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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.
FDA once in a while does its job, amazing
I disagree, in this case I think using CRISPR on humans might be a legitimate use. I don't agree with using it to try and edit the human genome to make taller offspring, or blue eyes, or 15 inch ding-dongs; but to cure a genetic disease... I think that's wonderful. Using CRISPR to cure certain defined genetic diseases is a far cry from designer-babies.
This is an area that needs careful oversight but we shouldn't just dismiss a technology because we fear a slippery slope or because we think a beneficial technology is creepy.
"That's the way to do it" - Punch
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.
no you have improper understanding.
Medicinal use of CRISPR sequences is in clinical trial stage only, in China and USA.
It is not a proven or approved treatment anywhere on planet earth, nor will it be for a very long time
Having the FDA raise the proper questions is not "dimissing". You seem to imagine they're holding up a line of dying patients
That is true, and hard to accept.
CRISPR brings promises of curing diseases that are right now causing terrible suffering and early death to billions of people.
Everyone is in a hurry. Many people are desperate enough to submit to experimental treatments, because they are at the end of their rope. In such a situation, getting in front of such experimentation "feels" morally wrong.
The common tendency to confuse morality and legality makes people experience confusion when they see the guardians of food and drugs standing in the way of important medical breakthroughs.
OK, this is weird, but bear with me.
My dog has experienced a total loss of nasal turbinates, cause unknown, he is a rescue. Subsequent to this he was diagnosed with nasal angiofibromas, benign, but ultimately destructive of the nasal cavity and probably will end with infiltration of the cranial cavity and euthanasia.
Now the oncologist would very much like us to pursue treatment, focused radiation, and then monitor for recurrence, primarily because there is very little known about these conditions. Few dogs receive documented treatment, and so the vet industry doesn't really know how the treatments work, outcomes are indeterminate, and such.
All they want is for us to pay $8,000+ for treatment, with neither any assurance of success, nor any prognosis, and certainly no assurance that a relapse or recurrence won't come, and soon.
And he's the most lovely dog, but I would not pay this for a car repair with similar conditions - *might* fix it, far exceeds objective value, might happen again.
It seems as if this canine oncology practice would love for us to pay to advance the current understanding of this condition. But we were close to asking, if it's so interesting to them, could they perhaps discount the treatment? But nothing about the appointment led us to believe this was anything but a business decision. We've decided no.
We decided to not make our dog the test case. He's probably going to have a shortened but happy life, and we will not let him suffer unnecessarily, which is, to say, very little. He could have been put down in the shelter, so this is a win, and he's such a lovely dog.
While the lure of CRISPR therapy is undeniable, the risks are substantial. The FDA has good reason to demand substantial safeguards, information, and explanations. Look at some of the recent revelations regarding lab tests (Theranos), multiple drug trials ending in failure, fabricated results, etc. Gosh, it seems as if you can't readily trust the industry in general.
This will bump into the 'right to try' movement, but I'm inclined to accept the FDA's reluctance as reasonable. This is a big deal, not as simple as trying it to 'see what happens'. No, it is not.
deleting the extra space after periods so i can stay relevant, yeah.
Meanwhile Chinese researchers, not bothered by any of those morality concerns ...
China has moral concerns. They just reach a different conclusion. After weighing the potential benefits to millions of potential beneficiaries against the risk to a handful of patients (who are informed consenting adults), I don't think their conclusion is wrong.
The West is cautious about medical experimentation because of historical abuses such as the Tuskegee experiments, and Nazi experiments. The Chinese don't have the same cultural baggage.