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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 »

Surgery Frees Lillee from Seizures Medication Couldn’t Stop

Lillee Haynes, 4, surrounded by her three older brothers.

When 4-year-old Lillee Haynes runs through the doors of Seattle Children’s South Clinic for her speech therapy appointment and heads straight for a table covered in crayons, it’s hard to imagine that nearly two years ago she faced hundreds of epileptic seizures each day.

“Her seizures happened so often that I installed a camera above her bed to record any she had at night,” said Aimee Haynes, Lillee’s mom. “One night the camera recorded 200 movements. I was shocked to see how many seizures disrupted her sleep.”

Lillee’s brain didn’t rest until she underwent not one, but two neurosurgeries at Seattle Children’s to remove the diseased area of her left brain, allowing her healthy brain to grow and develop.

“You could say Lillee is most definitely right-brain dominant,” laughed Haynes. “That might explain why she has such a spicy personality.” 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 »

Sounders FC Star Jordan Morris Inspires Kids With Type 1 Diabetes

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Jordan Morris with Kylie (left) and Cameron Hendry (right). Cameron, like Morris, has type 1 diabetes.

Kids with type 1 diabetes are used to getting a lot of questions, from “What does that tube on your pump go to?” to “What happens when you don’t feel good?” The natural curiosity of their peers often serves as a reminder of the condition they must constantly stay on top of.

Recently, the tables were turned when Sounders FC star Jordan Morris joined a roomful of type 1 diabetes patients at Seattle Children’s Bellevue Clinic and Surgery Center for a special Q&A.

Morris, who has risen through the ranks of youth, collegiate and professional soccer, can relate. He also has type 1 diabetes. So, in a rare afternoon off from fielding crosses on the soccer pitch, Morris volunteered to field questions from patients and their families about living and playing with the disease.

“When I was younger, I looked up to other professional athletes with diabetes who were doing what I wanted to do, and always thought ‘if they can do it, why can’t I?’” said Morris. “I love talking to the kids. I hope I can serve as that same inspiration to them.” 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 »

What to Know About New Guidelines for High Blood Pressure in Children

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Dr. Joseph Flynn served as the co-chair of the national committee responsible for developing the American Academy of Pediatrics’ new guidelines on high blood pressure.

Parents may notice a renewed focus on screening for hypertension at their child’s next well-child checkup thanks to new guidelines published by the American Academy of Pediatrics (AAP).

While hypertension, or high blood pressure, is common in adults, many people are unaware that approximately 3.5% of all children and adolescents also have the condition. If left untreated, over time hypertension can damage many organs of the body, including the heart, brain, kidneys and eyes.

“The goal is to ensure every child receives annual screening for high blood pressure starting at age 3,” said Dr. Joseph Flynn, an expert in treating children with hypertension at Seattle Children’s and co-chair of the national committee responsible for developing the new guidelines. “Evidence shows pediatricians aren’t routinely measuring blood pressure in younger children, or may not recognize when a child’s blood pressure is high, causing high blood pressure to go undetected and untreated.” Read full post »

Surgery and Rehab Help Arabelle Lasso Life in Junior Rodeos

On Thursday nights this summer, 7-year-old Arabelle Laddusaw is saddling up alongside other children and teens to compete in the Tomorrow’s Rodeo Champions’ summer rodeo series in her hometown of Billings, Montana. As she sits up straight and grips the reins of her horse in anticipation of the chute opening, her cerebral palsy is the last thing slowing down the thrill of events such as barrel racing, pole bending and the crowd favorite, goat tail tying.

However, the prospect of being able to sit naturally on a horse wasn’t always a future possibility for Arabelle who was diagnosed at birth with cerebral palsy, a neurological condition that affects muscle movement and limits mobility.

As a toddler, the muscles in Arabelle’s legs were so tightly wound they would scissor – one leg twisting over the other – making it difficult for her to stand upright or simply straighten her legs out in front of her when sitting.

“With her, it was challenging because she required constant assistance,” said Arabelle’s mother, Christine Laddusaw. “She wasn’t just a normal child that you could put down and let play.” Read full post »