As you have already noticed, I often criticize different medicines that are imposed on us more and more every year. Of course, I understand what big business is and that the top management of pharmaceutical companies need to maintain their business jets to fly to Ibiza 😁 But what they don't understand is the people who need treatment and the doctors who prescribe it.
This post is about a drug that gave false hope to many patients and was the most expensive drug in the world (~$2 million - 1 injection). It's about Zolgensma (Onasemnogen abeparvovec).
I don't know who comes up with such complicated names for these drugs, but as it turns out, they are also tested by NOT very smart guys (Although maybe it's the other way around. 😉)
In short, there is a group of incurable diseases under the common name - spinal muscular atrophy. So, the big businessmen from the big headlights commissioned scientists to develop a drug that treats this case, using new techniques to modify the patient's genes (ala viral vector as in the vaccine - "clot generator" - AstraZeneca).
The scientists thought, found faulty genes that needed to be corrected, and rolled out an experimental drug. Then they started to test it on mice, the control group of sick mice was injected with a viral vector of green fluorescent protein, and the experimental group was injected with the drug itself. Well, the logic is simple, if sick mice with the drug will survive sick mice without the drug, then it's a done deal. And according to the first statements of the scientists they survived and lived a full life 🙂In 2019, the FDA approved Zolgensma, i.e. they confirmed that the drug is safe and effective.
The drug was put on the market and started to be used by various medical institutions. The state and various foundations paid the pharmaceutical company-manufacturer for supplying the drug to their patients. Relatives of thousands of patients saw it as a panacea, although, in fact, the drug does not make the patients healthy, which personally gives me a wild feeling of inexpediency of such therapy 🙂After all, why prolong a person's agony and pay 2 million for it? Sounds cruel, but that's purely my opinion 😉
In short, big pharma started to make a profit; And it went on until patients started dying after injections. Those patients were two children from russia and kazakhstan. They were the first deaths from the drug. Of course, it is written that the causes of death there are different, but I was in doubt and maybe my intuition did not fail this time:
Some interesting facts have started to surface this month 🙂A 2010 scientific publication describing experiments on mice was retracted. It turned out that during the experiments, the researchers inaccurately recorded the results of observations of mice, and the report included unverified data.
Somewhere the described lifespan of the mice differed from the real one by a couple days, and somewhere it was as much as 19 days 🙂 Personally, I am perplexed by this information. The company itself announced it and started firing responsible employees and conducting a secret(!) investigation.
The most interesting thing in this story is that the "gurus" from the FDA were not embarrassed by this. A decision was made not to recall the drug from the market, quoting "'experts'": "the inaccuracies found do NOT affect patient safety or the efficacy and quality of the product." Although, in fact, the safety and efficacy of this product has not yet been 100% established😁
Who believes them? I certainly don't 🙂 Now think about how much more of this possible(!) fake science there is in other new drugs ? 🙂
https://www.nature.com/articles/nbt.1610
