Research

Taylor Tran (left) and her mother Mai Nguyen. Taylor underwent cancer treatment when she was 2 years old, causing her to go into early menopause when she was just 16.

“You pay the price for having cancer over and over again.”

Mai Nguyen’s words are loaded with sorrow as she speaks about her 17-year-old daughter, Taylor Tran, who is dealing with fertility concerns more than a decade after she survived late-stage cancer.

It’s easy to understand the exasperation Nguyen feels: Her daughter was diagnosed with stage 3 single-cell sarcoma of the kidney when she was 2 years old and was treated with intense chemotherapy and radiation. Now, the treatments that saved her life have put her into early menopause.

“It’s been traumatic,” Nguyen said. “We’ve tried so hard to allow Taylor to have a normal childhood and this feels like one more thing cancer has taken from her.”

Stories like Taylor’s inspired Seattle Children’s urologist Dr. Margarett Shnorhavorian to tackle a challenging area of research that was largely uncharted more than a decade ago. Since then, she’s helped change perspectives and protocols for fertility preservation in childhood cancer survivors. Read full post »

Ellie Osterloh, 17, participated in the clinical trial for Trikafta, a breakthrough cystic fibrosis therapy approved by the U.S. Food and Drug Administration in October 2019. Photo courtesy of Audrey Redfern.

When 17-year-old Ellie Osterloh spins high above the ground from a lyra, a circular hoop used in aerial acrobatics like Cirque du Soleil, she feels empowered.

“On the lyra, it’s an incredible feeling to be so high in the air with no harnessing,” Ellie said. “It’s a lot of adrenaline and I feel like I can do anything.”

Now, thanks to Trikafta, a new drug approved in October 2019 by the U.S. Food and Drug Administration (FDA), Ellie, who participated in the clinical trial for the drug, shares a similar zeal for her future.

“Even though it’s hard sometimes to be so optimistic, I’ve always thought it might be possible to go to college and have kids and a family of my own,” she said. “It’s crazy how my outlook has changed. I’m still processing all the possibilities.”

That’s because Ellie has lived her entire life with cystic fibrosis, a rare, progressive, life-threatening disease. She had her first appointment with Dr. Bonnie Ramsey, the director of Seattle Children’s Center for Clinical and Translation Research, still in her mother’s womb. Hours after birth she was transferred to Seattle Children’s where she began intensive therapy that she’s continued over the last 16 years.

“This is a really big step forward for Ellie and other people living with cystic fibrosis,” said Ramsey, a pioneer in cystic fibrosis treatment. “Ellie is a highly talented young lady with a bright future ahead of her.” Read full post »

Madeline Boese was one of the patients in Seattle Children’s PLAT-03 trial.

Chimeric antigen receptor (CAR) T-cell immunotherapy, which reprograms a child’s white blood cells so they can seek out and destroy cancer cells, is making a difference in children’s lives. Currently, Seattle Children’s has multiple trials open that could benefit children and young adults with relapsed or refractory cancers. In October, Seattle Children’s opened a new pediatric research facility, Building Cure, to accelerate discoveries such as immunotherapy.

Seattle Children’s researchers are continuing to realize the promise of CAR T-cell immunotherapy and improve outcomes in difficult to treat pediatric cancers. They are applying knowledge gained from ongoing clinical trials to study effects on the youngest patients, develop new interventions to prevent side effects and boost T-cell persistence, and to better understand resistance to therapy.

Research recently published in major scientific journals and presented at the 2019 American Society of Hematology (ASH) Annual Meeting contributes new insight guiding the evolution of the experimental therapy. Here, On the Pulse summarizes their findings. Read full post »

Neurons found in the brain stem gives new clue to nicotine, sudden infant death syndrome link. Source: Getty Images.

Researchers at Seattle Children’s Research Institute have discovered that populations of neurons in the brain stem have a previously unrecognized susceptibility to disruption by nicotine during early brain development.

Published in the Journal of Comparative Neurology, their findings offer a clue to how nicotine exposure in utero could have a lasting effect on the brain’s wiring and give rise to negative outcomes like sudden infant death syndrome (SIDS). Read full post »

A federal contract awarded to Seattle Children’s will establish a new center to study tuberculosis. Source: Getty Images.

Seattle Children’s Research Institute is one of three recipients of $30 million in first-year-funding provided by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), to establish centers for immunology research to accelerate progress in tuberculosis (TB) vaccine development.

The awards provide up to seven years of support for three Immune Mechanisms of Protection Against Mycobacterium tuberculosis (IMPAc-TB) Centers to uncover the immune responses needed to protect against TB infection. In addition to Seattle Children’s, other IMPAc-TB centers will be led by the Infectious Disease Research Institute, also in Seattle, and the Harvard T.H. Chan School of Public Health.

On the Pulse learned more about the significant research that will be funded by this award which has the potential to provide up to $83 million to the Cascade IMPAc-TB Center, led by Dr. Kevin Urdahl, a TB researcher in Seattle Children’s Center for Global Infectious Disease Research. Read full post »

The parasite that causes malaria waits in the salivary glands of the mosquito until it has the opportunity to move to a human host. New research exploring this stage of the parasite offers findings that could help identify new strategies to protect against malaria. Photo: Getty Images.

Within seconds after an infected mosquito bites, the malaria parasite navigates the host skin and blood vessels to invade the liver, where it will stay embedded until thousands of infected cells burst into the bloodstream, launching malaria’s deadly blood-stage infection.

