Wearing glittery pink sneakers and hugging a fluffy pink and white leopard print blanket, when 2-year-old Clara Jensen looks at you with those big brown eyes, all you see is an adorable, happy, healthy toddler. But that’s not how life started for this child.
A full-term infant, Clara’s parents and doctors were caught completely off guard when she was born Sept. 21, 2017 at AdventHealth (formerly Shawnee Mission Medical Center). That’s because she had a tumor on the back of her tongue—a tumor the size of a golf ball. In fact, it was so large Clara couldn’t close her mouth, or eat. Immediately, doctors transferred Clara to the Neonatal Intensive Care Unit at Children’s Mercy for a higher level of care.
Initially, no one was sure what the growth was, but doctors placed a gastrostomy, or G-tube for feeding. As an added precaution, they also placed a tracheostomy, or breathing tube, in case the growth became larger and blocked Clara’s airway. Once those were in place, Clara had an MRI, plus a surgical procedure to collect tissue for a biopsy, and to reduce the size of her tongue.
Based on preliminary test results, doctors determined the growth on Clara’s tongue was a malignant tumor. They weren’t sure of the exact tumor type though, so they sent the biopsy for more extensive testing. “It’s not routine at most hospitals, but we’re actually able to offer every patient who comes to Children’s Mercy molecular and genetic testing that may allow us to change their treatment,” said Erin Guest, MD, Director of the Cancer Genomics Program and the Cancer Center Biorepository. That additional step turned out to be critical in Clara’s case.
About three weeks after the biopsy was performed, Aaron and Jennifer learned their baby had a rare form of cancer called an inflammatory myofibroblastic tumor. “It was a bit of a shock when the doctors told us Clara’s diagnosis,” Aaron said. And the traditional treatment was more than a little scary for this Independence family. “The doctors said usually with this type of tumor, they remove it surgically, but because Clara’s tumor was on her tongue, no one wanted to do that,” Jennifer said.
“Clara’s tumor was extremely rare,” Dr. Guest said. “But with the results of the molecular testing, we were able to identify an abnormality in the ALK gene, which is present in about half of these types of tumors,” she explained. Those findings led Clara’s oncology team to search for a drug called an ALK inhibitor. Though they found a medication they thought would work, it wasn’t FDA-approved or commercially available to treat the type of tumor Clara had. “Crizotinib is a molecularly targeted drug that stops cells from growing. The tumor was dependent on that ALK gene rearrangement to grow, and if we could halt that, it would give the tumor an opportunity to shrink,” Dr. Guest said.
Crizotinib is approved to treat adults with advanced stage lung cancer whose tumors are ALK-positive. It’s not a form of chemotherapy, and it has very few side effects. But the drug had never been used to treat the type of tumor Clara had, and because she couldn’t swallow, Clara needed a liquid form of the medication so that she could receive it via her G-tube.
Inspired by this tiny girl with the big brown eyes, the oncology team not only found a liquid form of the drug in development, they applied for a compassionate use trial. “A compassionate use trial is basically something we can apply for when there are no other good treatment options available for a patient,” Dr. Guest explained. Though securing approval for this type of trial can take weeks, the Children’s Mercy oncology team received approval in days. “This was basically a clinical trial just for Clara,” Dr. Guest explained.
Before the Jensens knew it, Clara was receiving the life-saving medicine via her G-tube, and the tumor began to shrink. “Our other children were sick about the same time Clara started receiving the medication, so I couldn’t go see her for a week,” Jennifer said. “When I went back to the hospital, I said, ‘Wow! Look at her tongue!” Clara’s tongue was visibly shrinking, and it continued to shrink until the tumor was completely gone.
In all, Clara spent 80 days in the Children’s Mercy NICU being closely monitored for any side effects to the medication. “Other than some mild nausea, Clara didn’t experience any side effects,” Aaron said. When the Jensens took her home, they continued the medication, giving it as prescribed, twice a day for an entire year.
“Clara has had a complete remission of the tumor,” Dr. Guest said. “She’s not had to have any further surgeries or radiation or chemotherapy. Just that single medicine was enough to get rid of the rest of the tumor.” Plus, Clara no longer needs a tracheostomy or G-tube, and because this medication isn’t a chemotherapy drug, she won’t have to face the late effects of treatment many cancer patients do.
“We had so much anxiety and stress when Clara was sick,” Jennifer said. “There were days we would go to the hospital, watch her struggle and feel helpless.” But those days are long gone. Now 2 years old, Clara sees Dr. Guest every three months for follow-up in the outpatient oncology clinic. Her most recent MRI was all clear. And even though the tumor could have affected her tongue and speech development, amazingly, it hasn’t. “Clara is very articulate for a 2-year-old,” Aaron said. “We’re so grateful for everything that Children’s Mercy has done for us and other cancer patients,” Jennifer added. “We’ll never forget how dedicated they were.”