TGFβ Pathway Inhibitor Improves Survival in Patients With Pancreatic Cancer

Galunisertib plus gemcitabine may improve overall survival and progression-free survival compared to gemcitabine alone in patients with pancreatic cancer, according to a study presented at the American Association for Cancer Research Annual Meeting (April 16-20, 2016; New Orleans, LA).

Pancreatic cancer is one of the deadliest forms of cancer, with very few patients surviving 5 years after initial diagnosis. This is due in large part to the presence of the transforming growth factor-beta (TGFβ) signaling pathway, which is responsible for the disease’s growth and spread.


Related Content

Improved Outcomes for NSCLC Patients with Immunotherapy versus Chemotherapy

Immunotherapies and the Future of Cancer Care


Researchers led by Davide Melisi, MD, PhD, University of Verona, Italy, conducted a study evaluating the efficacy and safety of galunisertib, a novel therapeutic agent that inhibits the TGFβ pathway, in combination with gemcitabine for the treatment of patients with unresectable pancreatic cancer.

A total of 156 patients with stage 2 to 4 unresectable pancreatic cancer were enrolled as part of the study and were randomly assigned to receive galunisertib plus gemcitabine or gemcitabine plus a placebo. Galunisertib was administered orally twice daily for 14 days, followed by 14 days of no treatment.

Treatment that included galunisertib was found to significantly improve survival compared with placebo (9.10 months vs 7.59 months). Median progression-free survival was also significantly improved with the addition of galunisertib (3.65 months vs 2.79 months with gemcitabine plus placebo).

In patients with TGFβ levels of 4224 pg/ml or lower, overall survival was 10.9 months with galunisertib plus gemcitabine compared with 7.2 months with gemcitabine and placebo. Median progression-free survival was similarly improved (3.9 months vs 2.8 months).

The most common grade 3 or 4 treatment related adverse events associated with treatment with galunisertib plus gemcitabine or with gemcitabine and placebo were anemia (7.8% vs 13.5%), neutropenia (32.0% vs 26.9%), and thrombocytopenia (7.8% vs 9.6%).

Researchers concluded that adding galunisertib to gemcitabine treatment significantly improves outcomes in patients with unresectable pancreatic cancer. The findings also suggest that patients with lower TGFB1 levels may be the most likely to benefit.