Research

All Articles in the Category ‘Research’

Immunotherapy, Gene Editing Advances Extend to Type 1 Diabetes

Dr. Jane Buckner of the Benaroya Research Institute at Virginia Mason and Dr. David Rawlings at Seattle Children’s Research Institute are leading research to develop an immunotherapy for type 1 diabetes.

Advances in engineering T cells to treat cancer are paving the way for new immunotherapies targeted at autoimmune diseases, including type 1 diabetes. Now, researchers are also investigating therapies that reprogram T cells to “turn down” an immune response, which may hold promise for curing type 1 diabetes, as well as a number of diseases where overactive T cells attack a person’s healthy cells and organs.

“Instead of stimulating the immune system to seek and destroy cancer cells, treating autoimmune conditions will require programming a patient’s own T cells to tell rogue immune cells to calm down,” said Dr. David Rawlings, director of the Center for Immunity and Immunotherapies at Seattle Children’s Research Institute and chief of the Division of Immunology at Seattle Children’s Hospital.

Harnessing gene-editing techniques pioneered by Seattle Children’s, Rawlings and colleagues have already made headway in equipping T cells with the instructions needed to potentially reverse type 1 diabetes. In a new $2 million research project funded by The Leona M. and Harry B. Helmsley Charitable Trust, researchers will leverage these recent successes using this new form of T-cell immunotherapy into first-in-human clinical trials. Read full post »

In Scientific First, Researchers Engineer B Cells to Treat Disease

Primary human B cells could offer the next promising cell therapy. Credit: Human B Lymphocyte by NIAID (CC by 2.0)

Scientists at Seattle Children’s Research Institute have unlocked the ability to engineer B cells, uncovering a potential new cell therapy that could someday prevent and cure disease.

In a paper published in Molecular Therapy, the research team describes how they genetically reprogrammed primary human B cells to act as cell factories capable of delivering sustained, high doses of therapeutic proteins. The gene editing techniques used reprogrammed B plasma cells to secrete a protein that could treat patients with hemophilia B.

Dr. Richard James, a principal investigator in the Center for Immunity and Immunotherapies at Seattle Children’s Research Institute and an author on the paper, discusses the significance of their discovery.   Read full post »

With a Genetic Answer, Parents Find Comfort in Son’s Rare Epilepsy

Genetic testing helped diagnose Nolan Wood, 3, with KCNQ3 epilepsy.

Even though Nolan Wood hadn’t experienced a seizure in more than two years, his parents still had questions about their son’s future.

“We wondered if there were others out there that have what Nolan has,” said Emily Wood, Nolan’s mom. “If so, what does their life look like?”

The Woods’ search for answers began when Nolan, 3, was diagnosed with infantile spasms and regression of his motor skills when he was 6 months old. Before receiving seizure medications, Nolan had hundreds of daily subtle, reflex-like seizures. Due to the regression of his motor skills, he had stopped rolling over, smiling and crying. A condition known as cortical visual impairment had also rendered him legally blind. Read full post »

Pinpointing Pancreatitis: How Family History Played a Role in Amber’s Painful Illness

It’s holiday time in the Louden household. However, this year is unlike any other. For the first time in 11 years, 17-year-old Amber Louden will be able to join her family at the Thanksgiving table and indulge in some of her favorite dishes pain-free.

“I remember Thanksgiving two years ago; I ate so much food that I ended up in the hospital because of the horrible pain I was in,” said Amber. “Last year, I didn’t even get a chance to sit at the dinner table because I spent the holiday in the hospital where I stayed for 12 days.”

Amber’s decade-long battle with chronic pancreatitis prevented her from partaking in cherished holiday traditions.

It may be surprising that these traditions and the root of Amber’s struggle with pancreatitis share one common factor — and that happens to be family.

Read full post »

Newborn Screening for Rare Disorders Becomes Researcher’s Lifelong Mission

Kaitlyn and Ryan Wyckoff travel from their hometown of Wasilla, Alaska, to Seattle Children’s so Dr. Sihoun Hahn (center) can monitor and treat them for Wilson disease — a rare genetic disorder.

For the first 15 years of his life, Ryan Wyckoff appeared to be a perfectly healthy, active teenager, living with his family in Wasilla, Alaska.

But during New Year’s weekend in 2009, Ryan began to feel seriously ill. He was lethargic and had a high fever that could not be controlled by acetaminophen.

Ryan was so sick he could barely make the trip to his family doctor. The doctor thought Ryan looked jaundiced and referred him to their local hospital, but providers there found nothing wrong so they sent him home.

Ryan’s symptoms worsened. He gained 15 pounds in just a couple days as fluid built up in his abdomen. Ryan’s mom, Lisa Wyckoff, remembered how her tall, slender son looked like he was pregnant.

An MRI revealed Ryan had cirrhosis — advanced scarring in his liver. His condition was life-threatening, so he was flown to Seattle Children’s by Medivac.

“It’s terrifying to have something seriously wrong with your son that no one can figure out,” said Lisa. “We felt so helpless.” Read full post »

Researchers Put Youth Sports Safety and Concussion Awareness Ahead of the Game With Novel Program

Seattle Children’s researchers consulted with the Northwest Junior Football League before moving ahead with a CDC-funded program addressing safety and concussion awareness in youth sports. Photo courtesy of Brian Bodine Photography/NJFL

Seattle Children’s researchers will launch an innovative program in early 2018 aimed at shifting the culture of safety in youth sports and building concussion awareness during competitive play.

