Pfizer palbociclib potentially effective for certain types of appendix cancer

Researchers at University of California San Diego School of Medicine found an FDA-approved drug used to treat breast cancer has the potential to be an effective therapeutic for a specific type of appendix cancer. The clinical trial results, publishing in the October 16 online edition of the Journal of Clinical Oncology, showed the oral medication, Pfizer’s palbociclib, stabilized tumor growth and reduced blood tumor marker levels in patients with peritoneal mucinous carcinomatosis, or PMC. This form of cancer originates in the appendix and is often resistant to standard chemotherapy. The study cohort involved 16 participants diagnosed with PMC, most of whom had previously received other treatments without success. Researchers analyzed the genetic mutations present within these cancers and found that tumors with mutations in one specific gene, GNAS, responded well to the drug. In these patients, blood markers associated with cancer activity were reduced in more than 80% of patients. Laboratory results also showed that the drug slowed or stopped the multiplication of cancer cells, offering a well-tolerated alternative to chemotherapy, which is often associated with severe side effects. Next steps include investigating how combining this drug with both traditional chemotherapies and newer targeted therapies could further enhance its effectiveness.