Research

All Articles in the Category ‘Research’

Global Clinical Trial Aims to Improve Therapies for Pediatric Acute Leukemia

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.

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Genetic Study Evolves Understanding of Common Birth Defects of the Brain

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 »

Study Helps Parents Build Resilience to Navigate Child’s Cancer

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 »

The ‘Miracle’ that Saved Abram

Marilee Killpack describes the birth of her fourth son, Abram, as “magical.”

After a typical, full-term pregnancy, Abram was born in Provo, Utah, weighing 9 pounds. He seemed to be healthy and strong, with one exception: He had petechiae all over his body — red dots that appear on the skin when tiny blood vessels break.

Providers suspected the marks were bruises from his quick birth, but blood tests revealed his platelets were extremely low and his immune system was not making enough antibodies to fight infections.

Abram was taken to the neonatal intensive care unit at his local hospital for platelet and immunoglobulin infusions. Still, his blood counts continued to drop. The family was transferred to a nearby children’s hospital where providers determined he had mononucleosis, which they suspected was causing his immune deficiency.

“They said he would be fine in a few months and sent us home,” Killpack remembers.

But Abram was not fine. He developed severe, uncontrollable eczema when he was 3 weeks old.

“His body was shredded,” Killpack said. “We tried everything — lotions, oils, anything we could think of — but nothing worked. He was screaming; he was in so much pain.”

When providers saw Abram’s skin, they immediately sent a sample of his blood for genetic testing. They suspected he might have a rare, life-threatening disorder called Wiskott-Aldrich syndrome (WAS). Read full post »

My Daughter Inspires Me to Make a Difference for Children with Rare Diseases

The author with her daughter, Mariana, 7. Mariana has a rare disease called Pfeiffer Syndrome.

Often in life we have a vision of what we want or imagine our lives to be like, but along that journey, life presents obstacles and opportunities for us. It shapes us and makes us who we are.

My own journey started with its own twists and turns. I was born in Medellin, Colombia and moved to Seattle with my mom at the age of 7. Two months later I got sick. I couldn’t stop throwing up and had a hard time waking up. A year after being hospitalized on and off at Seattle Children’s, I was diagnosed with a cavernous malformation and had brain surgery two months later. My chances of surviving the surgery were small. In Colombia I would have died, but Seattle Children’s saved my life.

When I gave birth to my daughter, Mariana, 27 years later, I never imagined that she too would face a serious medical condition. Much of my life was impacted by my medical condition, but it was nothing compared to what our family would experience as we learned Mariana had an incurable rare disease. It was the beginning of a life-changing journey for our family. Read full post »

Seattle Scientists Teach Tibetan Monks Modern Cell Biology

Summer school is in session for researchers at Seattle Children’s Research Institute, and although there are text books and a final exam, very little else about the biology course taught by Dr. Philip Morgan and his fellow scientist and wife, Dr. Margaret Sedensky, is business as usual. That’s because their students are Tibetan monks and their classroom is at a monastic university in southern India. Read full post »

A Stroke of Genius

Dr. Andy Shih is studying how tiny strokes called microinfarcts develop and impact the developing brain.

Strokes come in many shapes and sizes. In children and adults, strokes often present sudden limb or facial numbness, confusion and dizziness.

But some strokes that cause clots to develop in the small blood vessels of the brain don’t exhibit any symptoms at all. Studies have shown that hundreds to thousands of these small, asymptomatic strokes, known as microinfarcts, likely occur over the course of decades in adult brains and may contribute to cognitive decline as we age. Even less is known about the occurrence and consequences of microinfarcts in young, developing brains.

Enter Dr. Andy Shih, principal investigator at Seattle Children’s Research Institute’s Center for Developmental Biology and Regenerative Medicine. Shih hopes to solve the mystery of microinfarcts by using advanced optical imaging — modeling them in the lab and visualizing their effects in real-time. On the Pulse sat down with Shih to learn more about his work and how he’s applying his discoveries from studying dementia in aging brains to understanding how blood vessels and clots first emerge in the brain. Read full post »

Sonny Stays Strong as Cell Therapy for His Rare Condition Advances

Sonny D’Ambrosio, age 7, here with his parents, was diagnosed with an autoimmune condition called IPEX as an infant.

Earlier this year, Nicole D’Ambrosio found herself in front of a room full of scientists that were gathered in part to discuss their progress on a novel cell therapy that has the potential to one day save her son’s life.

She had been asked to present her family’s story as part of a company-wide meeting at Casebia Therapeutics in Boston. As she began, she recounted how her only child, 7-year-old Sonny, has reached the brink of death more times than she can remember because of the rare autoimmune disorder he was diagnosed with as an infant. How the bone marrow transplant he received was the only thing that could save him, but caused endless complications, including skin necrosis and epilepsy.

How the thought of going through another transplant when the initial transplant failed compelled her and her husband to pack up their home and move 3,000 miles west to seek other options. How she lies awake at night praying that Sonny’s body can stay strong enough until a safer treatment comes along. How, despite everything he’s been through, Sonny is still a happy little boy with a wicked sense of humor. Read full post »

Madeline Pursues New Dream After Overcoming Cancer Four Times

Madeline Boese, with her mom Terri, cancer-free after a 12-year battle with acute lymphoblastic leukemia.

When Madeline Boese was 14, she had visions of pointe shoes, tutus and sugar plums dancing in her head. She dreamed of becoming a professional ballerina, and hoped for a future in the spotlight doing what she loved.

Unfortunately, her body had different plans.

One day in ballet class in December 2006, she noticed an odd golf ball-sized lump on her left thigh below her pink tights. Her mom, Terri Boese, said a trip to their doctor in their hometown of Plano, Texas, led to a bone-chilling discovery.

“I was terrified when I heard ‘malignancy detected,’” Boese said. “It was awful and so out of the blue. I felt like I was going to hyperventilate, and it took all I had to hold myself together.”

Madeline was immediately referred to a hospital in Dallas where she was diagnosed with acute lymphoblastic leukemia (ALL). From there, Boese said everything moved rapidly as Madeline began what would be two and a half years of grueling chemotherapy treatment.

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Researchers Ready B Cells for Novel Cell Therapy

Dr. Richard James is leading research to edit human B plasma cells to act as cell factories capable of delivering sustained, high doses of a therapeutic protein.

Scientists at Seattle Children’s Research Institute are paving the way to use gene-edited B cells – a type of white blood cell in the immune system – to treat a wide range of potential diseases that affect children, including hemophilia and other protein deficiency disorders, autoimmune diseases, and infectious diseases. If successful, their research would open the door to offering this experimental cell therapy as the first-of-its-kind in clinical trials at Seattle Children’s in as soon as five years. Read full post »