A prospective phase II trial of ONYX-015 adenovirus and chemotherapy in recurrent squamous cell carcinoma of the head and neck (the Baylor experience)

Ann Surg Oncol. 2000 Sep;7(8):588-92. doi: 10.1007/BF02725338.


Background: The E1-b attenuated adenovirus, ONYX-015 (Onyx Pharmaceuticals, Richmond, CA), has demonstrated antitumoral activity in patients with recurrent squamous cell carcinoma of the head and neck. This study evaluated the effects of intratumoral ONYX-015 injection combined with systemic chemotherapy.

Methods: Inclusion criteria included: (1) recurrent squamous cell carcinoma of the head and neck, not surgically salvageable, (2) target tumor amenable to direct injection, and (3) no prior chemotherapy for recurrent disease. Patients received ONYX-015 (10(10) plaque-forming units) intratumorally for 5 days, cisplatin (80 mg/m2) on day 1, and 5-fluorouracil (800-1000 mg/m2) on days 1-5. This cycle was repeated every 3 weeks. Serial physical examination and computed tomography were used to assess tumor size and treatment response.

Results: Fourteen patients were enrolled, and nine patients were evaluable for response at the time of enrollment. The mean age of the evaluable patients was 60.8 years (range, 46-71 years). Mean maximum tumor diameter was 4.8 cm (range, 1.9-10.5 cm). Treatment-related toxicity included nausea (n = 7, 77.8%), vomiting (n = 5, 55.6%), mucositis (n = 5, 55.6%), pain at the injection site (n = 5, 55.6%), constipation (n = 4, 44.4%), and fatigue (n = 4, 44.4%). Locoregional tumor control was obtained in all nine patients (100%) (mean observation time, 157 days). Complete clinical response was seen in three patients (33.3%), partial response was seen in three patients (33.3%), minor response was seen in one patient (11.1%), and two patients (22.2%) had stable disease. Median time to local progression of disease has not been reached (range, 35-356 days).

Conclusions: ONYX-015 adenovirus plus systemic cisplatin and 5-fluorouracil provides antitumor activity and local tumor control in patients with recurrent squamous cell carcinoma of the head and neck. This novel treatment approach offers hope for patients with limited treatment alternatives and provides the foundation for a phase III clinical trial.

Publication types

  • Clinical Trial
  • Clinical Trial, Phase II

MeSH terms

  • Adenoviruses, Human / genetics*
  • Aged
  • Antimetabolites, Antineoplastic / adverse effects
  • Antimetabolites, Antineoplastic / therapeutic use
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Carcinoma, Squamous Cell / drug therapy
  • Carcinoma, Squamous Cell / therapy*
  • Cisplatin / adverse effects
  • Cisplatin / therapeutic use
  • Disease Progression
  • Female
  • Fluorouracil / adverse effects
  • Fluorouracil / therapeutic use
  • Gene Deletion
  • Genetic Therapy / methods*
  • Head and Neck Neoplasms / drug therapy
  • Head and Neck Neoplasms / therapy*
  • Humans
  • Injections
  • Male
  • Middle Aged
  • Neoplasm Recurrence, Local / drug therapy
  • Neoplasm Recurrence, Local / therapy*
  • Remission Induction


  • Antimetabolites, Antineoplastic
  • Cisplatin
  • Fluorouracil