Gene-editing tool CRISPR to be used to treat humans for the first time
23 June 2016
Internet billionaire Napster founder Sean Parker, who was the first president of Facebook, has received federal backing to run the world's first-ever CRISPR/Cas9 trial, which could change the way many diseases, especially cancer, are treated.
Though CRISPR had been much talked about, in the media, it had not yet entered the clinic.
However, its preclinical activity had caused much hype given its gene-editing capabilities, with its core tech centred on a simple method for reengineering DNA - which may be expected to have profound consequences in treating a number of diseases.
A National Institutes of Health (NIH) panel yesterday ruled unanimously (but with one abstention) to allow the first CRISPR candidate run. Though this is only the first step, the FDA as also the medical centres where it would run would still need to clear the study before it actually started recruiting and testing patients.
If the trial were to gain regulatory approval, it would be run by a group of University of Pennsylvania researchers and funded by the Parker Institute.
According to Dr Carl June, one of the major innovators in T cell biology, who spoke at the Forbes Philanthropy Summit earlier this month, the idea was to make T cells, a type of white blood cell, ''better than nature made them.''
The designer immune cell technique had earlier been tested in cancer patients. In the technique doctors take a sample of patients' blood and spin out the white blood cells called T cells.
In previous trials, researchers used a virus to insert a section of DNA that searched for cancerous mutations, before infusing the lab-made blood cells back into patients. According to the scientists involved using CRISPR may make the treatment more effective, enabling them target cancers more precisely and bolster the T cells' capabilities and reproduction rate.
"It's an important new approach," Recombinant DNA Advisory Committee (RAC) clinical oncologist Michael Atkins said of the trial, The Verge reported. "We're going to learn a lot from this, and hopefully it will form the basis of new types of therapy," he added.