NIH R01 · 2024
A novel therapy for pancreatic neuroendocrine tumors
The incidence of the pancreatic neuroendocrine tumor (PNETs) has increased over the past two decades. The principal treatment for localized PNETs is surgical resection; however, there is no effective therapy for patients with advanced unresectable or metastatic disease. The progression-free survival rate is at the best 11 months with FDA approved agent Everolimus compared to 4.6 months with the placebo. However, the majority of the patients develop drug resistance and there is a void in our understanding of the mechanisms of resistance in PNETs. Frequent mutations in MEN1 (44%), DAXX/ATRX (43%), mTOR (15%) pathway genes and Von Hippel Lindau disease (VHL) alongside several other hereditary…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.