Phase 1 LITESPARK-001 study of belzutifan in participants with advanced solid tumors: results of the glioblastoma expansion cohort
Summary Recurrent isocitrate dehydrogenase (IDH) wild-type glioblastoma has no established standard-of-care treatment. Preclinical models have demonstrated that hypoxia-inducible factor-2 alpha (HIF-2α) contributes to stabilizing the hypoxic environment in glioblastoma. It was thus hypothesized that the first-in-class HIF-2α inhibitor, belzutifan, may improve outcomes in patients with recurrent IDH wild-type glioblastoma. The glioblastoma cohort of the phase 1 LITESPARK-001 trial enrolled participants who had received radiotherapy and temozolomide. Although targeting HIF-2α may have a role in advancing treatment options for patients with glioblastoma, single-agent belzutifan did not have antitumor activity in this cohort.
Authors
- David F. McDermott (ORCID: https://orcid.org/0000-0002-2675-5095)
- Kyriakos P. Papadopoulos (ORCID: https://orcid.org/0000-0002-0667-2620)
- Roy E. Strowd (ORCID: https://orcid.org/0000-0001-6651-5267)
- Todd Michael Bauer (ORCID: https://orcid.org/0000-0002-3078-5043)
- Rodolfo F. Perini (ORCID: https://orcid.org/0009-0002-4400-5260)
- Ke Chen (ORCID: https://orcid.org/0000-0002-2098-1921)
- Yanfang Liu (ORCID: https://orcid.org/0000-0002-4091-7679)
- Macarena de la Fuente
Institutions
- Merck & Co., Inc., Rahway, NJ, USA (United States) (US)
- Beth Israel Deaconess Medical Center (US)
- South Texas Accelerated Research Therapeutics (US)
- Tennessee Oncology (US)
- Sylvester Comprehensive Cancer Center (US)
- Wake Forest University (US)
Publication Details
- Journal
- Neuro-Oncology Advances
- Published
- 2025-11-19
- DOI
- https://doi.org/10.1093/noajnl/vdaf242
- Citations
- 2
- Primary Topic
- Cancer, Hypoxia, and Metabolism
- Type
- article
- Field-Weighted Citation Impact
- 1.87
Funders
- Bristol-Myers Squibb
- Eli Lilly and Company
- Pfizer
- Regeneron Pharmaceuticals
- Exelixis
- Incyte
- Eisai
- PharmaMar
- Genentech