Fred Hutch clinical researcher Kiem wins $5 million for gene therapy work

Principal investigators Kiem and Rawlings, along with other researchers, will collaborate and pursue various components of the therapy, with an aim of clinical trials for patients at the end of five years. This will be a first use of foamy viruses in a clinical trial.

A foamy virus is a type of retrovirus capable of allowing its genetic information to become part of the genetic information of a host cell. The use of this foamy virus vector as a clinical vector for gene delivery was developed in Kiem's Clinical Research Division lab. The Center's portion of the award is $5,025,500. "We think foamy virus vectors will be safer than other vectors being used for this disease," Kiem said.

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 this study. SCID is often called "bubble boy disease," due to publicity surrounding David Vetter, a boy with SCID who lived for 12 years in a plastic, germ-free environment.

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. A bone marrow transplant is the only curative therapy for patients that survive initial infections. Many children die of infection before they are diagnosed.

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 or bone marrow transplantation before they suffer from an infection," Rawlings and Kiem said. 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:

• Drs. Kiem and Brian Beard, also of the Clinical Research Division (modeling preclinical therapies, producing foamy virus vector for preclinical and clinical applications, and tracking vector integration)
• Drs. Rawlings, Andrew Scharenberg and Troy Torgerson, Seattle Children's Research Institute (modeling preclinical therapies and immune function studies)
• Dr. Peter Felsburg, University of Pennsylvania (assisting with preclinical therapies)
• Dr. Grant Trobridge (former Kiem Lab member), Washington State University (optimizing vector safety and tracking vector integration)
• Dr. Scott Wilbur, University of Washington (modeling preclinical therapies).

[Adapted from a Seattle Children's Research Institute news release]