How? I mean the way every drug for example is tested for safety, they give it to people (healthy volunteers) in escalating doses, and watch them for adverse effects.
Rarely, it happens that a drug that was expected to be safe based on animal testing turns out to be dangerous in humans, and healthy volunteers die. It’s a tragedy, it happens rarely, but it happens, and safety trials are still the best way to start the process of clinical trials for new drugs.
For some treatments, such as gene therapy, it’s not ethical to ask a volunteer to just try it to see if it’s safe. So they look for sick people, who are consenting to risking a new treatment with unproven safety, because their other choice is to not be treated.
This is how it’s always done. I wonder what you mean when you say the treatment should be tested for safety before giving it to patients.
Edit: this was a personalized experimental treatment developed specifically for this girl, on the parents’ initiative. The clinical team did test for antibodies against the virus before injecting the AAV into the csf. Lots of shady and wrong things on the research side, I’m not protecting them, just disagreeing with the previous comment that this tragedy would have been prevented by a simple test.
It has to be the exact virus or it’s irrelevant, and it kind of can’t be done.
The virus has to get to the brain and spread around and “infect” cells in order to edit the DNA. It’s why it had trillions of the virus. It needs a slow and unprepared immune response so it can do the editing.
If they did a small dose test, you just vaccinated her against the virus. Her immune system would more quickly go after and destroy the virus. They expected her to get sick. They didn’t expect it to get that bad. She needed more powerful immunosuppressants beforehand.
Neither of those things is what the patient received. Do you mean they should test the vector without the payload on the patient? If the adverse reaction is to the vector, the patient would then experience the negative effects without a chance of getting any benefits. So why not give the real thing then?
If you mean a control vector should be tested on healthy volunteers, idk, maybe, but it’s hard to come up with a relevant control that certainly doesn’t do anything to dna.
Wtf. If she died from the dna editing, doing the test you mention wouldn’t have prevented it. If she died from the formulation, she would have died from the test you talk about. I don’t see a scenario where a test with a control virus makes a difference.
She died in kidney failure and thrombotic microangiopathy, not an allergic reaction. They did test for antibodies against the virus. The injection was directly into the cerebrospinal fluid, not exactly the same exposure you get with a skin allergy test.
How? I mean the way every drug for example is tested for safety, they give it to people (healthy volunteers) in escalating doses, and watch them for adverse effects.
Rarely, it happens that a drug that was expected to be safe based on animal testing turns out to be dangerous in humans, and healthy volunteers die. It’s a tragedy, it happens rarely, but it happens, and safety trials are still the best way to start the process of clinical trials for new drugs. For some treatments, such as gene therapy, it’s not ethical to ask a volunteer to just try it to see if it’s safe. So they look for sick people, who are consenting to risking a new treatment with unproven safety, because their other choice is to not be treated.
This is how it’s always done. I wonder what you mean when you say the treatment should be tested for safety before giving it to patients.
Edit: this was a personalized experimental treatment developed specifically for this girl, on the parents’ initiative. The clinical team did test for antibodies against the virus before injecting the AAV into the csf. Lots of shady and wrong things on the research side, I’m not protecting them, just disagreeing with the previous comment that this tragedy would have been prevented by a simple test.
A virus that doesn’t actually do anything is how. Or a test injection that doesn’t have any virus at all, if the reaction was to something else in it.
It has to be the exact virus or it’s irrelevant, and it kind of can’t be done.
The virus has to get to the brain and spread around and “infect” cells in order to edit the DNA. It’s why it had trillions of the virus. It needs a slow and unprepared immune response so it can do the editing.
If they did a small dose test, you just vaccinated her against the virus. Her immune system would more quickly go after and destroy the virus. They expected her to get sick. They didn’t expect it to get that bad. She needed more powerful immunosuppressants beforehand.
Neither of those things is what the patient received. Do you mean they should test the vector without the payload on the patient? If the adverse reaction is to the vector, the patient would then experience the negative effects without a chance of getting any benefits. So why not give the real thing then? If you mean a control vector should be tested on healthy volunteers, idk, maybe, but it’s hard to come up with a relevant control that certainly doesn’t do anything to dna.
Yeah but she wouldn’t be fucking dead.
Wtf. If she died from the dna editing, doing the test you mention wouldn’t have prevented it. If she died from the formulation, she would have died from the test you talk about. I don’t see a scenario where a test with a control virus makes a difference.
An allergy test uses small amounts to test for a reaction.
She died in kidney failure and thrombotic microangiopathy, not an allergic reaction. They did test for antibodies against the virus. The injection was directly into the cerebrospinal fluid, not exactly the same exposure you get with a skin allergy test.