Scientist Researches Cure for Newborn Immune Deficiency

An inherited immune deficiency fatal in newborns is the target of a new research effort by a team that includes a Washington State University scientist.

Funded for five years by the National Institutes of Health, the gene therapy team includes Grant Trobridge from the Department of Pharmaceutical Sciences at the WSU College of Pharmacy. Funding from the NIH for the entire project is $12 million, with $1.8 million for the WSU component led by Trobridge. Trobridge’s research team will work to improve a virus used to transfer normal genetic material into chromosomes to replace a defective gene. The project also includes researchers from Seattle Children’s Research Institute, the Fred Hutchinson Cancer Research Center, the University of Washington and the University of Pennsylvania.

Gene therapy for the “X-linked Severe Combined Immune Deficiency” condition fatal to infants has worked in clinical trials, but damage to nearby genes from the transfer agent has resulted in leukemia in some patients, Trobridge said.

The transfer agent his lab uses is from a type of virus known as a “foamy virus” and appears to be safer, he said. With the new grant, the team will continue to modify it to make it even safer. One modification will include the addition of chromosomal elements to block the ability of the virus to interfere with normal genes.

“We hope these elements will act as insulation for the normal genes against the virus,” Trobridge said.

The project goal is to ready the virus for future clinical trials.

Trobridge’s other role on the team is to provide computer analysis—or bioinformatics—using software developed by his lab. Trobridge will set up a Web-based data analysis site so collaborating researchers can do some of their own analysis, and his lab also will perform some custom analysis.