Pediatric research: Gene therapy for cancer is no science fiction

I watch and read a lot of science fiction. And while the research I’m about to share with you might sound like it was ripped from the plot of a science fiction story set in the far future, it is something far better —real science, happening now.

A recent study published in Science Advances reports proof-of-concept results for a gene therapy cancer treatment. This study and the therapy it describes are novel and important for a few reasons.

First, it’s a gene therapy for cancer! Gene therapies involve using a delivery mechanism (usually a modified virus) to replace a gene that is either dysfunctional or missing.)

To date, those gene therapies have been used to treat a variety of genetic disorders involving a single gene.

In this study, Timothy Cripe, an oncologist and researcher, along with his team used adeno-associated virus (AAV) not to replace a gene, but to deliver a whole new gene. After the gene was delivered via intravenous infusion, cells in the body (mostly in the liver and muscle) of the preclinical model began producing the therapeutic protein and secreting it into the bloodstream. This enabled the model to combat malignant cancer cells in several ways. The study specifically tested the effectiveness of using the therapy to treat B cell malignancies.

The immune cells expressed the therapeutic protein for more than a year, enabling a sustained attack on the cancer cells, shrinking the tumors over time. The therapy was persistent and wore away at the tumor while not letting it grow back. The researchers also observed no adverse effects in the preclinical model.

Another reason this therapy is unique is that it is intended to be “off-the-shelf.” That is, it can be produced in bulk, stored and delivered to patients without needing customization. Most cancer immunotherapies must be made-to-order because they need to be made specific to individual patients and their cancers.

When you must wait for a custom immunotherapy, sometimes requiring donor cells, it can delay treatment. An off-the-shelf option is designed to be available when needed.

Finally, the therapy is designed to be on-demand. That is, once the gene is in the body, doctors can turn the production of the therapeutic protein off and on as needed. Once a tumor is gone, the gene can be turned off, but in the event of a remission, it could be turned back on. The ability to turn an immunotherapy on and off on demand could also have a dramatic benefit for chronic diseases where short-term, intermittent therapy is preferred, such as rheumatological disorders.

So what’s next for this new approach to cancer therapy? While it is not available for patients yet, the researchers are now working to establish clinical trials that would enable them to test the therapy in humans. They’re also still working in the lab to see what types of cancers or chronic diseases this therapy could help treat.

The technologies present in the therapy described in this paper could open a new era of possibility for using gene therapy to treat many different types of diseases — with no science fiction involved.

Abbie Roth is managing editor of Pediatrics Nationwide and Science Communication at Nationwide Children's Hospital.

Abbie.Roth@nationwidechildrens.org

This article originally appeared on The Columbus Dispatch: Pediatric research: Gene therapy for cancer is no science fiction