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Pancreatic neuroendocrine tumor

Conquest - Summer 2013

The treatment: A drug for the rarest of cancers

Related story: A magnificent seven


By Laura Sussman

In 2011, James Yao, M.D., associate professor in the Department of Gastrointestinal Medical Oncology, and his colleagues published pivotal findings from a Phase III randomized trial, led by MD Anderson, showing that everolimus dramatically improved progression-free survival for patients with advanced pNET (pancreatic neuroendocrine tumors). Before that, there were no large-scale, well-conducted randomized studies to guide treatment decisions and only one approved therapy for the treatment of this disease.

Disease: pNET is a more rare and less aggressive form of pancreatic cancer than the more common adenocarcinoma and involves cells that secrete a variety of hormones. Tumors can be functional and produce high amounts of hormones, or non-functional and not produce any. This disease accounts for approximately 1% of pancreatic cancers, and 3 in 1 million of all cancers diagnosed.

Oral medication: Everolimus, an immunosuppressant agent used to prevent rejection of organ transplants, is taken once daily.

Clinical trial: Showed a 65% reduction in risk of disease progression; an increase in median progression-free survival of 2.4-fold in those taking everolimus, compared to 9% in those receiving a placebo.

Results: The drug was approved for the treatment of pNET in May 2011.

“Our research had immediate clinical implications,” Yao says. “Ultimately, everolimus’ approval led to a definitive change in the standard of care for those patients with pancreatic neuroendocrine tumors.”

The patient: A gift to live cancer free

Miriam Jauregui simply does not have time for cancer. For the 36-year-old mother of three, there is always a softball game to attend, homework to help with and a house to run.

Miriam Jauregui
Photo: F. Carter Smith

Long a sufferer of acid reflux, Jauregui had learned to live with the condition — never thinking it was a sign of a cancer. However, when diagnosed with an umbilical hernia, a pre-surgery ultrasound revealed spots on both her pancreas and liver. Ultimately, at just 34 years old, she was stunned when told she had one of the rarest of cancers, pancreatic neuroendocrine tumor (pNET).

“My first worry was my children, to leave them alone so young. I would wake up in the middle of the night, crying, and asking God to make everything OK,” Jauregui says.

Her local surgeon in McAllen, Texas, informed her just how rare the tumor was, especially in someone so young. He immediately suggested she travel to MD Anderson for care.

Jauregui’s MD Anderson medical team soon put her at ease and let her know that while rare, the cancer was slow growing. Also, the treatment regimen had been pioneered at the institution. She first underwent an intricate surgery to remove the pancreas and was then put on the oral medication, everolimus. Two years later and still on everolimus, Jauregui has only a small trace of cancer on her liver.

“I thank God every day for my life and for my family,” she says. “I realize now that I can live relatively free from cancer, and for that I’m so grateful.”

— Laura Sussman

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