Data Shows Radiotherapy Drug is Effective and Safe for Patients With Pheochromocytoma and Paraganglioma

According to a story from, the results of a recent research trial demonstrate that the radiation therapy drug AZEDRA (ultratrace iobenguane I131) is an effective and safe treatment option for patients that have rare neuroendocrine cancer tumors such as pheochromocytoma and paraganglioma. AZEDRA is designed for patients that have run out of other options and are facing recurring, malignant, or unresectable cases of these cancers.
Pheochromocytoma is a neuroendocrine tumor that originates in chromaffin cells, which are found in the medulla of the adrenal gland. It can be benign or cancerous. The cause is typically genetic, and is found to be inherited in around a quarter of cases. Symptoms include high blood pressure and heart rate, flank pain, headaches, heavy sweating, palpitations, anxiety, weight loss, abnormal skin sensations, and pale skin. Paraganglioma is closely related to pheochromocytoma, but it can originate at different areas, such as the thorax, neck, abdomen, or head. It may not cause any symptoms, but when it does, they are similar to pheochromocytoma. About half of patients with paraganglioma will experience metastasis, which reduces the five year survival rate to 40-45 percent. Surgery is the most common treatment, although radiation therapy is also an option.

The results of this trial should be an encouragement for patients. There is an urgent need for more treatment options, as patients that do not qualify for surgery often have few backup plans to choose from. AZEDRA has already demonstrated enough potential to receive Orphan Drug, Breakthrough Therapy, and Fast Track designations from the U.S. Food and Drug Administration.

In the study, a total of 68 patients received at least a single dose of the drug. 92 percent of patients at least had a partial response to the treatment. 25 percent achieved the clinical benefit endpoint of the study, and 32 percent obtained the endpoint when given a second dose. The endpoint was at least a 50 percent decrease in the medications patients were using to manage disease symptoms like high blood pressure for at least six months. Cardiovascular issues are a major source of problems for patients.

The safety profile of AZEDRA was also considered good, with nothing occurring outside the normal range expected for radiation therapy. Hopefully, the FDA will approve this promising treatment soon.

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