Promising Results from Antibody-Drug Conjugate in Treating Rare Blood Cancer

A recent study has showcased encouraging safety profiles and response rates of a novel antibody-drug conjugate (ADC), pivekimab sunirine (PVEK), in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Led by researchers at The University of Texas MD Anderson Cancer Center, the Phase I/II trial demonstrated significant efficacy, especially in newly diagnosed patients. Out of 33 newly diagnosed BPDCN cases, the treatment achieved an overall response rate of 85%, with 70% achieving complete remission and a median overall survival of approximately 16.6 months.

BPDCN is a rare, aggressive blood cancer that often involves the skin, bone marrow, and lymph nodes, and currently has limited therapeutic options. The study data, presented at the 2025 ASCO Annual Meeting, suggest that PVEK might soon become a new standard of care. Compared to the existing treatment, tagraxofusp-erzs—which targets the CD123 protein present on BPDCN cells—PVEK offers a targeted approach by delivering cytotoxic drugs directly to cancer cells, leading to their death.

The global multi-center trial enrolled 84 adult patients with CD123-positive BPDCN, including both newly diagnosed and relapsed/refractory cases. Participants received the treatment intravenously every 21 days on an outpatient basis. The most common side effect was reversible peripheral edema, which was manageable.

Researchers are optimistic about the potential of combining PVEK with other therapies to enhance treatment outcomes further. The trial received support from pharmaceutical company AbbVie, with researchers also serving on advisory boards and as consultants.

Overall, these promising results indicate that PVEK could revolutionize treatment strategies for BPDCN, offering hope for improved patient outcomes in this challenging disease.

Source: https://medicalxpress.com/news/2025-06-antibody-drug-conjugate-safety-response.html