Seattle Children's Launches Personalized Medicine Trial Focused on IBD, GVHD

May 23, 2017
Seattle Children's Launches Personalized Medicine Trial Focused on IBD, GVHD
The PREDICT trial will apply next-generation T-cell receptor sequencing and single-cell gene expression analysis in a study intended to find new strategies to personalize treatment for pediatric patients. (Source: © Sebastian Kaulitzki/Fotolia)

Seattle Children’s said today it has launched the first clinical trial designed to better understand how the immune system drives both inflammatory bowel disease (IBD) in pediatric autoimmunity patients and graft-versus host disease (GVHD) in pediatric bone marrow transplant patients.

The PREDICT (Precision Diagnostics in Inflammatory Bowel Disease, Cellular Therapy and Transplantation) trial will apply next-generation T-cell receptor (TCR) sequencing and single-cell gene expression analysis in a study intended to find new strategies to personalize treatment for pediatric patients.

PREDICT is expected to first provide clinicians new information about why IBD arises in children, in order to help them tailor treatment plans to each patient. The trial will later expand to include bone marrow transplant patients, with the goal of identifying immunologic changes that occur when patients develop GVHD, the deadliest complication associated with bone marrow transplant.

“PREDICT seeks to change the paradigm of treatment by first changing the paradigm of diagnosis,” said Leslie Kean, M.D., Ph.D., the trial's principal investigator, and associate director of the Ben Towne Center for Childhood Cancer Research at Seattle Children's Research Institute.

“By gaining foundational molecular diagnostic knowledge about a patient's T cells, we hope to ultimately discover better treatment approaches for IBD and GVHD,” added Dr. Kean, whose lab focuses on T-cell immunology.

Dr. Kean and her team will perform TCR sequencing and gene expression analysis on samples collected from 100 IBD and 250 bone marrow transplant patients. Data resulting from the initial and follow-up analyses will help researchers pinpoint molecular pathways active within a patient's T cells that could serve as therapeutic targets in future studies.

Researchers will apply Adaptive Biotechnologies’ immunoSEQ® platform for high-throughput TCR sequencing, and conduct TCR repertoire analyses. Single-cell gene expression analysis will be performed using the Chromium™ Single Cell 3′ Solution supplied by 10x Genomics, which is designed to enable gene expression patterns to be discovered in each patient's individual T cells.

Over the next two years, Seattle Children’s said, Dr. Kean will work in partnership with transplant, gastroenterology and immunology physicians to first enroll IBD patients who are undergoing their diagnostic evaluation and treatment, with plans to open the bone marrow transplant 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. 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,” Dr. Kean added. “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.”

A Seattle Children's Research Institute pilot grant has provided initial funding for the PREDICT trial, with additional support from biopharmaceutical collaboration partners.

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