Moffitt is part of a national clinical trial that could lead to a new therapy for patients with cutaneous squamous cell carcinoma, the second most common type of skin cancer. Cutaneous squamous cell carcinoma, which develops from genetic damage caused by exposure to ultraviolet light, sees an estimated one million new cases diagnosed annually. More than 95 percent of patients are cured by surgery and radiation at the disease’s early stages. But for the fraction who progress to advance disease, there are no approved therapies approved. 

Dr. Nikhil I. Khushalani, Vice Chair of the Department of Cutaneous Oncology at Moffitt, and Dr. Christine Chung, Chair of Head and Neck Oncology Department served as site investigators for the national clinical trial of the immunotherapy drug cemiplimab, a PD-1 checkpoint inhibitor. PD-1 is a protein found on immune cells. When it binds to the protein, PD-L1, it shuts off the body’s immune response. Cemiplimab blocks that bond from occurring, allowing the immune cells to attack cancer cells.

“Nearly half of the patients who participated in this trial saw their tumors shrink,” said Dr. Khushalani. “This is the first treatment to have a significant impact in a favorable way and is well tolerated, similar to other immunotherapy drugs of this class. Our hope is this will lead to the FDA-approval of the drug.”

The results of the phase 2 study published in the New England Journal of Medicine showed 28 of 59 patients with metastatic disease (47.5 percent) had an objective response to cemiplimab, defined as at least 30 percent tumor shrinkage observed via imaging. Four were complete responses, 24 had partial responses, and 82 percent of responders remain on the drug.

The United States Food and Drug Administration (FDA) has granted breakthrough therapy status for cemiplimab, providing a faster potential route for FDA approval. Dr. Khushalani is hopeful that the drug will be FDA-approved by the end of October.