Gene therapy for cancer is not science fiction

I watch and read a lot of science fiction. And while the research I’m about to share with you may seem drawn from the plot of a science fiction story set in the distant future, it’s something much 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 new and important for several reasons.

First, it’s gene therapy for cancer! Gene therapies involve the use of a delivery mechanism (usually a modified virus) to replace a dysfunctional or missing gene.)

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

In this study, oncologist and researcher Timothy Cripe and his team used the adeno-associated virus (AAV) not to replace a gene, but to deliver an entirely new gene. Once the gene was delivered by intravenous infusion, cells in the body (mainly in liver and muscle) in the preclinical model began to produce the therapeutic protein and secrete it into the bloodstream. This allowed the model to fight malignant cancer cells in several ways. The study specifically tested the effectiveness of using the therapy to treat B-cell malignancies.

Immune cells expressed the therapeutic protein for more than a year, allowing a sustained attack on cancer cells, shrinking tumors over time. The therapy was persistent and eroded the tumor without letting it grow back. The researchers also observed no adverse effects in the preclinical model.

Another reason why this therapy is unique is that it is meant to be “off the shelf”. In other words, it can be produced in bulk, stored and delivered to patients without the need for customization. Most cancer immunotherapies must be made to order because they must be tailored to each patient and their cancer.

When you have to wait for personalized immunotherapy, sometimes requiring donor cells, this can delay treatment. An out-of-the-box option is designed to be available when needed.

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

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

The technologies present in the therapy described in this article could open a new era of possibilities for using gene therapy to treat many types of diseases, without any science fiction.

Abbie Roth is the editor of Nationwide Pediatrics and Science Communication at Nationwide Children’s Hospital.

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