Roswell Park Studies Highlight Emerging Treatment Options for Neuroendocrine Tumors

Renuka Iyer, MD

Two new studies led by Renuka Iyer, MD, Section Chief for Gastrointestinal Oncology at Roswell Park Comprehensive Cancer Center, highlight possible new treatment options for patients with neuroendocrine tumors (NETs) — a rare and hard-to-treat cancer.

The first publication focuses on a cancer immunotherapy developed by two of her Roswell Park colleagues that has been shown to help patients with brain tumors live longer.

Robert Fenstermaker, MD, Chair of Neurosurgery, and Michael Ciesielski, PhD, Assistant Professor of Neurosurgery, have reported that the novel vaccine they developed, SurVaxM, combined with standard therapy, gave brain tumor patients significantly longer survival rates compared with those who received standard therapy alone.

They stained NET specimens for the molecule the vaccine targets — survivin, a protein often found at high levels in cancerous tumors, where it promotes cancer cell survival through proliferation and metastasis. They found an unusually high level in the specimens, and brought in Dr. Iyer to take it to the next step.

Dr. Iyer and colleagues pursued further studies exploring whether the protein might serve as a prognostic and potentially therapeutic marker in patients with NETs. They have shared those findings in an article in the journal Oncotarget.

The team discovered survivin in 52% of the neuroendocrine samples they studied.

“We saw that the SurVaxM vaccine has the potential to help half our patients with NETs, especially patients with more aggressive tumors and those whose tumors originated in their lungs, for whom options are urgently needed,” Dr. Iyer says.

Dr. Iyer secured funding from the Neuroendocrine Tumor Research Foundation (NETRF) in 2019 for a clinical trial (NCT03879694) of SurVaxM in patients with NETs; that study is open and enrolling new patients.

In pursuit of another novel treatment for this rare cancer, Dr. Iyer also led a team of researchers from Roswell Park, Ohio State University Comprehensive Cancer Center and Memorial Sloan Kettering Cancer Center to study nintedanib (brand names Ofev and Vargatef), an oral antiangiogenic agent that targets key tumor cell-signaling pathways. Nintedanib inhibits the fibroblast growth factor receptor (FGFR), which is highly expressed in those tumors. For that reason, the team hypothesized, it could be active in patients with NETS.

The findings have been published in Cancer, a journal of the American Cancer Society.

“We found that nintedanib is effective as a treatment option for NET patients and can slow the cancer growth for almost a year. Nintedanib was well tolerated and delayed deterioration in quality of life,” Dr. Iyer says.

The most significant finding of this study was that nintedanib can slow cancer growth even when the patients have had many prior therapies. These results will help inform future trials targeting novel pathways, such as serotonin signaling, especially for nonpancreatic NET patients, for whom options are very limited.

“Both of these published reports are exciting findings as they pave the way for promising new treatment options for NET patients,” adds Dr. Iyer.

Both studies were supported in part by Roswell Park’s core grant from the National Cancer Institute (project no. P30CA016056). The survivin study also applied funds from a grant from the National Center for Research Resources (project no. S10OD019977), and the nintedanib work benefitted from funding through the National Comprehensive Cancer Network’s Oncology Research Program, from support provided by Boehringer Ingelheim Pharmaceuticals Inc.