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News Beat January 2018 Feature Story
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Seattle Children's Research Institute is building the cure for the childhood diseases 




In 2017, Seattle Children's Hospital announced a $1 billion campaign, It Starts With Yes, the largest in its 110-year history. The campaign includes a stunning new research facility in downtown Seattle and a $500 million investment in research to end childhood diseases. Dr. James Hendricks, President of Seattle Children's Research Institute, visited with Life Science Washington about this historical moment and what's ahead in precision medicine, immunotherapy and more. 





2017 was a tremendous year for Seattle Children Research Institute with the Building Cure groundbreaking and the announcement of the $1 billion campaign. What excites you most about the campaign and the new research space in downtown Seattle?


Building Cure is Seattle Children’s investment in cures for childhood diseases. When the new building opens in 2019, it will double the size of our research institute campus to more than 1 million square feet. Building Cure will help us accomplish more life-changing research that will make a difference for kids all around the world with cancer, diabetes, sickle cell anemia and other childhood diseases. For example, we’ll be able to treat 1,000 cancer patients a year with groundbreaking T-cell immunotherapies, compared to the 150 patients a year we can treat with current resources. 



How will the new space facilitate more collaborative efforts in life science leaders across our industry?


Seattle Children’s is a world leader in developing a type of cell therapy called gene therapy. Gene therapies fix the genes that cause disease by inserting new, synthetic genes into cells, repairing the genes in those cells, disabling faulty genes or manipulating genes, to change a cell’s function. Joint efforts with other industry leaders in this space will be key to moving research advances forward from our labs to clinical trials that reach patients. Through collaborations with biotech companies and other institutions, we’ll create new ways to engage the immune system to treat a wide range of diseases, from cancer to HIV to diabetes and other autoimmune diseases.


To support clinical trials that result from these collaborations, Building Cure will house a larger facility for manufacturing cell therapies, substantially boosting our capacity to develop T-cell and stem cell products, viral vectors, and other innovative cell-based products. Called the “Cure Factory” the new facility will speed our progress and help our researchers pursue cell therapies for dozens of childhood diseases.



The Research Institute also announced this fall that it has licensed a new gene therapy technology to Casebia Therapeutics. What made Casebia the right partner for Seattle Children’s and what will be the most important outcomes of your work with them?


The research collaboration and exclusive license agreements with Casebia build on pioneering discoveries made by teams at Seattle Children’s to engineer, manufacture and expand T-cell therapies. Through significant funding for research at Seattle Children’s and access to Casebia’s innovative gene-editing tools, the agreements provide an opportunity to rapidly advance this work. Specifically, the teams will combine efforts to develop gene-edited T cells to treat a broad-spectrum of autoimmune diseases. Such therapies would offer considerable improvement over current therapies, which broadly suppress the immune system and make the body vulnerable to infection and other malignancies.  



Numerous organizations and companies in our industry are working in this area of immunotherapy as well. What do you think will be the most important outcomes to work toward in the next decade?


In 2017, Seattle Children’s opened three new clinical trials offering innovative chimeric antigen receptor (CAR) T-cell immunotherapies for children and young adults with relapsed or refractory acute lymphoblastic leukemia. Currently there are four Pediatric Leukemia Adoptive Therapy clinical trials underway at Seattle Children’s (PLAT-02, PLAT-03, PLAT-04 and PLAT-05), with others expected to open in 2018. While first generation CAR T-cell immunotherapies have proven effective at initially eliminating cancer in patients who are very sick, preventing relapse continues to present a problem in immunotherapy trials across the U.S.


In phase 1 of the ongoing PLAT-02 trial at Seattle Children’s, 93% of patients achieved complete initial remission. However, about half of those patients relapsed within a year. Our researchers are dedicated to improving the therapy with the goal of long-term remission for all patients – and ultimately – a cure. PLAT-03, PLAT-04 and PLAT-05 each feature an innovative immunotherapy strategy to address why cancer is able to return after the initial CAR T-cell therapy. As we continue to make progress in leukemia, we are confident that the promise of immunotherapy can extend to other cancer types, as well as to other diseases like HIV and type 1 diabetes. 



Another exciting announcement recently was the $50 million gift to create the Brotman Baty Institute for Precision Medicine, a collaborative effort between University of Washington, Fred Hutchinson Cancer Research Center and Seattle Children’s. What will be the biggest challenges and opportunities for the institute as you pursue this new frontier of personalized patient care?


Precision medicine has been a strategic priority of Seattle Children’s and will continue to remain at the forefront of our research with the launch of the Brotman Baty Institute for Precision Medicine. We’re already witnessing how sequencing technologies can help provide a genetic diagnosis for children with epilepsy. In some cases, this information can help a neurologist better direct which therapies may be most effective in stopping the child’s seizures. In cancer, we’re matching patients to therapies based on the genetic makeup of their cancer through the High-Risk Leukemia Program. Another trial is studying the use of a novel drug to target a genetic mutation found across different types of cancer.


With precision medicine and genetic sequencing, come vast troves of data. Interpreting this data into clinically significant advancements for patients will be important work of the Institute. The efforts will support ongoing research to identify the genetic mutations behind diseases, so that we can develop individualized therapies that target the root cause of a patient’s disease. Our goal is to find new strategies with the potential to cure disease, not just treat the symptoms.   







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