FDA Halts One of the First Human CRISPR Studies Before it Begins (technologyreview.com)

A trial planning to use the gene-editing tool CRISPR on sickle cell patients has been put on hold due to unspecified questions from US regulators. From a report: CRISPR Therapeutics, which is developing the therapy, sought approval from the US Food and Drug Administration in April to begin the study. The therapy involves extracting stem cells from a patient's bone marrow and editing them with CRISPR in the lab. Once infused back into the patient, the idea is that the edited cells would give rise to healthy red blood cells. But according to a statement on Wednesday from CRISPR Therapeutics, the FDA ordered the company not to proceed with its study until it answers questions about its CRISPR treatment.

Comments

Very Hard to Measure Safety

[Artagel]

One problem with these gene-editing treatments is that it is very hard to measure the safety of the treatment. It could be that the company tried to show how it would measure safety, but FDA wasn't satisfied with the process.

Chemical and Engineering News (probably behind a paywall) has an article about how companies are trying to come to a consensus on how to measure safety. https://cen.acs.org/pharmaceut...

A huge problem here is that DNA breaks all the time in our cells and gets repaired. That is the exact process CRISPR leverages to make its edits. So, how do you tell a natural break and mis-repair from a misdirected CRISPR edit. Not an easy thing to tell. FDA wants the applicant to show safety, not for someone else to show dangerousness. Proving a thing that is very difficult to measure in the first place is a great challenge, and may keep these treatments from advancing at FDA.

The Europeans have a similar issues with their beta-thalassemia trial. https://www.bionews.org.uk/pag...

Re:proper

[michelcolman]

Meanwhile Chinese researchers, not bothered by any of those morality concerns, carry on perfecting the technology. I wonder who's going to be first to market...

Re:Measure twice, cut once

[bill_mcgonigle]

Why do you think you're at all qualified to determine the level of caution that is prudent? The FDA's over abundance of caution has caused, to date, 15 million unnecessary deaths, and that's the middle estimate. They grossly over reacted to the thalidomide scare by causing one and a half Nazi Holocausts worth of death and suffering. Sickle cell anemia may not be immediately fatal, but it does cause suffering, and people should be free to choose what is done with their own bodies.