Research

All Articles in the Category ‘Research’

Out of Heartache, Hope Surfaces for Colton’s Metabolic Disorder

Colton Iverson holds a photo of his older sister, Cody. Photo courtesy Copper Ridge Photography.

Before his first breath, Colton Iverson had already received the gift of a lifetime. Just days old, he became the youngest patient to go on a drug recently approved by the U.S. Food and Drug Administration (FDA) for the treatment of a life-threatening genetic condition called very long-chain acyl-CoA dehydrogenase, or VLCAD, deficiency.

For his parents, the hope it inspired did not come without heartache.

“Colton wouldn’t be here today without our first born, his older sister Cody,” said his mom, Lisa Iverson.

A few days after coming home as excited new parents of a healthy baby girl with an Apgar score of 10, Cody went lifeless in Lisa’s arms one morning. She and her husband, Ty Iverson, rushed Cody to their local hospital in northeastern Washington.

“They did everything they could to save her and they couldn’t,” Lisa said. “For about week, we had no idea what happened to Cody.”

On the day of Cody’s service, the Iversons heard from their family medicine doctor, Dr. Geoffry Jones.

“As we were driving home, I remember the exact spot on the road when we got the call from Dr. Jones,” Lisa said. “He said her newborn blood test showed she had a genetic condition called VLCAD. He recommended we get in touch with a specialist at Seattle Children’s to learn more.” Read full post »

Scientist Develops New Way to Test for COVID-19 Antibodies

A newly developed cell-free test can rapidly detect COVID-19 neutralizing antibodies and could aid in vaccine testing and drug discovery efforts.

When Dr. Stephen Smith of Seattle Children’s Research Institute came down with muscle aches, gastrointestinal distress and a sudden loss of smell in late February, he suspected he had COVID-19. The testing criteria had yet to be expanded to include individuals with Smith’s symptoms and so he did what many scientists with his expertise would do: he developed a way to test himself.

The fruits of his curiosity, now published in the The Journal of Infectious Diseases, offer a reliable way to quantify whether an individual has neutralizing antibodies that could prevent the novel coronavirus from infecting cells using a method that is more broadly applicable than those currently available.

Read full post »

Making Sense of Restless Sleep Disorder in Children

Emily Caveness, 9, had always been a very active sleeper. When her lack of restful sleep started disrupting her social and school life, her parents sought the help of sleep medicine experts at Seattle Children’s where they first learned of restless sleep disorder in children.

An international panel of sleep experts is adding a new pediatric sleep disorder they call restless sleep disorder, or RSD, to parents’ and pediatricians’ radars.

Led by Seattle Children’s pediatric sleep specialist, Dr. Lourdes DelRosso, the group shares their consensus on a medical definition of RSD in a new paper published in Sleep Medicine. Known to occur in children 6-18 years old, RSD can lead to attention impairment, mood and behavioral problems and other issues at home and school due to poor sleep quality.

“For many years, those of us in sleep medicine have recognized a pattern of sleep that affected a child’s behavior but didn’t fit the criteria for other known sleep disorders or conditions linked to restless sleep like obstructive apnea or restless legs syndrome,” DelRosso said. “This work provides consensus on a definition and diagnostic criteria for RSD, offering a new tool to help more children suffering from restless sleep.” Read full post »

Six Years Later: Immunotherapy’s Tiniest Trial Participant Is Living With “So Much Joy”

Greta Oberhofer, now 7 years old, was the first patient under age 2 included in Seattle Children’s cancer immunotherapy clinical trials. Her parents once considered hospice care for their 13-month-old daughter. Now, Greta has been in remission for six years.

In the fall of 2013, Maggie and Andy Oberhofer watched their tiny, 8-month old daughter, Greta, fight for her life in the Pediatric Intensive Care Unit (PICU) at Doernbecher Children’s Hospital in Portland, Oregon.

Greta had just been through a bone marrow transplant to treat her highly aggressive leukemia. Shortly after, she took a turn for the worse and spent a perilous month in the PICU.

Greta eventually recovered from the transplant and was able to go home, but the family’s reprieve only lasted three months. In March 2014, Greta’s cancer relapsed. Her chance of survival was now 10% or less.

Her parents couldn’t imagine dragging Greta through another bone marrow transplant, so they began to consider end-of-life care for their 13-month-old daughter.

“It was gut-wrenching, knowing her odds were so low,” Maggie said. “We were preparing ourselves to say goodbye to her.”

That’s when they learned about a new option — a cancer immunotherapy trial at Seattle Children’s. Read full post »

Reaching New Heights: Champion for Pediatric Research Reflects on National Role

Recently appointed to the U.S. Department of Health and Human Services Secretary’s Advisory Committee for Human Research Protections (SACHRP), Dr. Douglas Diekema is a passionate champion for the patients and families who participate in research studies. Here, Diekema is photographed enjoying another passion: hiking and mountaineering.

As a newly appointed member of the U.S. Department of Health and Human Services Secretary’s Advisory Committee for Human Research Protections (SACHRP), Dr. Douglas Diekema has always had a passion for interpreting and applying the regulatory laws for research involving human subjects to support the children and families that participate in research at Seattle Children’s.

