Jennifer Adair Fred Hutch gene therapy

Fred Hutch’s Dr. Jennifer Adair stands before the portable “gene therapy in a box” device that she began developing when she became head of her own lab in 2014 | Photo by Robert Hood / Fred Hutch News Service

‘Gene therapy in a box’ could bring the benefits of highly tailored cancer and HIV therapies to even the poorest countries, according to a study published last week in Nature Communications. 

The tabletop device, developed by scientists at Fred Hutchinson Cancer Research Center, delivers modified blood stem cells that were as good as those developed in a multi million-dollar clean room. The system also required less than half the usual number of staff. In two different animal models the adapted cells successfully repopulated the blood system. 

As yet there have not been any test in humans. 

“We either had to think about how to build million-dollar infrastructure and clean-room facilities in clinics all around the world, which is not feasible, or we had to think about simplifying this process into what I originally envisioned as a black box,” said Fred Hutch researcher Dr. Jennifer Adair, the study’s lead author. “This was the first proof that ‘gene therapy in a box’ could work. Gene therapies or cell therapies that involve genetically modified cells are not restricted to a very small number of highly sophisticated facilities anymore.”

While no gene therapy has been approved for clinical use in the US as yet, thousands of patients with cancer, inherited conditions and infectious diseases are being treated with experimental therapies. Gene therapy studies are typically limited to a handful of research centres that can provide the high-tech facilities and staff training required. The study authors hope that this new approach will expend the availability of gene therapy. 

“This is truly transformative,” said senior author, oncologist Dr. Hans-Peter Kiem. “It will change the way we manufacture and deliver cell and gene therapy products and will have a major impact on making stem cell gene therapy and transplantation and likely also immunotherapy available to patients with genetic diseases, HIV and cancer worldwide.”