell carcinoma. On the basis of these results, belzutifan became the first therapeutic agent to be approved for the systemic treatment of cancer associated with VHL disease.

Refers to Jonasch, E. et al. Belzutifan for renal cell carcinoma in von Hippel–Lindau disease. N. Engl. J. Med. 385, 2036–2046 (2021).

This is a preview of subscription content

Access options

Buy article

Get time limited or full article access on ReadCube.

$32.00

Buy

All prices are NET prices.

References

  1. Escudier, B. et al. Renal cell carcinoma: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann. Oncol. 30, 706–720 (2019).

    CAS  Article  Google Scholar 

  2. Jonasch, E. et al. Belzutifan for renal cell carcinoma in von Hippel–Lindau disease. N. Engl. J. Med. 385, 2036–2046 (2021).

    CAS  Article  Google Scholar 

  3. Kaelin, W. G. Jr. Molecular basis of the VHL hereditary cancer syndrome. Nat. Rev. Cancer 2, 673–682 (2002).

    CAS  Article  Google Scholar 

  4. Gnarra, J. R. et al. Mutations of the VHL tumour suppressor gene in renal carcinoma. Nat. Genet. 7, 85–90 (1994).

    CAS  Article  Google Scholar 

  5. Stebbins, C. E., Kaelin, W. G. Jr & Pavletich, N. P. Structure of the VHL–ElonginC–ElonginB complex: implications for VHL tumor suppressor function. Science 284, 455–461 (1999).

    CAS  Article  Google Scholar 

  6. Kondo, K., Kim, W. Y., Lechpammer, M. & Kaelin, W. G. Jr. Inhibition of HIF2α is sufficient to suppress pVHL-defective tumor growth. PLoS Biol. 1, E83 (2003).

    Article  Google Scholar 

  7. Walther, M. M., Lubensky, I. A., Venzon, D., Zbar, B. & Linehan, W. M. Prevalence of microscopic lesions in grossly normal renal parenchyma from patients with von Hippel-Lindau disease, sporadic renal cell carcinoma and no renal disease: clinical implications. J. Urol. 154, 2010–2014, discussion 2014–2015 (1995).

    CAS  Article  Google Scholar 

  8. Chen, W. et al. Targeting renal cell carcinoma with a HIF-2 antagonist. Nature 539, 112–117 (2016).

    CAS  Article  Google Scholar 

  9. Choueiri, T. K. et al. Inhibition of hypoxia-inducible factor-2α in renal cell carcinoma with belzutifan: a phase 1 trial and biomarker analysis. Nat. Med. 27, 802–805 (2021).

    CAS  Article  Google Scholar 

  10. Comino-Méndez, I. et al. Tumoral EPAS1 (HIF2A) mutations explain sporadic pheochromocytoma and paraganglioma in the absence of erythrocytosis. Hum. Mol. Genet. 22, 2169–2176 (2013).

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Department of Urology, Peking University First Hospital, Beijing, China

    Jingcheng Zhou & Kan Gong

  2. Institute of Urology, Peking University, Beijing, China

    Jingcheng Zhou & Kan Gong

Authors
  1. Jingcheng Zhou
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Kan Gong
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Kan Gong.

Ethics declarations

Competing interests

The authors declare no competing interests.

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Zhou, J., Gong, K. Belzutifan: a novel therapy for von Hippel–Lindau disease. Nat Rev Nephrol 18, 205–206 (2022). https://doi.org/10.1038/s41581-022-00544-5

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41581-022-00544-5

Search

Advanced search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing