Experimental Therapy for Midgut Neuroendocrine Tumors Investigated in Clinical Trial

A new drug has been shown to improve progression-free survival in patients with rare midgut neuroendocrine tumors, according to researchers from Northwestern Medicine. The results of the global Phase III clinical trial were published in the New England Journal of Medicine.

Midgut endocrine tumors are a rare form of cancer that affect the small intestine and the colon. A somatostatin analogue is the first-line treatment for these types of tumors, however patients who experience disease progression have few options when it comes to alternative therapies. Patients with metastatic disease currently have an estimated 50 percent five-year survival rate.

The current study involved 229 patients with progressive midgut neuroendocrine tumors, who were randomly assigned to one of two treatment groups. Some patients received a high dose of the somatostatin analogue, octreotide long-acting repeatable (LAR), while others received the experimental drug, 177Lu-Dotatate.

177Lu-Dotatate is a peptide receptor radionuclide therapy (PRRT), capable of delivering a dose of radiation to the site of the tumor cells. The drug has not yet been approved by the FDA.

By the 20^th month of the study, patients given the experimental 177Lu-Dotatate had a 65.2 percent estimated rate of progression-free survival. This was compared to just 10.8 percent for those patients given the standard of care, octreotide LAR.

While patients in the control group showed a treatment response rate of three percent, those patients given 177Lu-Dotatate showed an improved response rate of 18 percent. The study authors concluded that the disease progression and death risk for patients given the experimental therapy was 79 percent lower than those taking the first-line treatment.

“Northwestern Medicine is a major site for the treatment of neuroendocrine tumors,” said study co-author Dr. Al Benson, professor of Medicine in the Division of Hematology/Oncology. “Upon FDA approval, we look forward to being able to routinely offer our patients this therapy as an important advancement in care.”