INDIANAPOLIS (WTWO/WAWV) — An Indiana University researcher will lead a multi-institute effort geared toward improving hemophilia therapies with the help of a $12 million grant from the National Heart Lung and Blood Institute.
Led by IU School of Medicine’s Roland Herzog, the program project award will fund studies exploring three major themes in a gene therapy approach that could lead to safer treatments for the disorder.
“Several companies have taken this forward into clinical trials, and in some of these trials, the patients initially looked like they were cured,” Herzog, who is the Riley Children’s Foundation Professor of Immunology, said. “But what they all have in common is that they need to deliver a lot of the virus in order to get the desired results, and over time clotting factor levels started to decline. So, it’s clear that we need to further study the biology of this phenomenon.”
The program will focus on three major projects in gene therapy for hemophilia A:
- Project 1 will focus on cellular toxicity and stress that can be induced by FVIII protein production. This project is led by Randal J. Kaufman, director of the Degenerative Diseases Program at the Sanford Burnham Prebys Center for Genetic Disorders and Aging Research.
- Project 2 is led by IU School of Medicine professor of pediatrics Weidong Xiao. Xiao’s research project focuses on molecular virology and the development of viral vectors used in gene therapy to deliver the FVIII-encoding gene.
- Project 3 will examine the immune system and its role in the interference of FVIII production over time. It is jointly led by Herzog and Ype de Jong, MD, PhD, assistant professor of medicine at the Sanford I. Weill Medical College of Cornell University.
To help support the program, the grant will also fund a core that generates human liver cells and a core that manufactures gene therapy vectors and analyzes them on a molecular level. Additionally, the program will receive regular counsel with an internal and external scientific advisory board.
“This is an incredibly significant and urgent medical question, and it requires the synergy ofmultiple groupswith different expertise to cometogether and solve a problemthat they wouldn’t be able to solve on their own,” Herzog said. “My hope is that our studies will help the field as a whole move toward curing hemophilia A.”
