New Drug Gives Young Thyroid Cancer Patient a Second Chance at Life

blue capsules coming out of bottle

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Key Takeaways

  • The FDA approved pralsetinib (Gavreto), a selective RET-inhibitor drug, for RET fusion-positive non-small cell lung cancers (NSCLC) on September 4, 2020, and RET-altered thyroid cancers on December 1, 2020.
  • This medication, and others like it, provide a more targeted treatment option for patients with these diagnoses.
  • Willing clinical trial participants can shape the future of cancer care for others.

Morgan Romero was 17 when she was diagnosed with papillary thyroid cancer—the most common thyroid cancer type, frequently diagnosed in women under 30. While thyroid cancer is not rare and highly treatable, Romero's particular case quickly became complicated because of how far the cancer had spread while remaining undetected. And if it weren't for a car accident, it would have gone undetected even longer.

“I was in the car with my mom, and all of a sudden, an 18-wheeler was in front of us,” Romero tells Verywell, detailing a 2012 accident.

Romero did not appear to have any immediately life-threatening injuries, but because she has spina bifida, emergency room doctors were concerned about a potential whiplash injury.

“They did lots of tests and said, ‘Your neck is fine, but we found spots in your lungs,'" she says.

A Complicated Diagnosis

Romero saw a pulmonologist in her hometown of Dallas, Texas, who determined that the concerning areas were from papillary thyroid cancer that had spread to her lungs. 

Romero underwent a thyroidectomy, surgical removal of the thyroid, and radioactive iodine treatment to kill any remaining thyroid tissue in her body, yet she was not improving. Her physicians recommended hospice care, but Romero was not ready to accept that plan.

“No, I’m too young for this,” Romero recalls telling her doctors.

Vivek Subbiah, MD, who treated Romero, tells Verywell that it is common for younger patients to go undiagnosed with this type of cancer until it reaches its later stages.

“In younger patients, we see cancers present later because they feel well,” he says.

With her mom’s support, Romero decided to seek out MD Anderson Cancer Center's expertise in Houston, Texas, in 2013. Their expert multi-disciplinary endocrine team treated her with two sequential therapies, lenvatinib and then sorafenib. They worked for some time, but eventually, Romero's cancer progressed, and she became quite ill.

Romero was hospitalized for pneumonia and required mechanical ventilation three times in January of 2018. Her weight dropped to 70 pounds.

Because her tumor had something called a RET mutation, Romero began to receive care from the Clinical Center for Targeted Therapy, the MD Anderson clinical trials department.

What Are RET Fusion-Positive Cancers?

In some thyroid cancers, specific alterations in the rearranged during transfection (RET) gene cause the cells to make an abnormal form of the RET kinase protein. This altered protein fuels the growth of cancer cells. In some cases, like Romero's, these cells can spread to the lungs, but not all RET fusion-positive lung cancers come from the thyroid. Some RET-fusion positive cancers begin in the lung.

RET fusion-positive tumors occur in about 2% of non-small cell lung cancers (NSCLCs) and approximately 10 to 20% of papillary thyroid cancers, Subbiah says.

A New Clinical Trial Provides Hope

“When Morgan [entered our clinical trial] in 2018, she was sick, and she was declining rapidly. She was admitted to the hospital multiple times for oxygen, and she was having trouble breathing,” Subbiah says.

Subbiah and the MD Anderson treatment team felt that Romero was an appropriate candidate for an experimental medication called pralsetinib (Gavreto), a selective RET-inhibitor, which showed promising results with certain cancers.

RET-inhibitors attack the RET protein in thyroid cancers and lung cancers with the RET gene mutation.

In addition to pralsetinib, the Food and Drug Administration (FDA) approved selpercatinib (Retevmo), another RET inhibitor, on May 8, 2020. These drugs are taken by mouth as capsules, typically once or twice a day.

After years without improvement, Romero was eager to try the medication.

“When I got out of the hospital and was able to swallow, I said ‘Let’s go on medicine!’” she remembers.

After being on the ventilator, Romero had to learn how to swallow again and was fearful of choking on pills. Still, she says she remained optimistic about her chances for improvement.

“Morgan was one of the first papillary thyroid cancer patients to enroll in this trial,” Subbiah says. “She and her family were highly motivated.”

Common Side Effects of RET-Inhibitors

  • Dry mouth
  • Diarrhea or constipation
  • High blood pressure
  • Feeling tired
  • Swelling in the hands or feet
  • Skin rash
  • High blood sugar levels
  • Muscle and joint pain
  • Low white blood cell, red blood cell, or blood platelet counts
  • Changes in other blood tests

A Second Chance at Life

Romero and her treatment team noticed an immediate, positive response to pralsetinib.

“I’m doing pretty well for the most part, and I haven’t had any major issues,” Romero says. “I am hungry again. My hair is growing back.”

“Morgan had more energy, her ability to perform activities of daily living improved, and within two months, she stopped requiring oxygen,” Subbiah says. “The tumor has shrunk, and clinically she looks well. She is a different person.”

Romero continues to take pralsetinib every day. She has health complications due to spina bifida but has not required any further hospital stays for breathing difficulties. Her lung growths, while still present, remain stable.

Romero recently fulfilled a long-time dream of hers. She graduated from college with a bachelor’s degree in psychology.

The Importance of Clinical Trials

Subbiah says that patients with rare cancers like Romero can make a big impact by participating in clinical trials. Traditional treatments can cause major side effects in many patients, and newer therapies provide better options. But the only way for a newer therapy to become widely available is by going through a clinical trial.

"Clinical trials are an essential and basic element of new drug treatment discovery and development," Subbiah says. "Clinical trials are a prerequisite by FDA before a new product can be 'FDA-approved' and brought to the market. FDA approval opens doors for many patients who can not come to large cancer centers. Morgan made it possible for real patients in the real world to get access to this drug,”

Subbiah also advocates for more frequent molecular screening tests, which help identify rare cancer types treated with newer, advanced therapies.

“Molecular testing is increasingly commonplace and available, but it’s still not standard,” Subbiah says. “Patients should advocate for molecular testing, especially if theirs is a rare diagnosis, cancer has spread, or there are not many treatment options. Labs hold on to samples, so it is possible to ask for molecular testing. Cancer can also change as it spreads, so it may be worthwhile to ask for molecular testing on a new biopsy sample from metastatic sites.”

5 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read our editorial process to learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
  1. U.S. Food & Drug Administration. FDA approves pralsetinib for lung cancer with RET gene fusions. September 8, 2020.

  2. Blueprint Medicines. Blueprint Medicines Announces FDA Approval of GAVRETO™ (pralsetinib) for the Treatment of Patients with Advanced or Metastatic RET-Mutant and RET Fusion-Positive Thyroid Cancer. December 1, 2020.

  3. American Cancer Society. Targeted Drug Therapy for Thyroid Cancer. December 18, 2020.

  4. American Cancer Society. Targeted Drug Therapy for Non-Small Cell Lung Cancer. December 22, 2020.

  5. U.S. Food & Drug Administration. FDA Approves First Therapy for Patients with Lung and Thyroid Cancers with a Certain Genetic Mutation or Fusion. May 8, 2020.

By Cyra-Lea Drummond, BSN, RN
 Cyra-Lea, BSN, RN, is a writer and nurse specializing in heart health and cardiac care.