Genentech’s GDC0449 – a Promising Treatment for Basal Cell Cancer Under Clinical Trials
GDC-0449 is a synthetic Hedgehog Pathway Inhibitor meaning that it blocks signals in the Hedgehog Pathway that is known to play a role in tumor growth. Specifically GDC-0449 hinders the activities of the Hedgehog-ligand cell surface receptors PTCH and/or SMO and thus restrains Hedgehog signaling.
GDC-0449 is being actively investigated by Genentech as a single-agent therapy for patients with operable basal cell carcinoma (BCC). The molecule has been developed by Curis Inc and licensed to Genentech for Clinical development.
Hedgehog Pathway and its Role in Basal Cell Cancer
Hedgehog pathway is normally active during early embryonic development and plays a critical role in channelizing cell differentiation and growth. Abnormal activation or lack of control of the Hedgehog pathway is believed to play a critical role in the growth of certain cancer cells, including in basal cell carcinoma, medulloblastoma and other cancers.
Basal Cell Carcinoma is one of the the most common form of all cancers. Every year some 1 million Americans are diagnosed with BCC (this number is 2.5 million worldwide). BCC grows from the basal cells, a layer of cells found at the bottom of the epidermis (outer skin layer).
GDC-0449 is a small molecule drug developed by Curis Inc that is designed to act as an inhibitor of the Hedgehog Pathway. The drug has been licensed to Genentech to conduct clinical trials. Genentech has introduced a Phase II clinical trial of GDC-0449. The company recently completed a Phase II clinical trial of GDC-0449 in advanced ovarian cancer. The trial showed that the average time for the disease to progress was 7.5 months for patients who received GDC-0449 compared to 5.8 months for patients who received placebo.
Conducting of trial of GDC-0449 will help in exploring the treatment of basal cell cancer. Curis Inc and Genentech are also in collaboration with NCI to conduct various clinical trials of GDC-0449 in the treatment of pancreatic, small cell lung cancer, esophageal, stomach, breast and prostate cancers along with several others.