Gene therapy — the process of genetically altering cells to treat disease — is a highly promising process being studied as a way to cure devastating conditions like genetic disorders, HIV, and even cancer.
But despite the great need for medical advances in these areas, gene therapy can only be performed at a handful of high-tech clinics around the world and require highly trained staff, meaning that it may never be accessible to the millions of people whose lives it could save.
Enter “gene therapy in a box,” a table-top device developed at the Fred Hutchinson Cancer Research center in Seattle, which could provide gene therapy treatments without the expensive and rare medical infrastructure currently needed.
A study published today in Nature Communications found that the device produced stem cells that were as good or better than cells prepared in a traditional clean room, and required less than half the staff needed for traditional methods. The study’s findings could have huge implications for developing countries, particularly areas with high rates of HIV.
“My hope is that this technology will be available in clinical centers around the world, even in less developed regions, and could open the door to saving millions of lives,” Dr. Jennifer Adair, a medical oncology researcher at Fred Hutch and the study’s lead author, told GeekWire in an email.
Generally speaking, gene therapy involves removing blood stem cells from a patient’s blood sample, multiplying the stem cells, then genetically altering them using a virus. The altered stem cells are then returned to the patient’s blood via an IV.
Normally this process requires several pieces of specialized equipment, a specialized clean room, and hours of work by highly trained staff. The new device is point-of-care, meaning it is used in the same room as the patient, and takes up less time, space, and staff than traditional approaches.
Check out the graphic below to compare the process of traditional clean room gene therapy treatment, and the process using the new point-of-care device.
The Fred Hutch team used an instrument from Miltenyi Biotec as the basis for their device, then altered it to perform the complex steps required for gene therapy. The finished device costs about $150,000 to purchase, and a kit of the materials required for one treatment would cost about $26,000.
Those are relatively low numbers compared to the cost of operating and staffing a clean room, or the lifelong treatment cost for individuals with HIV, for example. The device also shrinks the space needed for treatment from 500 square feet to about five, and the staff required from five to ten people to only one or two people.
Although the device must still pass FDA hurdles and be vetted by clinical trials, Adair is excited about its potential.
“I am optimistic this technology could be widely available in the next few years,” Adair said. The unaltered devices are available off the shelf, and Adair said her team is working with Miltenyi Biotec to make the altered device available. She also said they are working with Rocket Pharmaceuticals to study the device in treating Fanconi anemia, a rare genetic disease that makes patients highly susceptible to developing cancer.
“Diseases currently being treated with stem cell gene therapy in clinical trials include inherited immune deficiencies, hemoglobinopathies, sickle cell disease, cancer, HIV, leukodystrophies and other inherited disorders,” and all of these could potentially be treated using the device, she said.
That could save lives in areas like Sub-Saharan Africa, where over 25 million people are currently living with HIV but there are few resources for treatments like gene therapy.
The device could also be used to prepare CAR T immunotherapy treatments for cancer, a process which involves altering a patient’s T cells — important actors in the immune system — to attack cancer cells.
The process has shown promise in early studies, and Adair and her team programmed the device to perform the extra steps required for the treatment, although they were not included in the study released today.
Although the device has a ways to go before it can be used in hospitals, its early promise shows may be possible to make gene therapy available to anyone who needs it, not just a few rich countries and individuals.