Meet Dr. Roland Herzog: Discovery leads to “dream results”

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Roland Herzog, Ph.D.,

Gifts from Riley Children’s Foundation donors are helping Riley Hospital recruit some of the best and brightest investigators from around the globe to our research facility, the Herman B Wells Center for Pediatric Research.

Today, we introduce you to a newly-recruited Wells Center research scientist Roland Herzog, Ph.D., who is building a new program using genetic therapy and immunotherapy to transform lives of children born with hemophilia.

Q: How did you become interested in genetic and immunology research, and what steps led you to Riley and the Wells Center for Pediatric Research?

A: I was born in Germany and came to the U.S. for graduate school in 1992. My Ph.D. from Auburn University is in microbiology, but Auburn doesn’t have a medical school. In terms of biomedical research I knew very little and wasn’t used to it. 

My interest was gene transfer. The field of gene therapy was exploding in the mid-90s. It was a very young field. It seemed to me to be the ultimate application of gene technology: if you could correct someone’s disease with a molecular biology approach, it would be a fantastic thing to accomplish.

I saw an ad that the Children’s Hospital of Philadelphia (CHOP) was looking for a post-doc to work in gene therapy for hemophilia. I knew nothing about hemophilia but I applied anyway and was ultimately offered the position. It took off from there quickly. Within six months I had a paper accepted, which eventually led to a clinical trial for patients with hemophilia. I was hooked at that point. 

Through a casual conversation at a gene therapy society meeting, I was invited to interview at the University of Florida. It was a great opportunity to be in a center that does a lot of gene therapy, and again I was with the Department of Pediatrics. When I was approached by the Wells Center for Pediatric Research, I heard about all of the exciting development―new programs in immunotherapy, and gene and cell therapy. We ultimately decided on a great fit for my interests: I’ll be serving as the director of the new gene and cell therapy program, where I’ll recruit faculty and build this program from the ground up.   

Q: Can you explain some of the work you are doing with hemophilia? 

A: It’s a genetic disease that people are born with. It is on the X chromosome, and only boys are born with the disease. They have a mutation in the gene that normally makes a blood clotting factor. If the gene is missing or doesn’t work, those boys have a problem with their blood not clotting properly and they can have spontaneous internal bleeds. This is a lifelong disease. Patients constantly need injections of clotting factor protein. Within a couple of days, that protein is gone and you have to inject again. 

The goal with gene therapy is to transfer the correct gene into the patient so the patient’s body will start making the proper clotting factor all the time. That means the patient no longer needs injections.   

Early in my career we did an experiment using an engineered virus to transfer the clotting factor gene into the liver cells of a dog with hemophilia. It worked. The dog’s cells started making the right protein. One injection of this virus resulted in lifelong correction of this dog’s disease. During his 13-year life, he never bled once. 

Eventually, various groups around the world have adapted this approach, and there have been a number of clinical trials. In one trial, patients were treated in a similar fashion as the dog using the same type of gene therapy to transfer the gene. They had correction of their disease for more than eight years. 

Several companies are now going into Phase 3 trials. We could see FDA approval for this type of treatment within the next two years. It really is a dream result for a researcher. But there is still more for us to figure out, especially for the 20 to 30 percent of hemophilia patients who reject their therapy because their immune system makes an antibody to the protein.  

Q: What is your message to donors about the importance of the gifts they give to pediatric research?

A: These funds are always super helpful. For example, we will have junior faculty starting on my team and these funds help get them started in their research careers so they can carry the torch later.   

Donations ultimately mean that my team can help the futures of boys who are born with hemophilia, giving these patients a one-time therapy that will correct their disease for many years.

There is a need. This research makes a cure happen. We will help make it safer, make it last, and make sure kids’ immune systems don’t reject the therapy that holds so much promise.

Riley Blogger

The Riley Blog is written and/or edited by members of the Riley Children's Foundation Communications Staff.

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