Research

At Seattle Children’s Research Institute, scientists are genetically-engineering zebrafish to harbor human DNA mutations known to contribute congenital conditions in children.

More than five years ago, when Dr. Lisa Maves, a scientist at Seattle Children’s Research Institute, first started using CRISPR to make genetic alterations in zebrafish, she saw the potential for the minnow-sized fish to help doctors understand how genetic mutations contribute to a child’s condition.

“Essentially, we set out to make a patient’s fish,” Maves said. “The zebrafish has a genome that is remarkably similar to humans. As new gene editing technology was just becoming available, I wondered whether we could use this technology to create a fish that mimicked the complex genetic conditions we see in children.”

Maves hypothesized that genetically engineering the fish in this manner would help uncover how different genes affect development and cause disease. Read full post »

At age 2, Erin Cross was diagnosed with acute lymphoblastic leukemia. She achieved remission through her initial cancer treatments, but relapsed in 2016. Out of treatment options, her family found hope in Seattle Children’s PLAT-02 T-cell immunotherapy clinical trial. Erin, now 8, just celebrated her two-year anniversary of being cancer-free. Photo by Jane Mann

Each morning, 8-year-old Erin Cross springs out of bed excited to go to school. A third grader in Chester, England, she loves science and math, and imagines a future as a researcher making “potions” in a lab. She loves cracking jokes, rugby and playing make-believe games with her friends on the playground. For Erin, who spent most of her life in the hospital and away from others her age, she cherishes each day she is able to just be a kid.

“It’s amazing to see Erin back to living a normal life,” said her mother, Sarah Cross. “We’re so thankful that we’re able to enjoy time as a family doing regular things like taking picnics, playing on the beach or going to the zoo. It’s time that we never take for granted.”

Nearly three years ago, Cross faced the devastating reality that she may never see her daughter grow up. At age 2, Erin was diagnosed with acute lymphoblastic leukemia (ALL). She was able to achieve remission through her initial cancer treatments, but in 2016, her family received the shattering news that she had relapsed and was out of treatment options.

That was, until they found hope in Seattle Children’s Pediatric Leukemia Adoptive Therapy (PLAT-02) chimeric antigen receptor (CAR) T-cell immunotherapy clinical trial for children and young adults with relapsed or refractory ALL who are not likely to survive with current treatments. In July 2016, Erin’s family arrived in Seattle for the trial.

“Seattle Children’s threw us a lifeline,” said Cross. “We knew we had to get her there. We moved mountains to save our daughter’s life.”

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Avery Berg, 13, was diagnosed with a rare brain tumor at the age of 10. She endured six weeks of radiation, five brain surgeries, and six months of high-dose chemotherapy. Avery has been cancer-free for more than a year, but her mom Kristie says that cancer immunotherapy offers hope that other children can become cancer-free without having to endure such harsh therapies.

T-cell immunotherapy continues to take center stage as one of the most promising new cancer therapies of our time. What once sounded like a dream – reprogramming a person’s own immune system to fight cancer – is remarkably becoming a reality. What’s more; doctors and researchers in our own backyard are leading the way in developing this therapy for children and young adults around the world.

From covering the opening of the first T-cell immunotherapy trial when I was an anchor at KING 5 TV, to now seeing this therapy being tested in seven open clinical trials at Seattle Children’s and applied to a variety of cancers, I’ve been amazed to watch the enormous strides researchers have made in the field over a few short years.

The results also speak for themselves – 93% of patients with relapsed or refractory acute lymphoblastic leukemia in Seattle Children’s phase 1 PLAT-02 trial achieved complete initial remission. About 50% were still in remission one year after therapy. Some patients, who were otherwise unlikely to survive with traditional therapies, are still in remission nearly five years after receiving the experimental treatment. This is encouraging news, especially since leukemias are the most common childhood cancers.

And on Oct. 12, I will witness yet another major milestone – Seattle Children’s will bring their groundbreaking therapies to a global stage. Read full post »

Milton Wright III has returned to work at the hospital that saved his life.

Milton Wright III has only worked at Seattle Children’s for a couple months, but the hospital has been his second home for much of his life.

Milton’s childhood unfolded within Seattle Children’s walls — making friends, experiencing loss and facing death more times than he can count.

Today, Milton is back at Seattle Children’s — not as a patient, but as an employee and a symbol of hope.

“I want to do something that’s worthy of my life being saved,” Milton said.

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At Seattle Children’s, many children and young adults with cancer are finding hope in T-cell immunotherapy – an experimental treatment that boosts a patient’s immune system and uses it to fight a disease.

Seattle Children’s researchers are leading clinical trials in which a patient’s T cells are reprogrammed to express a chimeric antigen receptor (CAR) on the surface of the cell. The CAR is like a puzzle piece that’s designed to attach perfectly to a specific antigen or marker on the surface of the cancer cell. When they attach, the CAR T cells attack the cancer cells as if they were fighting an infection.

