Researchers at Seattle Children’s are constantly asking questions and investigating new treatments with the goal of improving care for our patients. Two investigators from Seattle Children’s Research Institute recently came together to determine the best therapy for children suffering from infantile hemangiomas.
A breakthrough treatment
Right after Lorene Locke gave birth to her daughter Shakira, she noticed what looked like a rash on the newborn’s face. Three weeks later, doctors found an abnormal clump of vessels, called an infantile hemangioma, growing out of control inside Shakira’s throat and on her neck, face and ear, blocking her airway and leaving her gasping for air.
While most hemangiomas go away on their own and don’t cause problems, children like Shakira need multiple surgeries and procedures to remove the growths. Dr. Jonathan Perkins, an otolaryngologist and an investigator at theat Seattle Children’s Research Institute, has spent years studying hemangiomas in search of a less-invasive approach. When French researchers discovered that a blood pressure medicine called propranolol could shrink away hemangiomas, Perkins found the breakthrough he was waiting for.
“In 2008, I put Shakira on the drug, making her one of the first patients in the region to receive it,” Perkins said. “Within four months the tumors that obstructed her airway were gone.”
Today, Perkins estimates that he performs 75% fewer surgeries to remove hemangiomas because the blood pressure medication is so effective.
He’s now teaming with Dr. Mark Majesky, a principal investigator at Seattle Children’s Research Institute’sand an expert on how blood vessels form, to find out more about how propranolol works and to determine the best ways to use it in patients.
“I don’t necessarily think like someone who studies these deep biological questions,” Perkins says. “Mark knows which questions to ask and the fastest ways to answer them.”
The “a-ha” moment
Propranolol works for about 60% of patients with hemangiomas, so researchers are working to identify which will respond to the drug. Perkins and Majesky started testing propranolol on hemangioma tissue and met every other week to discuss the results and plot their next steps. The “a-ha” moment came last year, when the researchers discovered a new biomarker and found that levels of it were dramatically higher in blood taken from patients with hemangiomas than in healthy patients’ blood.
“It was so clear I just about jumped out of my chair,” Perkins recalls.
The researchers are patenting a blood test that uses the biomarker to track whether propranolol is working in a particular patient. This will help doctors know if a hemangioma is shrinking even if it’s inside a patient and out of sight. The research earned Perkins the Robert Ruben Scientific Achievement Award from The Society for Ear, Nose and Throat Advances in Children.
“None of this research would be possible if I had not been able to collaborate with Dr. Mark Majesky, as well as Dr. Bill Dobyns at the University of Washington,” Perkins said.
Ultimately, Perkins and Majesky hope to develop a similar test that can predict whether propranolol will be effective, helping doctors quickly decide when patients need surgery and saving patients from the trial and error of taking a drug that may not be a good match for them.
“It’s about making kids’ lives better,” Majesky says.