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Seattle Children’s trial to help treat IBD, bone marrow transplant patients
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Seattle Children’s Research Institute has opened the first precision medicine clinical trial, which applies novel T-cell receptor sequencing and single-cell gene expression analysis, to help treat the immune system of pediatric IBD and bone marrow transplant patients, according to a press release.
The trial — known as the Precision Diagnostics in Inflammatory Bowel Disease, Cellular Therapy and Transplantation, or PREDICT — will allow clinicians to personalize treatment of IBD and help to identify graft-versus-host disease (GVHD), a deadly complication of bone marrow transplant.
“PREDICT seeks to change the paradigm of treatment by first changing the paradigm of diagnosis,” Leslie Kean, MD, PhD, the trial’s principal investigator and associate director of the Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute, said in the release. “By gaining foundational molecular diagnostic knowledge about a patient’s T cells, we hope to ultimately discover better treatment approaches for IBD and GVHD.”
Standard treatments to suppress or modulate the immune response do not always effectively manage diseases like IBD and GVHD, suggesting that new, targeted therapies are necessary. Therefore, Kean and colleagues aim to perform T-cell receptor (TCR) sequencing and gene expression analysis on samples collected from 100 IBD and 250 bone marrow transplant (BMT) patients to help pinpoint molecular pathways active within a patient’s T cells at baseline and follow-up analyses. Identifying these specific pathways can serve as therapeutic targets in future studies.
In collaboration with Adaptive Biotechnologies and 10x Genomics, the researchers will implement a high-throughput TCR sequencing and conduct TCR repertoire analyses and a single-cell gene expression analysis. Over the next 2 years, they will enroll pediatric patients with IBD undergoing evaluation and treatment at Seattle Children’s, then will open the BMT cohort later this year.
“IBD and GVHD have a lot more in common than meets the eye when it comes to the underlying immune response they trigger,” Kean said. “PREDICT aims to bridge IBD and GVHD, shedding new light on the immunologic similarities they share and identifying the molecular causes of each patient’s disease. This will create a unique opportunity to make significant headway in the treatment of both diseases, with the focus on each child and their unique disease signature.” – by Savannah Demko
Disclosures: Kean is the associate director of the Ben Towne Center for Childhood Cancer Research at Seattle Children’s Research Institute.