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Medicine Science

FDA Halts One of the First Human CRISPR Studies Before it Begins (technologyreview.com) 109

Posted by msmash from the baby-steps dept.
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.
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FDA Halts One of the First Human CRISPR Studies Before it Begins More

FDA Halts One of the First Human CRISPR Studies Before it Begins

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  • Measure twice, cut once (Score:4, Insightful)

    by SlaveToTheGrind ( 546262 ) on Thursday May 31, 2018 @09:55AM (#56705390)

    I'm generally not big on layers of bureaucratic red tape, but for an emergent technology like this it seems absolutely appropriate to take an extra cautious approach for the moment.

    • Re: (Score:2, Informative)

      by bill_mcgonigle ( 4333 ) *

      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.

      • Why do you think you're at all qualified to determine the level of caution that is prudent?

        Setting aside the fact that I didn't say I personally was qualified, are you suggesting that the manufacturer standing to profit from the therapy is in a better position to determine the prudent level of caution? Usually we prefer not to let the fox totally guard the henhouse.

        The FDA's over abundance of caution has caused, to date, 15 million unnecessary deaths

        If you'd prefer to nuke or effectively neuter the FDA, then call your congresscritter and advocate for that. Good luck.

      • people should be free to choose what is done with their own bodies.

        But these people aren't really free to choose. They have a "gun" to their head in the form of a sickness, and they're being told to do whatever it takes at *any* cost to cure it. They're ripe for being taken advantage of by well-meaning scientists hoping to solve their problem

        There absolutely needs to be someone to step in and say "Hold up, let's make sure this is a smart approach first." You could tell these people that sucking on a tube of U-238 will cure them and they'd give it a try. Hell, people will g

      • I don't know if you actually know you're doing it, but this is a classical fascist tactic. Immediately attack the questioner that they are incompetent to assess the issue. State that only specialists can do it, and the involvement of the interested public is inappropriate. This is of course used to protect a medical technique that may combine extreme risk with low efficacy. It will make someone a ton of money, potentially at the cost of some number of poor people being killed or having their health made wor

      • 15 million unnecessary deaths

        Serious cite needed on this. I cant find *any* reference to this claim online, even the kook anti-govt sites. In fact the only page on the net with this claim, according to google, is this very thread.

        So give us a cite for that, or its reasonable to conclude, you just made that up.

  • I think they should allow Human trials on terminal patients

  • Very Hard to Measure Safety (Score:5, Informative)

    by Artagel ( 114272 ) on Thursday May 31, 2018 @10:16AM (#56705518) Homepage

    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... [acs.org]

    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... [bionews.org.uk]

  • IF the FDA uses its usual deliberate speed, we'll have CRISPR treatments, oh, after I die from something that could have been treated with CRISPR.

    If they have questions, ASK THEM. QUICKLY. Get answers, get out of the way.

    • The aim is to keep you from dying faster or living what time you have left in a significantly worst condition because the "treatment" is dangerous and ineffective. Just ask all the idiots that went to countries with lax oversight to get themselves injected with the last "miracle cure," stem-cells
  • The FDA is a massively expensive formality, an obstacle of saving lives with allegations of corruption all over the place.

    If the FDA told me to eat undercooked meat would I do it. No way !

    Did the FDA write The Jungle expose that uncovered the unsanitary practices of food handling? No, it was a private citizen, Upton Sinclair.

    How many medical/pharmeceutical breakthroughs happen in over regulated Europe? None.

    Does it do anything? Maybe, but better off without it.

  • So the article is just about some bureaucratic mumbojumbo like _always_ when such trials start.

    I guess form 643a got stuck in the mail and now we get a crappy story here.
    Tell us when they cured the guys but not every step to get the authorization to begin.

  • The more interesting part is the following, from a CRISPR press release: The planned initiation of a Phase 1/2 trial of CTX001 in Europe in adult patients with transfusion dependent Beta-thalassemia is unchanged, and the companies expect to initiate the trial in the second half of 2018. Therefore, ex vivo CRISPR gene-edited therapies are actually about to go into trails. The SCD treatment has hit a very small bump in the road, which is expected and less of a story.

  • The more coupling there is between corporations and the federal agencies that are supposed to regulate them, the less you need a tinfoil hat to wonder if influential companies might use the FDA to obstruct research that could result in products that threaten their business. We live in an age of medical maintenance, not cures. How long has it been since the last eradication of a major disease? I have to wonder if we'll ever see cures for diseases such as diabetes that support multi-billion dollar maintenance

  • So what are the reasons?
    What about "Right to try"?

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