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Summer 2015 : A prescription for hope

A new drug delivers renewed optimism

The FDA-approved lenvatinib is helping thyroid cancer patients who don’t respond to standard treatment

BY Ron Gilmore

Steven Waguespack, M.D. Photo: F. Carter Smith

Rafael Pantoja had all but lost hope.

The thyroid cancer he had battled for 14 years was back and spreading.Then, earlier this year, he began taking lenvatinib, a drug that was tested at MD Anderson and approved by the Food and Drug Administration (FDA) in February. Lenvatinib treats patients such as Pantoja, whose thyroid cancer doesn’t respond to the more traditional radioactive iodine therapy. It uses targeted radiation to destroy any thyroid tissue or cancer cells that remain after the thyroid is surgically removed.“At this point in my treatment, lenvatinib was my only option,” says Pantoja, a father of six and certified public accountant who lives in Caracas, Venezuela. “We went for it.”

Pantoja, 62, is one of nearly 300,000 people diagnosed with thyroid cancer each year in the world.

Rafael Pantoja
Photo: F. Carter Smith

For decades, the standard treatment was to administer repeated and often ineffective doses of radioactive iodine, and sometimes chemotherapy,” says Steven Sherman, M.D., associate vice provost for Clinical Research. “About 10 years ago, with new scientific discoveries, we began recognizing the potential for treating patients with drugs that stop tumors from growing their own blood vessels.”

That’s exactly how lenvatinib works. A global study of the drug, also known as Lenvima, demonstrated a dramatic improvement in progression-free survival, with a 65% response rate.

“These are unprecedented results for thyroid cancer patients with such advanced disease,” says Sherman, who led the study.

Results from the study were published this February in the New England Journal of Medicine, coinciding with the FDA’s approval of the drug. Mouhammed Habra, M.D., associate professor of Endocrine Neoplasia and Hormonal Disorders, led the study’s Phase III component at MD Anderson.

Pantoja, who has been under the care of Steven Waguespack, M.D., professor of Endocrine Neoplasia and Hormonal Disorders, directly benefited as a result of the clinical trials. So far, he hasn’t experienced many of the side effects that sometimes accompany the drug, including diarrhea, fatigue, nausea and weight loss. He is, however, among the 40% of patients whose blood pressure rises after taking Lenvima. He’s now on medication to keep his blood pressure in check.

“Lenvima appears to be working,” says Pantoja, who underwent several surgeries to remove sections of his arm bone after the cancer spread. “I’m already seeing a 75% reduction in thyroglobulin.”

The decline in thyroglobulin — a protein produced by both normal and cancerous thyroid tissue — is usually good news for patients being treated for advanced thyroid cancer.

Pantoja’s grasp of the medical procedures that determine his fate are testament to the long road he’s traveled. His journey began in 2000 when doctors removed a small nodule from his thyroid.

Since then, his thyroid has been removed and he’s undergone radioactive iodine treatments and arm surgeries, including one to implant a prosthetic bone that replaced much of his humerus.

This March, the cancer appeared to be moving into his chest.

“We were really getting worried,” he says. “And then Dr. Waguespack said, ‘You won’t believe this, but there’s this new drug, just approved, that might work for you.’”

Pantoja remains optimistic.

“It’s been a long fight,” he says. “But I think we’ve finally found a reason to be happy.”

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