Efficacy and tolerability of neoadjuvant therapy with Talimogene laherparepvec in cutaneous basal cell carcinoma: a phase II trial (NeoBCC trial)
We present a single-arm, phase II, neoadjuvant trial with the oncolytic virus talimogene laherparepvec (T-VEC) in 18 patients with difficult-to-resect cutaneous basal cell carcinomas. The primary end point, defined as the proportion of patients, who after six cycles of T-VEC (13 weeks), become resectable without the need for plastic reconstructive surgery, was already achieved after stage I (9 of 18 patients; 50.0%); thus the study was discontinued for early success. The objective response rate was 55.6% and the complete pathological response rate was 33.3%. Secondary end points included safety, relapse-free survival and overall survival, time to occurrence of new basal cell carcinomas and biological read outs. Only mild adverse events occurred. The 6-month relapse-free survival and overall survival rates were 100%. In two patients a new basal cell carcinoma was diagnosed. T-VEC led to a significant increase in cytotoxic T cells (P = 0.0092), B cells (P = 0.0004) and myeloid cells (P = 0.0042) and a decrease in regulatory T cells (P = 0.0290) within the tumor microenvironment. Together, neoadjuvant T-VEC represents a viable treatment option for patients with difficult-to-resect basal cell carcinomas (EudraCT no. 2018-002165-19).
Authors
- Victoria Bachmayr
- Nina Zila (ORCID: https://orcid.org/0000-0003-1193-1014)
- Thomas Silly
- Florian Halbritter (ORCID: https://orcid.org/0000-0003-2452-4784)
- Julia Tittes (ORCID: https://orcid.org/0000-0003-0844-3626)
- Matthias Farlik (ORCID: https://orcid.org/0000-0003-0698-2992)
- Anna Kusienicka (ORCID: https://orcid.org/0000-0003-0933-2610)
- Ana-Maria Stepan
- Philipp Tschandl (ORCID: https://orcid.org/0000-0003-0391-7810)
- Julia Maria Ressler (ORCID: https://orcid.org/0000-0001-8602-0878)
- Lisa E. Shaw (ORCID: https://orcid.org/0000-0003-2273-7655)
- Christoph Höeller (ORCID: https://orcid.org/0000-0003-1192-0227)
- Peter Petzelbauer (ORCID: https://orcid.org/0000-0001-7080-5259)
- Werner Haslik (ORCID: https://orcid.org/0000-0002-2731-2720)
- Maud Plaschka (ORCID: https://orcid.org/0000-0003-1137-7623)
- Rita Silmbrod (ORCID: https://orcid.org/0009-0005-2506-9893)
- Florian Roka
- Rainer Kunstfeld (ORCID: https://orcid.org/0009-0002-5678-5057)
- Wolfgang J. Weninger (ORCID: https://orcid.org/0000-0003-0575-0073)
- Franz Koenig
Institutions
- Statistics Austria (AT)
- Hochschule Campus Wien (AT)
- St Anna Children's Hospital (AT)
- St. Anna Children's Cancer Research Institute
- Medical University of Vienna (AT)
Publication Details
- Journal
- Nature Cancer
- Published
- 2025-01-16
- DOI
- https://doi.org/10.1038/s43018-024-00879-x
- Citations
- 10
- Primary Topic
- Nonmelanoma Skin Cancer Studies
- Type
- article
- Field-Weighted Citation Impact
- 13.54
Funders
- Alex's Lemonade Stand Foundation for Childhood Cancer
- Amgen
- Österreichischen Akademie der Wissenschaften
- Austrian Science Fund
- Universität Wien
- Medizinische Universität Wien