Researchers Develop ‘Gene Therapy in a Box’

Device bypasses expensive ‘clean rooms’

Scientists at the Fred Hutchinson Cancer Research Center in Seattle have created a table-top device that would allow medical staff to genetically manipulate a patient’s blood to deliver potential new therapies for cancer, human immunodeficiency virus (HIV) infection, and other diseases, thus eliminating the need for multimillion-dollar “clean rooms.”

The so-called “gene therapy in a box” delivers modified blood stem cells that are as good as or better than those manufactured in highly regulated clean rooms and requires less than half the staff, according to the researchers. The adapted cells successfully repopulated the blood system when tested in two different animal models. The team’s findings were published in Nature Communications.

“Gene therapies or cell therapies that involve genetically modified cells are not restricted to a very small number of highly sophisticated facilities anymore,” said lead author Dr. Jennifer Adair.

No gene therapies have been approved for clinical use in the United States, but thousands of patients with inherited or infectious diseases and cancers are being treated with experimental gene therapies, and many are showing promising results, the researchers said.

Because the process of modifying blood stem cells is complex, requiring high-tech facilities and highly trained staff, studies are limited to approximately a dozen research centers in eight countries—the United States, France, Germany, Italy, Spain, the United Kingdom, China, and Australia. The new semi-automated “point of care” delivery system reduces the space required to produce the modified cells from 500 square feet to less than 5 square feet, and the staffing from five to 10 people to one or two––and it does the job in less than half the time, according to the authors.

Adair and her team worked to adapt a machine called the CliniMACS Prodigy. This device is a closed system, meaning there is no exposure to ambient air; it can be automated; and its interface is similar to that of an apheresis machine.

The scientists reconfigured and reprogrammed the CliniMACS Prodigy device so that it could perform the clean-room jobs of adding the viral vector and removing residual reagents, and of developing components specific to each disease. These components would be available in “kits” and kept in pharmacy freezers. Included in each kit would be disposable tubing to carry the patient’s blood cells from a sterile bag into the machine. After attaching the bag to the machine, a nurse would add chemical reagents from the kit to pull out the stem cells; nutrients to support the growth of those cells; and the viral vector engineered to perform the gene transfer for that particular disease. Additional disposable tubing would carry the modified cells to a second sterile bag, which would provide the cells to the patient via his or her intravenous line.

The “box” itself costs approximately $150,000 to purchase, and each individual kit would cost about $26,000.

Source: Fred Hutchinson Cancer Research Center; October 20, 2016.