Making gene therapy available at the point of care

24 October 2016

New ‘gene therapy in a box’ could deliver gene modified stem cell treatments at the point of patient care, modifying cells to treat specific diseases without the need for a specialised lab.

Although the potential applications of stem cell therapies are many and promising, the infrastructure needs and the cost of implementing therapies on a national or global scale is extensive. Now a team has developed a system which they have dubbed ‘gene therapy in a box’ which works at the point of patient care, reducing costs and speeding up the process.

The research and development was spearheaded by Dr Jennifer Adair, from the Fred Hutchinson Cancer Research Center and the University of Washington. 

Gene-therapy trials have delivered promising results but the process is slow and unwieldy, requiring extensive laboratory work in expensive clean rooms. In total, the process can take around 86 hours per patient, and requires extensive facilities, staff and equipment.

Dr Adair’s team looked instead at modifying and reprogramming an existing device into a machine capable of handling the whole process in a sealed environment. Their results, published in a paper in Nature Communications, show a substantial reduction in the time, staff and material cost to produce modified blood cell products for transplantation. The final product appears to be as effective as the standard gene therapies when tested in mouse models of human disease.

Although currently untested on humans, Dr Adair hopes that the device will democratise gene therapy, and that the relatively low cost and accessibility improvements will facilitate "more people being able to explore these processes and potentially incorporate their own changes".