Research

The human brain has over 400 miles of total vasculature, yet little is known about the tiny capillaries that make up much of this intricate labyrinth. Understanding how this vast network regulates blood flow in the brain could hold the key to new treatments for neonatal and childhood neurologic conditions, such as stroke and hypoxia, and issues of aging like dementia and Alzheimer’s disease.

While the human brain has over 400 miles of total vasculature, little is known about the tiny capillaries that make up much of this intricate labyrinth of blood vessels critical for delivering oxygenated blood and nutrients to billions of brain cells.

According to Dr. Andy Shih, a principal investigator in the Center for Developmental Biology and Regenerative Medicine at Seattle Children’s Research Institute, understanding how this vast network regulates blood flow in the brain could hold the key to new treatments for neonatal and childhood neurologic conditions, such as stroke and hypoxia, and issues of aging like dementia and Alzheimer’s disease.

“Insufficient blood flow contributes to many of the common neurologic problems seen in children and adults,” he said. “Yet, because we can’t see the capillaries, which measure about 1/10th the thickness of hair, with in vivo clinical imaging techniques, determining how blood travels through this densely packed bed of vessels has remained elusive.”

Wanting to get a closer look, Shih and fellow scientists, Dr. Andree-Anne Berthiaume and Dr. David Hartmann, applied special techniques called two-photon imaging and optogenetics to isolate and study brain capillaries in animal models. Their findings published today in Nature Neuroscience describe the dynamics that govern capillary blood flow in the brain and have broad implications for future avenues of brain research.
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The Therapeutics Cell Manufacturing facility at Building Cure translates laboratory discoveries into real-world treatments.

The U.S. Food and Drug Administration on Feb. 5 approved Bristol Myers Squibb’s Breyanzi, a chimeric antigen receptor (CAR) T-cell therapy used to treat adults with certain types of large B-cell lymphoma who have not responded to or who have relapsed after standard treatments.

The approval was supported by research at Seattle Children’s, including the chimeric antigen receptor (CAR) T-cell product, patient product manufacturing for Juno Therapeutics’ TRANSCEND trial, and data from the Pediatric Leukemia Adoptive Therapy (PLAT-02) clinical trial. In the PLAT-02 clinical trial, 93% of patients with relapsed or refractory acute lymphoblastic leukemia achieved initial remission, and about 50% were still in remission one year after therapy. Read full post »

Ezra Anpo (right), here with his sister Aria, participated in a research study investigating a liquid biopsy approach to providing a genetic diagnosis in children with lymphatic malformations.

Doctors at Seattle Children’s are investigating whether a simple liquid biopsy containing a small amount of fluid from a patient may someday provide an easier route to a genetic diagnosis in children with vascular or lymphatic malformations.

The work is a collaborative effort led by Dr. James Bennett, a clinical geneticist and co-director of the molecular diagnostic laboratory at Seattle Children’s and Dr. Jonathan Perkins, an otolaryngologist and director of the Seattle Children’s Vascular Anomalies Program. Liquid biopsy offers an alternative to the more invasive surgical biopsies required – when a genetic, or molecular diagnosis, is needed to help guide a patient’s treatment.

“We can now provide a specific genetic diagnosis for a lot of vascular malformations,” Bennett said. “That’s important for families for a variety of reasons with one being it’s just extremely healing and powerful to know the reason why your child has these differences.” Read full post »

Pictured from left to right: Yu Chen, Malika Hale and Christopher Thouvenel of the Rawlings lab at Seattle Children’s Research Institute.

For close to a decade the labs of Dr. David Rawlings at Seattle Children’s Research Institute and Dr. Marion Pepper at the University of Washington have collaborated on a project studying the immune response in malaria infections.

As the COVID-19 pandemic gripped the U.S., they turned their expertise and the techniques pioneered for malaria to a new line of inquiry: Did mild infection from the new coronavirus stimulate the immune system to generate antibodies that would offer future protection from the virus? And if so, could they engineer those neutralizing antibodies in the lab to develop potent new therapeutic options?

Rawlings, the director of the Center for Immunity and Immunotherapies at Seattle Children’s and a professor of pediatrics at UW, discusses their encouraging findings now published in Cell. Learn why he says their research is good news for efforts to control COVID-19 and what’s next for his lab. Read full post »

This year has been filled with unprecedented challenges – physically, mentally, financially – and families are looking forward to putting 2020 behind them. As we collectively usher in a new year, it’s an opportune time to think about small changes we can make to better children’s health in 2021.

Dr. Pooja Tandon, a researcher in the Center for Child Health, Behavior and Development, says this year has caused all kinds of disruptions to children’s lives, unlike anything we’ve seen before. Routines have been shattered, physical activity has decreased, sleep has been affected and the hardships of the year like uncertainty and isolation have impacted children’s mental health.

“Many things are hard right now,” Tandon said. “But for the things we have control over, we can make little changes that can promote health.”

Below, three experts break down three key areas to help support better health in 2021 – physical activity, sleep and nutrition. Read full post »

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 »

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.

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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 »

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 »

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 »