Now, for the first time, a team from Seattle Children’s Research Institute describes how malaria Plasmodium parasites prepare for this journey as they lie in waiting in the mosquito’s salivary glands. Researchers say this knowledge may help identify new strategies to block transmission of the parasite – a critically important step needed for the eradication of malaria, a disease that continues to sicken over 300 million people and kill an estimated 435,000 people worldwide every year. Read full post »

Isabelle Zoerb, 13, has a rare genetic disorder called primary ciliary dyskinesia (PCD).

Like a typical 13-year-old, Isabelle Zoerb plays volleyball and tap dances. She also regularly uses an inhaler, takes antibiotics to minimize lung inflammation and wears a therapy vest that vibrates to help clear her lungs. A device in her chest provides intravenous medication when needed.

This is because Isabelle has primary ciliary dyskinesia (PCD), a rare genetic condition. Cilia are tiny hair-like cells in airways that beat in a coordinated way that clear out germs, mucous and particulates like dust from the respiratory tract. In people with PCD, cilia do not beat properly, which prevents bacteria from clearing the lungs, sinuses, nose and ears.

When Caroline Zoerb adopted Isabelle from China as a toddler, Isabelle’s organs were reversed like a mirror image. She had been born with a hole in her heart and was constantly sick. Seeking answers, the family met Dr. Margaret Rosenfeld, an attending physician and researcher at Seattle Children’s, who thought she might have PCD based on her symptoms.

Seattle Children’s is the only PCD referral center in the Pacific Northwest, with patients coming from Wyoming, Idaho, Oregon, Alaska and Montana. Providers see patients for regular follow-ups to make sure their needs are being met and they are responding to their therapies.

“We always seem to make our way to Seattle Children’s because they have the expertise to help someone with such a rare disease,” Zoerb said. Read full post »

Seattle Children’s will embark on a groundbreaking clinical trial that will potentially transform treatment methods for children with relapsed acute pediatric leukemia.

In collaboration with The Leukemia & Lymphoma Society (LLS), Dr. Todd Cooper, an oncologist and director of the Seattle Children’s High-Risk Leukemia Program, is part of a team leading the effort to launch a global precision medicine master clinical trial called the LLS Pediatric Acute Leukemia (PedAL) Initiative. The goal of the trial, which is part of The LLS Children’s Initiative: Cures and Care for Children, is to test multiple targeted therapies simultaneously at up to 200 clinical sites, including Seattle Children’s, worldwide.

Cooper, the Clinical Trial Lead, will oversee the master screening trial where children with newly diagnosed and relapsed acute leukemia can choose to have their clinical and biologic information included in an international database. This database will serve many purposes, including helping to determine an individual child’s eligibility for a number of targeted clinical trials. The data will also be used to uncover new targets for therapy and as a rich source for groundbreaking discoveries.

On the Pulse sat down with Cooper to discuss the specifics of the trial and how it will possibly revolutionize the types of cancer treatments available for children.

Read full post »

Daniel Sokoloff and his mom, Lara Sokoloff, photographed here when Daniel was younger, were one of 100 families who contributed to a study aimed at understanding common birth defects of the brain.

In the largest genetic study of the most common birth defects of the brain diagnosed during pregnancy, researchers from Seattle Children’s Research Institute say their findings evolve our understanding of brain development. The findings will also change the information given to expecting parents when cerebellar malformations, such as Dandy-Walker malformation and cerebellar hypoplasia, are detected prenatally.

With funding support from the Dandy-Walker Alliance and the Philly Baer Foundation, the results gathered from 100 families provide the most accurate information to date about the genetic and non-genetic causes of birth defects involving the cerebellum. They will also help doctors counsel parents about their child’s prognosis after they’re born and their risk of having another child with the abnormality.

“For those who are pregnant, you certainly don’t want to have a problem with the baby, but if doctors detect a brain abnormality, you want accurate information about what that means,” said Dr. William Dobyns, the senior author on the paper published in the American Journal of Human Genetics and an investigator in the Center for Integrative Brain Research. “It’s the same after birth. If you have a child at risk for developmental challenges, it helps to know the cause and what’s going to happen. This study significantly advances our ability to answer those questions.” Read full post »

Credit: E. Dubois Photography

Tatum Fettig remembers when her family’s lives changed forever. In 2016, her daughter Teagan began vomiting and struggling with balance. At Seattle Children’s, Teagan, then 2, was diagnosed with a pediatric brain tumor, medulloblastoma. Through the grueling process of chemotherapy treatment and radiation, Fettig and her husband were by Teagan’s side, trying to cope with the uncertainty of whether they would see their youngest child grow up. They mourned the loss of their former life.

“Having a child with cancer is traumatizing,” Fettig said. “It doesn’t mean you’re broken, but it affects the whole family. People tell parents to take care of themselves, but when your kid is sick, you can’t think of anything else.”

Fettig’s experience is not uncommon. Research shows that parents of children with cancer experience psychological stress during the child’s treatment. After treatment is complete, parents report higher anxiety, depression and posttraumatic stress than the average population. However, formal mental, emotional and social support for parents is not typical after a child’s cancer diagnosis.

In a study published in JAMA Network Open, Seattle Children’s researchers addressed this need, adapting an intervention previously used for teens and young adults with cancer. They found that one-on-one sessions teaching skills through a tool called Promoting Resilience in Stress Management for Parents (PRISM-P) improved resilience and benefit finding, or personal growth, among parents of children with cancer. Read full post »