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Targeted Therapies Provide Treatment Options for Poorly Differentiated Pancreatic Neuroendocrine Carcinomas
Marine Gilabert1, Young Soo Rho, Petr Kavan
1Department of Medical Oncology, Paoli-Calmettes Institute, Marseille, France.
Abstract:
Poorly differentiated pancreatic neuroendocrine carcinoma (PD pNECs) is a rare disease that has a poor prognosis and is treated with systemic chemotherapy as the standard of care. We present 6 cases of chemo-naïve patients diagnosed with PD pNECs who refused systemic chemotherapy and received targeted therapies with sunitinib (37.5 mg/day, 5 patients) or the mammalian target of rapamycin (mTOR) inhibitor everolimus (10 mg/day, 1 patient) as the first-line treatment. We evaluated the drugs' toxicities and survival. The median age of the patients was 55 years (4 males, 2 females, functioning tumor in 1 of 6 patients). The median of the Ki67 index was 45% (range 20-80). Targeted therapies were combined with somatostatin analogues in 4 of 6 patients (30 mg Sandostatine LAR monthly). Toxicities (acute and late) were manageable and no toxicities necessitated cessation of treatment. All patients had progression-free survival during the 15-month treatment and an overall survival of more than 2 years after diagnosis. Even though this is a small cohort of selected patients, we conclude that sunitinib or everolimus are both feasible and safe and have encouraging results of efficacy as first-line therapies for PD pNEC.
Insights
Targeted therapies sunitinib or everolimus show promise for poorly differentiated pancreatic neuroendocrine carcinoma (PD pNEC) when chemotherapy is refused. These treatments were safe, manageable, and demonstrated encouraging efficacy in a small patient cohort.
Area of Science:
- Oncology
- Endocrinology
- Pharmacology
Background:
- Poorly differentiated pancreatic neuroendocrine carcinoma (PD pNEC) is a rare malignancy with a poor prognosis.
- Systemic chemotherapy is the current standard of care for PD pNEC.
- Limited treatment options exist for patients who decline conventional chemotherapy.
Purpose of the Study:
- To evaluate the safety and efficacy of targeted therapies sunitinib and everolimus as first-line treatment for chemo-naïve PD pNEC patients.
- To assess drug toxicities and survival outcomes in this patient population.
Main Methods:
- Retrospective analysis of 6 chemo-naïve PD pNEC patients.
- First-line treatment with sunitinib (n=5) or everolimus (n=1).
- Combination therapy with somatostatin analogues in 4 patients; toxicity and survival data collected.
Main Results:
- Median patient age was 55 years; median Ki67 index was 45%.
- Toxicities were manageable and did not require treatment cessation.
- All patients achieved progression-free survival during treatment and over 2 years of overall survival.
Conclusions:
- Sunitinib and everolimus are feasible and safe first-line options for PD pNEC.
- These targeted therapies show encouraging efficacy in selected patients who refuse chemotherapy.
- Further investigation in larger cohorts is warranted to confirm these findings.
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