Imagine being able to sense temperature, pressure and texture, but not pain. Scientists have discovered that for a few people with an extremely rare genetic disorder, that is their reality. Of course, for them, lacking one of the body’s major red flags for danger, the condition presents a permanent risk of harm.
However, with this discovery, scientists believe that treatment at the genetic level could change the lives of people suffering chronic pain. Using a gene-editing tool, they believe they can make selective changes to a patient’s DNA to repress a gene responsible for sending pain signals through the nervous system.
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Established technique
The technique is one already established for other conditions. Recently, it has been used to help people suffering sickle cell disease as a result of a defective gene which resulted in the production of abnormal haemoglobin. By altering the defective gene, patients revert to having normal haemoglobin.
Navega Therapeutics
The technique is being developed by a small company in the US, Navega Therapeutics. Their CEO, Ana Morena, explains that although the technology required is extremely complex, the principle is actually very simple:
“You can either activate or repress a gene of interest, without creating permanent changes. So we can repress the gene that’s known to cause sensitivity to pain.”
How would the treatment work in practice?
The treatment involves placing the gene-editing tool inside a harmless virus. These virus particles are then injected into a patient’s spine; similar to having an epidural. The particles will then enter neuron cells and once inside they release the editing tool which silences the pain gene.
What is the time-scale?
Navega wish to begin testing in human volunteers from 2021. If the technique proves to be medically safe and clinically effective, the necessary applications for approval for clinical use could be submitted within five years.
If (and it remains a big ‘if’) it proves both safe and effective, gene-editing has the potential to dramatically improve the quality of life for millions of people suffering chronic pain.
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