The first US trial using CRISPR to treat disease in people is imminent, a significant step in testing the true power of gene editing in the clinic. In the future, the US government is hoping that therapies for cancer and other diseases using gene editing will reach a lot more patients. To get there, this week it announced it will dole out $US190 million ($236 million) in research grants over the next six years to help move things along.
Gene editing techniques such as CRISPR have radically altered the field of biomedical research, presenting scientists with the possibility of treating disease by simply editing it out of a person’s body. There are still big hurdles, though, in moving the technology from research labs to the clinic. One recent paper, for example, suggested that CRISPR could trigger an immune response in many people that could render clinical treatments with CRISPR either ineffective or dangerous. And to treat many diseases, better techniques must be developed to efficiently deliver CRISPR to the right place in the body. Further, there are still many unknowns surrounding the safety of CRISPR and potential off-target effects.
The National Institutes of Health’s new Somatic Cell Genome Editing will award funds over six years to researchers working to solve problems that will help accelerate research across the field, such as developing better delivery mechanisms or better methods for gene editing. (The agency is only funding research for human somatic cells, the body’s non-reproductive cells, meaning no designer babies.)
“The focus of the Somatic Cell Genome Editing program is to dramatically accelerate the translation of these technologies to the clinic for treatment of as many genetic diseases as possible,” NIH Director Francis Collins said in a statement.
There is one fairly large caveat: The agency has enough funding to launch the program this year, but annual budget appropriations could affect program funding in the future.
Either way, this is a fairly big sign that the US is placing a lot of stock in gene editing to solve the problems of disease in the future.