LEXINGTON, Kentucky (August 26, 2021) — UK HealthCare recently launched a new pediatric neuroendocrine tumor clinical and research program to improve the treatment of children diagnosed with or at high risk of developing rare neuroendocrine tumors (NETs). This program is a joint effort of the University of Kentucky’s Markey Cancer Center and the Kentucky Children’s Hospital and is one of the few centers in the world that specializes in this field.
Medical oncologist Aman Chauhan, MD, pediatric hematologist/oncologist Caryn Sorge, MD, and pediatric surgical oncologist David Worhunsky, MD, lead the program, which will use resources from both Markey and KCH to treat children 18 years and younger. who have been diagnosed with a neuroendocrine tumour, or who have been diagnosed with a condition that predisposes them to developing these tumours.
NETs develop from neuroendocrine cells found in the digestive tract, lungs, pancreas, and other sites. They may present unique diagnostic challenges, as neuroendocrine tumors can be easily misdiagnosed for another type of tumor at the same site of origin – for example, pancreatic neuroendocrine tumors may initially be mistaken for pancreatic adenocarcinoma.
Diagnosis of neuroendocrine tumors in children is rare, and treatment options for pediatric patients are limited—in fact, there are currently no Food and Drug Administration (FDA)-approved medications to treat metastatic pediatric NETs. Young patients often receive the same treatments as adults, but not enough research has been done on the pediatric population yet to develop an FDA-approved standard of care. To address this issue, Chauhan says the UK will soon open a new clinical trial for pediatric NET patients using peptide receptor radionuclide therapy (PRRT), commonly known as Lutathera.
“We use treatments based on our understanding of how we treat neuroendocrine tumors in adults, but there are no FDA-approved therapies for children with this disease,” Chauhan said. “It’s very important to conduct clinical trials and get some treatments approved for this condition. This is just our modest effort to kick-start that process of expanding new therapy options in this age group.”
Children who have a strong genetic link to someone diagnosed with NETs — such as a parent or sibling — are at a much higher risk of developing the disease because of an inherited condition known as multiple endocrine neoplasia (MEN) syndrome. In addition to treating diagnosed pediatric NETs, the UK program is working to diagnose children at risk and closely monitor for signs that a NET is developing. Children with MEN syndrome will have regular physical exams, blood tests, and optional imaging to ensure that any tumors are spotted quickly before they can metastasize.
“These tumors can be asymptomatic for a long time in the early stages,” Chauhan said. “And by the time they present, they’ve metastasized — and when that happens, again, there’s no FDA-approved treatment. For these patients, it’s paramount to catch it early.”
Although NETs are often considered rare, they are now diagnosed six times more often than they were about four decades ago. In Kentucky, that problem is even more pronounced: A 2018 study led by Chauhan showed a 10-fold increase in net incidence in the Commonwealth compared to four decades ago. Improved diagnostics and tumor classifications are likely responsible for some of this increase, although it is not yet clear whether there are specific environmental factors contributing to the increased cases.
What is clear, Chauhan says, is that there is a real need for this type of specialized care in the pediatric population.
“There is an absolute unmet need,” Chauhan said. “What I am hearing from parents is that there is a significant lack of multidisciplinary programs targeting neuroendocrine cancer in children. At the UK’s Markey Cancer Center we have a multidisciplinary neuroendocrine tumor program and we combine that capability with the expertise from the pediatric oncology program at Kentucky Children’s Hospital, so we came together to form this program to provide specialized care to this unique subset of patients who would otherwise not receive this care elsewhere.”