![]() “We want to replicate that progress for many other cancers and diseases.” Fixing faulty genesĭrs. Jim Hendricks, president of Seattle Children’s Research Institute. “We have a long way to go, but we’re getting the first glimpse of a therapy that could wipe out cancer and be far less toxic than today’s treatments,” says Dr. We’re leading one of the nation’s first clinical trials of T-cell therapies for neuroblastoma. Now our researchers are developing ways to prevent relapse and designing immunotherapies for other more complicated cancers. “We’re so grateful for what this therapy did for Erin – and it’s amazing to think that immunotherapy for leukemia is just the beginning,” says her mom, Sarah Cross. The experimental treatment put her cancer into remission and helped her get healthy enough to undergo a bone marrow transplant that could cure her cancer once and for all. Michael Jensen’s team at the Ben Towne Center for Childhood Cancer Research develops these therapies, tests them in our labs and creates them in our special FDA-approved manufacturing facility.Įrin and her parents flew to Seattle to participate in our first immunotherapy clinical trial. This approach reprograms T cells by inserting genes that tell them to find and kill cancer cells. ![]() Then her family, of Chester, England, heard that Seattle Children’s was studying a cell therapy called cancer immunotherapy. She was 5 years old and out of treatment options. ![]() The Cross family came to Seattle Children’s from Chester, England, so their daughter, Erin, could join our cancer immunotherapy clinical trials.Įrin Cross’s leukemia relapsed last March. “The next step is to find cures.” Redefining cancer therapy Bruder Stapleton, Seattle Children’s chief academic officer. “In the past few decades, researchers have made tremendous progress on treatments that control diseases or their symptoms,” says Dr. Now we want to invest big to speed up our progress and help our researchers pursue cell therapies for dozens of other diseases - from diabetes to HIV. In our initial studies, these therapies have shown promise against leukemia and severe immune disorders. “Other times, the instructions fix genetic mistakes that cause a disease, with the goal of curing it at the source.” “Sometimes, the new instructions tell the cells to fight back against disease,” says Dr. Then they change the genetic instructions in those cells and infuse them back into the patient. To create cell therapies, researchers remove a small number of cells from a patient’s blood. Instead, they’ll get cell therapy – a single infusion of cells that cures their disease forever. Researchers at Seattle Children’s are working toward a day when kids like Sophia and Preston won’t need lifelong treatment. “I never stop worrying about what might happen to my kids.” They both died much earlier than they should have,” Miele says. “My uncle had diabetes and had both legs partly amputated. It’s just one example of how Nguyen and her husband, Chris Miele, spend their lives on high alert, acutely aware that how well they manage Sophia and Preston’s diabetes affects their children’s long-term risk of stroke, heart disease and other serious health problems. Then she waits up to make sure their blood sugar gets back into the safe range. But most nights Nguyen has to walk into her kids’ bedrooms to deliver an insulin shot or stuff fruit snacks into their cheeks. On a good night, their levels are normal. Sophia, 9, and Preston, 7, both have type 1 diabetes – if their blood sugar falls too low or climbs too high, they could have seizures or slip into a coma. Several times a night, Julie Nguyen wakes up and squints at the smart watch that tracks her children’s blood sugars. Since her children Preston, (left) now 7, and Sophia, now 9, were diagnosed with type 1 diabetes, Julie Nguyen must track their blood sugar levels around the clock to manage the short- and long-term risks associated with their illness Published in Connection magazine, Spring 2017 We’re inserting new genetic instructions into cells to develop therapies that could cure diseases once and for all.
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