Although he just assumed his role on the national committee that guides medical research activity across the U.S. this July, Diekema is no stranger to research oversight: he has served as the chair of Seattle Children’s Institutional Review Board (IRB) for the last two decades. In his time as chair, he’s witnessed Seattle Children’s Research Institute grow from a fledgling initiative into the burgeoning enterprise it is today, overseeing hundreds of research studies across nearly every pediatric specialty.

Here, Diekema reflects on what he’s most looking forward to as a member of SACHRP and why it’s very likely you’ve never heard of an IRB before. Read full post »

With Cancer on the Back Burner, Champion Junior Chef Cooks Up His Dreams

Fuller Goldsmith, 16, has always dreamed of being a top chef.

After winning the Food Network’s Chopped Jr. reality TV cooking competition, Fuller Goldsmith, 16, was well on his way to achieving his dreams of becoming a professional chef. It was a future that was soon in jeopardy when life for the aspiring chef took an uncertain, but all too familiar turn. In late 2018, Fuller learned his cancer had returned for a fourth time.

Having undergone treatment for acute lymphoblastic leukemia (ALL) since age 3, Fuller was out of standard treatment options. Their local oncologist told the Goldsmiths about the cancer immunotherapy clinical trials at Seattle Children’s. He thought the experimental chimeric antigen receptor (CAR) T-cell immunotherapy, which engineers a patient’s own immune cells to target and eliminate cancer cells, might offer the best hope for Fuller. Read full post »

New Discovery Paves Way for Next-Generation Malaria Vaccine

Drs. Debashree Goswami (front) and Nana Minkah are working with their Kappe Lab colleagues to develop a vaccine for malaria. New research published by Goswami and her teammates paves the way for a novel, next generation vaccine against Plasmodium falciparum, the parasite that causes the deadliest form of malaria in humans.

In an unprecedented first, scientists at Seattle Children’s Research Institute have developed a genetically attenuated parasite (GAP) that arrests late in the liver stage of human malaria. Their findings published in JCI Insight pave the way for a novel, next-generation GAP vaccine against Plasmodium falciparum, the parasite that causes the deadliest form of malaria in humans.

According to Dr. Debashree Goswami, a fellow in the Kappe Lab at the research institute’s Center for Global Infectious Disease Research and lead author of the paper, a vaccine candidate based on their findings has the potential to offer protection to those living in regions where the transmission of malaria is widespread throughout the population. Read full post »

Engineered T Cells for Type 1 Diabetes Move Closer to Clinic

Dr. Jane Buckner of the Benaroya Research Institute at Virginia Mason and Dr. David Rawlings of Seattle Children’s Research Institute are leading gene editing research to develop new therapies for autoimmune conditions like type 1 diabetes.

For much of the last decade, Dr. David Rawlings, director of Seattle Children’s Research Institute’s Center for Immunity and Immunotherapies, has dreamed of developing a therapy for children with type 1 diabetes that doesn’t involve insulin injections but uses a person’s own immune cells to target and treat the disease.

Now, new research and a fresh infusion of funding bring this dream closer to reality, and nearer to opening a first-in-human clinical trial of an experimental therapy at Seattle Children’s in collaboration with research partner Benaroya Research Institute at Virginia Mason (BRI).

“What started as a dream is now within reach,” Rawlings said. “My hope is that our research will lead to a new treatment that turns off the destructive immune response leading to development of type 1 diabetes in children.” Read full post »

Are Children Making Antibodies That Will Protect Against Future COVID-19 Infections?

More than 50 research studies to understand, detect, treat and prevent the novel coronavirus in children and families have launched at Seattle Children’s since the virus emerged in late 2019. The following post is part of the “Quest(ion) for Discovery” series highlighting this research in progress and the search for answers that could result in major scientific breakthroughs that save lives and slow the spread of the virus.

When Dr. Janet Englund, a member of the Center for Clinical Translational Research at Seattle Children’s Research Institute, received news in late February that a Seattle Children’s patient had tested positive for the novel coronavirus and represented the first example of community transmission in Washington state, she knew things as we knew them were never going to be the same.

Englund, who has long studied respiratory viruses and their transmission in children and frequently provides her expertise as part of national scientific advisory panels, believed we were facing something very different from any pandemic Seattle – or the world for that matter – had ever seen before.

She and her collaborators quickly proposed supplements to their existing studies primarily focused on seasonal influenza, so they could begin to learn about how the new coronavirus impacts children. One study, which she discusses in greater detail here, asks: Are children making antibodies that will protect against future COVID-19 infections? Read full post »

How Might COVID-19 Reshape this Generation of Children?

More than 50 research studies to understand, detect, treat and prevent the novel coronavirus in children and families have launched at Seattle Children’s since the virus emerged in late 2019. The following post is part of the “Quest(ion) for Discovery” series highlighting this research in progress and the search for answers that could result in major scientific breakthroughs that save lives and slow the spread of the virus.

An expert in screen time for children, Dr. Dimitri A. Christakis, leads a team of researchers out of Seattle Children’s Research Institute’s Center for Child Health, Behavior and Development. In a recently published editorial in JAMA Pediatrics, Christakis argues that it’s critical to focus on overlooked decisions, such as the return to school, in the wake of the COVID-19 surge because of its lasting implications for children.

Here, he shares research his team has proposed to understand some of the effects of COVID-19 on child development and wellness while addressing the question: How might COVID-19 reshape this generation of children? Read full post »