A table-top 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 multi-million-dollar "clean rooms," making gene therapy more possible for even the poorest of countries.
Gene therapies or cell therapies that involve genetically modifying cells are available at only a limited number of research centers that can afford the necessary technology and the highly trained staff. There are a dozen or so worldwide.
‘Gene therapies in a box is emerging as an affordable treatment that is available to all clinics worldwide. It can be used deliver potential new therapies for cancer, HIV and other diseases.’
No gene therapies are approved yet for use in the United States. But thousands of patients with at least 15 or 20 inherited or infectious diseases and cancers are being treated with experimental therapies, and many are showing promise.
More than 25 million of the estimated 36.7 million people worldwide living with HIV are in sub-Saharan Africa, according to the World Health Organization. No country there could support multi-million-dollar clean rooms or afford the sky-high costs of whatever therapies might come out of them.
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 that will be published in Nature Communications
, the adapted cells also successfully repopulated the blood system when tested in two different animal models.. It is yet to be tested 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."
The semi-automated "point of care" delivery system developed by Adair's team using instrumentation available from Miltenyi Biotec 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.
"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."
The "box" itself costs about $150,000 to purchase -- a one-time investment that would be used for thousands of patients. An individual kit specific to the disease being treated would cost about $26,000, Adair said. Though not inexpensive, the box could drive down costs of gene therapy because it requires less infrastructure and staffing. Even putting aside the clean-room and other infrastructure costs, it could be less than what cell-based gene therapy treatment costs research institutions now -- between $38,000 and $55,000, according to Adair.