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Anti-viral T-cell therapy research for pediatric patients gets $4.8 million boost

Children’s National Health System and Children’s Hospital of Los Angeles awarded grant to train T-cells to attack life-threatening viruses in pediatric patients December 19, 2017

WASHINGTON – Children’s National Health System and Children’s Hospital Los Angeles (CHLA) have been awarded $4.8 million by the California Institute for Regenerative Medicine (CIRM) to study the use of T-cell therapy to prevent and fight dangerous viral infections in immunocompromised patients. This grant establishes the largest multi-center pediatric clinical trial studying anti-viral T-cell therapy. 

“It is our hope that with these trained T-cells, we can help the most vulnerable patients fight off life-threatening viral infections,” said Michael Keller, M.D., the Children’s National lead for the trial. “This trial builds on the cellular therapy trials already underway at Children’s National, and we’re thrilled to see this therapy expand to children across the country through this collaboration,” said Keller. 

The trial will target three common viruses: Epstein-Barr virus (EBV), cytomegalovirus (CMV) and adenovirus. While these viruses cause fatigue, sore muscles, sore throat and swollen glands in otherwise healthy patients, they can lead to severe organ damage, and in some cases, death in patients with weakened immune systems. 

“When patients have severe inherited immune deficiencies or are intensely immune suppressed after a bone marrow transplant, standard antiviral medications are sometimes not enough, and patients can die from common viral infections,” explained Michael Pulsipher, M.D., the CHLA lead for the trial. “Patients often need at least some function of their own immune systems in addition to anti-viral medications in order to clear these infections, but sometimes the patient’s own T-cells are not up to the task.”

As part of this clinical trial, Keller, Pulsipher and their collaborators will use T-cells from healthy donors that have been trained and expanded to target these specific viruses, then preserved in a donor “bank” at Children’s National for use in the trial. Once the cells are individually matched to specific patients based upon their genetic makeup and the viral infection the patients are experiencing, they are shipped to individual centers for infusion. After infusion, the virus-specific targeted T-cells can not only control the active infection, but also can help prevent other infections.

The study, which is expected to include up to 30 centers, will be run through the Pediatric Blood and Marrow Transplant Consortium (PBMTC) Operations Center at CHLA. It was developed and is being performed in collaboration with the Primary Immune Deficiency Treatment Consortium (PIDTC). Cell manufacturing for use in the clinical trial will be conducted by the Program for Cell Enhancement and Technologies for Immunotherapy (CETI) at Children’s National Health System.

Media Contact: Rebecca Porterfield Siddon | 202-476-4500

About Children's National Health System

Children’s National Health System, based in Washington, D.C., has served the nation’s children since 1870. Children’s National is one of the nation’s Top 5 pediatric hospitals and, for a second straight year, is ranked No. 1 in newborn care, as well as ranked in all specialties evaluated by U.S. News & World Report. It has been designated two times as a Magnet® hospital, a designation given to hospitals that demonstrate the highest standards of nursing and patient care delivery. This pediatric academic health system offers expert care through a convenient, community-based primary care network and specialty outpatient centers in the D.C. Metropolitan area, including the Maryland suburbs and Northern Virginia. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is the seventh-highest NIH-funded pediatric institution in the nation. Children’s National is recognized for its expertise and innovation in pediatric care and as a strong voice for children through advocacy at the local, regional and national levels. 

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