NIH Awards $12 Million for Gene Therapy Work

Gene repair lab

Seattle Children’s Research Institute, Fred Hutchinson Cancer Research Center, U Penn, UW, WSU push toward clinical trials for Severe Combined Immunodeficiency


Seattle Children’s Research Institute and partner organizations have been awarded more than $12 million over the next five years from the National Institute of Allergy and Infectious Diseases of the National Institutes of Health to develop foamy virus gene replacement therapy for patients who suffer from Severe Combined Immunodeficiency. Researchers from Fred Hutchinson Cancer Research Center, University of Pennsylvania, University of Washington and Washington State University will collaborate with Seattle Children’s Research Institute and pursue various components of the therapy, with an aim of clinical trials for patients at the end of five years. There has never been a clinical trial conducted using foamy viruses.

A foamy virus is a type of retrovirus, which means it has the capability of allowing its genetic information to become part of the genetic information of a host cell. The use of foamy viruses as a clinical vector for gene delivery was developed in Seattle.

Severe Combined Immunodeficiency, or SCID, is a rare syndrome caused by mutations in at least 13 different genes. The most common form, SCID-X1, is the focus of the new study. SCID is now often called "bubble boy disease,” becoming more widely known during the 1970 and 1980s when the public learned about David Vetter, a boy with SCID who lived for 12 years in a plastic, germ-free environment. The “bubble boy” story was featured in a made-for-TV movie starring John Travolta and on an episode of the popular television show, “Seinfeld.”

Patients with SCID have defective B- and T-cell function, which results in the onset of one or more serious infections within the first few months of life. These infections—including pneumonia, meningitis and bloodstream sepsis—are life-threatening emergencies requiring complex medical care. Treatment using marrow transplantation represents the only curative therapy for patients that survive initial infections.

“SCID is a rare condition and affects about one in 30,000 newborns,” said David Rawlings, MD, of Seattle Children’s Research Institute, co-leader of the project with Hans-Peter Kiem, MD, Fred Hutchinson Cancer Research Center. “Among Hispanic newborn babies, that figure might be even higher, or one in 15,000. Many children still die of infection before they are diagnosed,” said Dr. Rawlings, who is also chief of the Immunology Division at Seattle Children’s Hospital.

Scientists in Seattle are actively developing several different facets of gene therapy, including gene replacement and gene repair for SCID and related diseases.

“In the future, we hope that all SCID babies are diagnosed by newborn screening, and cured using foamy viral gene therapy before they suffer from an infection,” said Dr. Rawlings. Newborn screening for SCID is now in place in 34 states and is under consideration in Washington state.

The experts aligned on this project are among the top in the field of gene therapy. The senior leaders of the team include:
• David Rawlings MD, Andrew Scharenberg, MD, and Troy Torgerson, MD, PhD, Seattle Children’s Research Institute (modeling preclinical therapies and immune function studies)
• Hans-Peter Kiem, MD, Brian Beard, PhD, and Ralf Luche, PhD, Fred Hutchinson Cancer Research Center (modeling preclinical therapies, producing virus and tracking vector integration)
• Peter Felsburg, VMD, PhD, University of Pennsylvania (modeling preclinical therapies)
• Grant Trobridge, PhD, Washington State University (optimizing vector safety and tracking vector integration)
• Scott Wilbur, PhD, University of Washington (modeling preclinical therapies)

The majority of the $12 million award will remain in Washington state, and will help boost the state’s economy. This comes at a time of dwindling federal grants and a much more competitive process from the National Institutes of Health. Several laboratory positions at Seattle Children’s Research Institute will be preserved, thanks to the award.

The research project is supported by the National Institute of Allergy And Infectious Diseases of the National Institutes of Health under Award Number P01AI097100.

Seattle Children’s Research Institute, Fred Hutchinson Cancer Research Center, U Penn, UW, WSU push toward clinical trials for Severe Combined Immunodeficiency

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Seattle Children’s mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Children’s Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

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