Selected Grantee Publications
Pembrolizumab and Cabozantinib in Recurrent Metastatic Head and Neck Squamous Cell Carcinoma: A Phase 2 Trial
Saba et al., Nature Medicine. 2023.
https://www.doi.org/10.1038/s41591-023-02275-x
A multicenter clinical trial was conducted in 33 evaluable (36 enrolled) patients with recurrent metastatic head and neck squamous cell carcinoma (RMHNSCC) on a regimen combining cabozantinib, a tyrosine kinase inhibitor, with the standard of care of anti–programmed cell death protein 1 agent pembrolizumab. Results showed that 17 patients (52%) exhibited partial response and 13 (39%) exhibited stable disease, with an overall clinical benefit rate of 91%. Median progression-free survival (PFS) was 14.6 months, and the 1-year PFS was 54%. The pembrolizumab and cabozantinib regimen was well tolerated in patients with RMHNSCC. The promising clinical benefit warrants further investigation. Supported by ORIP (S10OD021644), NCI, and NIDCR.
Advancing Human Disease Research with Fish Evolutionary Mutant Models
Beck et al., Trends in Genetics. 2021.
https://pubmed.ncbi.nlm.nih.gov/34334238/
Model organism research is essential to understand disease mechanisms. However, laboratory-induced genetic models can lack genetic variation and often fail to mimic disease severity. Evolutionary mutant models (EMMs) are species with evolved phenotypes that mimic human disease. They have improved our understanding of cancer, diabetes, and aging. Fish are the most diverse group of vertebrates, exhibiting a kaleidoscope of specialized phenotypes, many that would be pathogenic in humans but are adaptive in the species' specialized habitat. Evolved compensations can suggest avenues for novel disease therapies. This review summarizes current research using fish EMMs to advance our understanding of human disease. Supported by ORIP (R01OD011116), NIA, NIDA, and NIGMS.