Speaking this week at the EmTech conference in Cambridge, Massachusetts, Editas CEO Katrine Bosley said the company hopes to start a clinical trial in 2017 to treat a rare form of blindness using CRISPR, a groundbreaking gene-editing technology.
…The condition Editas is targeting affects only about 600 people in the U.S., says Jean Bennet, director of advanced retinal and ocular therapeutics at the University of Pennsylvania’s medical school.
I don’t think that the FDA is prepared for what is coming.
For one thing, this trial is probably a pilot for the overall treatment technology- mayne because this is a simple application. So if the FDA treats it as if this ultra-rare blindness remedy might be itself unleashed onto the market, there is no other way to say it other than they’re doin it wrong.
Well, 20 years ago they were green-lighting trials for gene therapy for some pretty rare diseases. It doesn’t make sense to imagine they were doing that knowing that they’d never approve it for medical use without clinical trials of infeasible size, scope, and duration.
Gene therapy is a pretty old topic. The specific mutation for Cystic Fibrosis – which causes people with the disease all kinds of problems to include a typical lifespan under 30 years – was discovered back in 1989. It’s pretty rare – only 3 births in 10K or so, and that’s without genetic screening and selective reduction which is recently more common. Even in a country as large as the US, only a thousand babies are born with the condition in a year.
But it wasn’t long after the gene’s discovery, and still more than 20 years ago, that people tried to see if they could do virus-mediated ‘gene-therapy’ to either correct the mutation or knock it out with a reverse code.
Well, that strategy didn’t work out with the old tools, and actually produced some pretty awful results. That’s probably because we were trying to get certain viruses to do things they weren’t designed to do, on full-grown adults, and hoping in the meantime they wouldn’t do the harmful things they were designed to do, or that the body’s immune system wouldn’t react against it.
However, gene editing – especially at the embryonic stage – has a much better chance of working out with the new CRISPR-generation of tools. We stole these from nature in what may turn out to be the most impactful discovery of all time after fire (in the judgment of our descendants) and which evolved as a sophisticated immunological system for primitive cellular organisms over billions of years, which is something like a hundred trillion generations, and in a way that was perfected in those circumstances to do exactly what we are doing with it. Thanks nature! “Couldn’t have done it without you,” is probably a literal statement in this circumstance.