In just five years, Seattle Children’s cancer immunotherapy program has grown tremendously to include trials that target leukemia, brain and spinal cord tumors and solid tumors. Curious how these clinical trials work? Read on to learn more about the immunotherapy clinical trial process at Seattle Children’s.

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Harper was diagnosed with acute lymphoblastic leukemia when she was just 10 months old.

When asked about the birth of her daughter Harper, Sydney Beare lights up.

“Harper was 8 pounds, 1 ounce, 21.5 inches and the most beautiful baby I’ve ever seen!” she said.

By all accounts, Harper was an exceptionally happy, and seemingly healthy, baby. She began sleeping though the night when she was just a few days old and almost never fussed, even when teething. Beare said her daughter was “totally content.”

But in July 2017, when Harper was 9 months old, she became seriously ill.

Harper first developed an ear infection, a staph infection and had an infected cut on her finger. During the next month she became lethargic and pale.

Beare noticed bruises on her legs, and later on her back and face. Harper began having diarrhea and vomiting. She also slept all the time. Despite all this, Harper’s well-child checkup in August revealed no concerns.

Then, on Aug. 21, Harper woke up with a fever.

“She was just lying there, with dry, cracked lips, screaming,” Beare remembered, choking back tears. “I was worried something was wrong but I pushed that idea aside because I didn’t want to think anything bad could happen to my baby.”

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Forrest Potter with his younger brother Bryson Potter. Bryson was diagnosed with Leigh Syndrome (LS) as a child, a progressive neurodegenerative disorder.

Forrest Potter grew up at Seattle Children’s watching by the bedside as his little brother faced a debilitating diagnosis, Leigh Syndrome (LS), a progressive neurodegenerative disorder.

Nearly 17 years after his brother’s diagnosis, Potter hopes to once again find himself by the bedside, this time wearing a white coat. When he was younger, there was little he could do for his brother. Today, he’s hopeful he’ll be able to help those in need and that his experiences at Seattle Children’s will help him as he begins a medical journey of his own.

My introduction to medicine was one rooted in fear. Over the course of two weeks, I had developed a routine: Sit in the waiting room collecting my courage, wash my hands, get cleared by the nurse to walk into the pediatric intensive care unit (PICU), get up on the step stool to talk to my unconscious brother for five minutes while looking as little as possible at the giant food processor like machine whirring with his blood. I felt helpless, but there was little more I could do to support my younger brother. Read full post »

Gene therapy holds promise of a potentially safer, more effective path to a cure in infants born without the critical infection-fighting cells of the immune system.

Out of every 60,000 births, a baby arrives to face the world without a fully functioning immune system leaving them unequipped to fight even the most common infections. Children with this rare life-threatening genetic condition, known as severe combined immunodeficiency (SCID), have the best chance at a healthy future if they undergo a stem cell transplant before they are 3 ½ months old.

Seattle Children’s recently opened a clinical trial that is seeking a potentially safer, less aggressive and equally effective path to a cure by using a novel gene therapy to fix the faulty gene that causes the most common type of SCID.

On the Pulse met with the trial’s principal investigator, Dr. Aleksandra Petrovic, a pediatric transplant specialist and researcher at Seattle Children’s Research Institute’s Center for Immunity and Immunotherapies, to learn more about the experimental therapy available through this trial. Read full post »

Can a child’s neighborhood affect his or her weight status, diet, and activity level? According to new research published today in Obesity, the answer is yes.

Dr. Brian Saelens, a principal investigator at Seattle Children’s Research Institute who led the study, said children living in neighborhoods with favorable nutrition and activity environments, meaning the neighborhoods had at least one high quality park and were more walkable and there was access to healthy foods or less access to less healthy foods, continue to have lower rates of obesity when compared to children living in less favorable neighborhoods. Less favorable neighborhoods were defined as having no or lower quality parks and either no supermarket or a higher concentration of fast food restaurants. Read full post »

At Seattle Children’s, Emma received the correct diagnosis and treatment for her hemangioma.

Donna West remembers her daughter, Emma, being born with a mark on her face. Part of her right cheek was raised and dark purple, like a bruise. A dermatologist diagnosed the mark as a benign extravascular hemangioma, a term that is no longer used, and said not to be concerned. A hemangioma is a collection of extra blood vessels in the skin.

However, as years passed, the hemangioma grew larger and began to hurt. When Emma, now 11, would hang upside down in the playground, it would throb. If she coughed, the mark would get inflamed and shiny.

“When she would have a coughing spell from her asthma or a cold, the pain was like clockwork,” West said. “We were told it could go away, but that wasn’t happening.”

Until receiving care from Dr. Jonathan Perkins, clinic chief of vascular anomalies at Seattle Children’s, who performed surgery on Emma with the help of a new technique called facial nerve mapping, the family didn’t have an accurate diagnosis for the hemangioma or knowledge of treatment options. Read full post »