GFRα4 May Represent a Unique Therapeutic Target in Medullary Thyroid Cancer

Roger B. Cohen, MD, is a professor of medicine at the Hospital of the University of Pennsylvania (Penn) and associate director of clinical research at Penn's Abramson Cancer Center. Along with his colleagues at Penn, he is investigating a novel CAR T-cell product as a treatment for adults with medullary thyroid cancer (MTC).

MTC is a rare disease, accounting for 1% to 2% of thyroid cancer cases. The prognosis for these patients is poor, and there are few treatment options for those with relapsed/refractory disease.

Donald Siegel, MD, PhD, Dr. Cohen's scientific collaborator, discovered GFRα4, a protein uniqely expressed on the parafollicular cells that become malignant in MTC. In the clinical trial, Dr. Cohen will assess whether a one-time infusion of autologus GFRα4 CAR T cells can kill tumor cells without causing the toxicities common to immunotherapy.