Gene Therapy Going Global with Portable Device
News Oct 25, 2016
A tabletop device that enables medical staff to genetically manipulate a patient’s blood to deliver potential new therapies for cancer, HIV and other diseases would eliminate the need for multimillion-dollar “clean rooms,” making gene therapy more possible for even the poorest of countries.
The so-called “gene therapy in a box,” developed by scientists at Fred Hutchinson Cancer Research Center, delivered modified blood stem cells that were as good as — or better — than those manufactured in highly regulated clean rooms and required less than half the staff, according to a study published in Nature Communications. The adapted cells also successful repopulated the blood system when tested in two different animal models, the study noted. It hasn’t yet been tested in humans.
The portable device suggests a solution to one of the most vexing challenges of gene therapy: how to make these emerging, high-tech treatments accessible and affordable, beyond a handful of specialized research centers to clinics worldwide.
“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.” said Fred Hutch researcher Dr. Jennifer Adair, the study’s lead author.
The semi-automated “point of care” delivery system developed by Adair’s team reduces the space required to produce the modified cells from 500 square feet to less than 5 square feet and the staffing from five or 10 people to one or two, according to oncologist Dr. Hans-Peter Kiem, a Fred Hutch and University of Washington cell and gene therapy researcher and the paper’s senior author. It does the job in less than half the time.
“This is truly transformative,” Kiem said. “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.”
In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell’s internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building. This gives researchers a way not only to eliminate a mutated gene sequence, but to influence how the gene is expressed and regulated.