The program, called One Team, emphasizes community engagement in conducting brief pre-game safety huddles involving coaches, officials, parents and athletes, with a goal of addressing both sportsmanship and the importance of removing an athlete from play if they potentially have a concussion.

Dr. Sara Chrisman and Dr. Emily Kroshus, both members of the Seattle Pediatric Concussion Research Collaborative and Seattle Children’s Center for Childhood Health, Behavior and Development, designed the program.

“We want to change how children, parents and coaches relate to injuries, and reinforce a line in athlete safety that shouldn’t be crossed, even in a competitive atmosphere,” Chrisman said.

Read full post »

Mast Cells May Hold Key to Preventing Group B Strep Infection

Group B streptococcus. Credit: NIAID. This work is licensed under a Creative Commons Attribution 4.0 International License.

Mast cells. Few know that mast cells are the first responders of the immune system. Even fewer study their role in group B streptococcus, a widespread bacterial infection that can cause preterm birth, stillbirth or dangerous sepsis in infants.

Many women who have given birth will likely remember the group B strep screening in their third trimester.

“Group B strep is normally found in up to 30% of women, but because it doesn’t cause illness, we don’t think about it until there is a risk in pregnancy,” said Dr. Lakshmi Rajagopal, a principal investigator in the Center for Global Infectious Disease Research at Seattle Children’s Research Institute. “In babies, the infection can lead to serious and deadly health consequences such as pneumonia, sepsis and meningitis.”

To ensure the infection is not transmitted to the baby, women testing positive for group B strep receive antibiotics during labor. However, no therapeutic strategies exist to prevent the incidence of systemic group B strep infection that can occur in babies before labor or after birth.

Rajagopal and Dr. Adrian Piliponsky, a principal investigator in the Center for Immunity and Immunotherapies at Seattle Children’s, believe part of the answer to preventing group B strep may lie in mast cells. In a study published in the Journal of Allergy and Clinical Immunology, the researchers reveal new insight into how mast cells defend against bacterial infections. Read full post »

Transforming Care for Children With High-Risk Leukemia

Dr. Todd Cooper leads Seattle Children’s High-Risk Leukemia Program.

Seattle Children’s is getting set to launch a program that will redefine how we care for children with “high-risk” leukemia – or leukemia that doesn’t respond well to standard treatments and/or has relapsed after therapy.

Unfortunately, less than 40% of children with high-risk leukemia will live for more than four years after they’re diagnosed. Our new High-Risk Leukemia Program aims to cure more of these children by uniting their doctors onto one team, and by using state-of-the-art diagnostic tests to match kids with the latest treatments and clinical trials. The program will also partner with researchers to pursue new treatments and cures.

The first-of-its-kind program is expected to attract patients and families from across the country, and it’s being led by Dr. Todd Cooper as part of his lifelong mission to improve care for children with high-risk leukemia. On the Pulse sat down with Cooper to learn about how the new program will transform care and bring new hope to children and families. Read full post »

In Quest to Diagnose Puzzling Stomach Ache, Family Turns to Clinical Trial

Isabella Wallis with her older brother and two older sisters.

As the youngest in her family, Isabella Wallis is always following in the footsteps of her older siblings. While it has its perks in helping her learn the ropes, getting to be the first in her family at anything is few and far between.

That was until the 9-year-old from Olympia, Washington, became the first patient to enroll in a first-of-its-kind clinical trial at Seattle Children’s.

“It’s exciting to be able to help other people and give them more information about why they are sick,” said Isabella, who enrolled in the Precision Diagnostics in Inflammatory Bowel Disease, Cellular Therapy and Transplantation (PREDICT) trial after experiencing persistent inflammatory bowel disease (IBD) symptoms for more than six months.

When she didn’t feel well, Isabella, who had always been healthy and loved playing outside, would retreat to her bedroom, finding some comfort in watching reruns of her favorite show, The Simpsons, while lying down. But her upset stomach never went away.

Unclear what was causing Isabella’s illness or what treatment options may exist, her parents turned to Seattle Children’s at the advice of their pediatrician. Read full post »

Promising Drug Combination Silences the Rage of Graft-Versus-Host Disease

London Bowater fought for her life for nearly 180 days in the hospital when she developed severe GVHD after a cord blood transplant.

To pass the nearly 180 days she was a patient in Seattle Children’s Cancer Unit with graft-versus-host disease (GVHD), London Bowater took orders from her doctors, nurses and other patients and families for friendship bracelets that she would braid from her hospital bed.

While her handicraft would help fill the time between treatments, it did little to help ease the severe GVHD she developed after a cord blood transplant for acute lymphoblastic leukemia.

“Her hair was falling out and her intestines were hemorrhaging blood, yet somehow my little ray of sunshine still managed to stay positive and touch all the amazing doctors and nurses with her kindness,” said Nicholas Bowater of his then 8-year-old daughter. “At one point the hemorrhaging was so bad, I melted.  I went into the empty room next door and lost it. I knew we would need a miracle.” Read full post »