Forget GMO. Get ready for GMH.
CRISPR, the genome-editing technology that has taken biomedical science by storm, is finally nearing human trials.
On 21 June, an advisory committee at the US National Institutes of Health (NIH) approved a proposal to use CRISPR–Cas9 to help augment cancer therapies that rely on enlisting a patient’s T cells, a type of immune cell.
Where some gene modification techniques have involved the brute-force insertion of genetic material (including shooting at cells with an air pistol), CRISPR is a much more exacting technique that offers multiple approaches to carefully editing genes, including human genes.
What CRISPR offers, and biologists desire, is specificity: the ability to target and study particular DNA sequences in the vast expanse of a genome. And editing DNA is just one trick that it can be used for. Scientists are hacking the tools so that they can send proteins to precise DNA targets to toggle genes on or off, and even engineer entire biological circuits — with the long-term goal of understanding cellular systems and disease.
The trial approved by NIH is intentionally small, and is focused more on whether the technique is safe to use in human beings than on whether this specific use of the technique is effective in stopping cancer. Even so, the precision of the edits in the trial shows just why scientists are so excited about CRISPR and related techniques, and give a hint at how broadly this toolset may be used to address disease in the future.
The researchers will remove T cells from 18 patients with several types of cancers and perform three CRISPR edits on them. One edit will insert a gene for a protein engineered to detect cancer cells and instruct the T cells to target them, and a second edit removes a natural T-cell protein that could interfere with this process. The third is defensive: it will remove the gene for a protein that identifies the T cells as immune cells and prevent the cancer cells from disabling them. The researchers will then infuse the edited cells back into the patient.
In this initial trial CRISPR will be used to insert a gene, remove a protein, and remove a gene. And that’s the first trial in humans.
The interest around these gene editing techniques is continuing to grow. Medical companies based on similar procedures are exploding onto the business landscape, and so are the potential use-cases. This may be the first attempt to use the technique in humans, but unless some unforeseen problem occurs, don’t expect it to be the last.