After years of randomized trials and nearly two decades of European data detailing peptide receptor radionuclide therapy, Advanced Accelerator Applications (AAA), a Novartis company, is pleased to announced that the FDA has approved the use of this therapy for patients with neuroendocrine tumors. The approval comes at a time when incidence of neuroendocrine cancers of the pancreas, lungs, and intestines are on the rise. About 7 in 100,000 people are diagnosed with neuroendocrine tumors, or NETS, each year. In the state of Minnesota, that roughly translates to about 400 patients diagnosed each year.
Marketed under the brand name, LUTATHERA®, the drug works by attaching a radionuclide to a somatostatin analogue, which is absorbed by neuroendocrine tumors that possess the SSTR2 receptors. It is administered by an intravenous infusion and as the tumors absorb the radiolabeled medication, it delivers radiation to cancer cell and destroys it from within. (See illustration above.) Somatostatin analogues are given to patients and often slow, stop or reduce the progression of the disease. When somatostatin analogues no longer hold back progression of the disease, there are very few treatment options available. This new therapy provides patients and their medical teams a new option for treatment.
In 2014, data from the NETTER-1 studies met its primary endpoint showing 79% reduction in risk of disease progression or death compared to 60mg long-acting octreotide therapy. Many patients are not diagnosed early in their disease as symptoms tend to either be vague or misdiagnosed for years before a patient has advanced clinical presentation. This new therapy gives hope to patients who have been ineligible for treatments due to advanced progression. LUTATHERA® has been given orphan drug designation from the FDA and the European Medicines Agency (EMA). LUTATHERA® has been administered to more than 2,000 patients on a compassionate use and named patient basis for the treatment of NETs and other tumors over-expressing somatostatin receptors in 10 European countries and in the US under an Expanded Access Program (EAP).
As with all treatments, there can be side effects, the most common being lymphopenia (low levels of lymphocytes), increased liver damage with high levels of gamma-glutamyl transferase, elevated aspartate aminotransferase, and alanine aminotransferase, as well as vomiting, nausea, hyperglycemia (high blood sugar) and hypokalemia (high blood serum potassium).
Centers most versed in the U.S. with PRRT therapy is the University of Iowa and Excel Diagnostics in Houston. PRRT was also given as therapy during NETTER-1 trials by Mayo Clinic Rochester, who is ramped up to offer for this new treatment option. Northwoods NETS is excited to see this treatment finally recognized for use in the United States as it provides new treatment and hope for patients who have otherwise exhausted other therapies. It has been approved for all foregut, midgut, and hindgut neuroendocrine tumors.