A company I consulted for was considering licensing a cell therapy from a large, well regarded US research hospital. When we looked at the manufacturing data it became apparent that the hospital really had no idea how to characterize the cells they modified and gave to patients. It was basically take cells from a patient, purify them and grow them up in culture, add a few nucleic acids, then readminister. No testing to see if the nucleic acids got into the cells, how many cells got them, whether the cells changed unacceptably during culture, haphazard testing to see if they modified the cell's surface protein levels, no specs as to what level of protein expression were acceptable, etc. They did do safety testing so it isn't likely that the cells would be harmful, but its very likely they would have no effect. There was no malicious intent on the part of the researchers or hospital, they just didn't know how to do commercial quality QA/QC on cell therapies. At the time, no one did. Even so there was some stuff they should have known to do but didnt do
This was ~5 years ago so things QA/QC for cell therapy has advanced since then, but its still evolving. Would be interested to know how these hospitals handle this
That’s very interesting to read, thanks for sharing your professional perspective.
I’m guessing we need to find the sweet spot on the continuum between dangerously slow regulatory bureaucracy and dangerously regulation free snake oil?
I would hope that the selling of medical hope includes some control measures to help shift decision making from the emotional to the logical.
If it’s immediate and transactional, I fear emotion will trump logic based on human behaviour under stress.
I’m a big supporter of experimental therapies, as long as the process is robust and logical.
What the article title didn't stress is the word "a few selected".
Just to re-iterate what it says in the article, the intent was that this draft policy change to re-allow (currently banned since 3 years ago) "select elite hospitals in China to sell experimental therapies would give people with terminal illnesses faster access to potentially effective treatments."
First, this proposed lifted ban is only for select elite hospitals NOT all hospitals.
Second, for terminally illness, experimental therapies are indeed used as a last resort treatment. Just like many other countries. And many anecdotes we've heard. E.g. Was virus treatment FDA approved? No! Did the doctors have any other way to save this patient on a life-and-death situation? Not really. [1]
Note, a "few selected" == "roughly 1,400 elite hospitals"
From what my friends who do business with, or are physicians from, China tell me, medicine in China can often be a pretty brutal assembly line, complete with bribes being passed, literally, under the table.
Having worked in a number of "top" US hospitals, I would be interested in what the 1400th hospital looks like in this scenario.
Cures can't be found without testing and it's clear that some people just don't have 10-20 years to wait for extensive tests and for investors willing to pay for risky investments/treatments.
I just hope the patients are monitored/documented well so that the results can be improved/replicated.
Indeed. My Dad has terminal cancer and all conventional treatments are at best just (slightly) delaying the inevitable, while having a negative impact on his quality of life. As a family we're always investigating experimental treatments and would jump at the chance of something like this.
Way back in 2008 [1] there was a guy on reddit who said he went to China and got stem cell therapy and went from being a paraplegic to not being one. Everyone in the thread refused to believe him. They did this recently in an experimental procedure in America and suddenly, it's reality.
There's something really deep in American culture that says that all medical breakthroughs must occur in the United States or they're frauds.
It's the same mindset that leads to people repeatedly blaming the pilots in the recent 737 MAX crashes in spite of all of the available evidence. They were foreign, so they must have been inadequately trained and experienced compared to American pilots, right?
I don't think it's just Americans, and in the case you cite, people were completely justified in their skepticism. The reddit account was apparently created for that single AMA post and had no activity before or since. The company he allegedly went to, Beike Biotech, was notorious at the time for using various forms of astroturf and deceptive advertising to promote its unproven stem cell treatments by testimonial, a problem that is rife in stem cell marketing. The Chinese government ended up cracking down on domestic cell therapy clinics in 2015 after years of ineffective enfprcement measures, which would be a very unlikely thing to do if it had faith that local hopsitals had developed an effective treatment for spinal cord injury.
They've been around since 2005 and have a large network of clinics. You'd think they would have got shut down by now if they were a scam like Theranos.
My take was its hard to find proteins unique to the cancerous target that isnt also present in healthy tissue. It even mentions a patient being treated where the target proteins where also present at lower levels in the patients brain. So I guess the programmed T-cells run amuck typically in the course of treatment.
I hope that intense competition to control the most effective new cancer treatments accelerates development. This is once the treatments become mainstrain, not really addressing the article.
Even US-approved CAR T-cell is currently so toxic with a high chance of severe adverse effects that it should only be used as a last resort treatment. From a linked article:
“Right now it is heroic medicine,” he says — a grueling treatment deployed only in people for whom all else has failed.
“Patients are taken sometimes to within an inch of their lives.”
I feel like China's health care system is currently in a strange/incapable position to decide when all else has truly failed, not when plenty of basic medication is impossible to get, or not covered under any insurance.
I don't agree with this but it seems like a functional method to fund research. Are we seeing breakthroughs from other countries via the traditional private funding/patent system?
1. "But since the ban [on selling untested treatments], hospitals have found it difficult to recruit participants for clinical trials"
That sounds like a lack of faith in the system, that charging for untested treatments again isn't going to solve.
2. How does a self described Communist country ideologically defend charging for hospital treatment? Not getting in to the rights or wrongs of Communism or free health care, this just seems contradictory.
They know the consequences of not trying any risky/experimental treatment: death.
I know people willing to become guinea pigs for non-life threatening conditions (i.e back pain).
It's clear that the current approach to develop new treatments/drugs that requires billions and years or even decades of tests is not fast enough for many people.
With the amount of lawsuits in the US for people that are willingly harming themselves or abusing drugs: Cigarette companies still getting sued for smokers dying of cancer..even though it's been known for 50 years that it causes cancer and Big Pharma getting sued for the Opiod crises means that this will never happen here until we have major tort reform and an assurance that a company can't be sued in any court if someone dies as a result of taking untested treatments.
"It's clear that the current approach to develop new treatments/drugs that requires billions and years or even decades of tests is not fast enough for many people"
It takes years of testing because the risk if health or death is too great. It's foolish to think that allowing anyone to be a human guinea pig for untested/unfinished treatments will result in any less deaths or suffering from a terminal illness.
Imagine that you have a 99.99 chance of death. There is treatment that appears to work in 50% of people, but it's early days, it's only been tried on 10 people. What do you do? Do you want a bear certain probability of death or a 50% chance of a cure? I think it is entirely proper to allow people with the money to pay for it, to take that gamble. It's their life, not ours. It's their money, not ours. Sure, we need to be sure that the correct data is presented to the client, they mustn't be mis-sold. But otherwise I have no issue with people being free to take a gamble. I would absolutely furious if this chance for life was withheld from me. And so would you be.
Intelligent people understand what a trade-off is. Testing experimental and potential risky treatments on humans does not make it less scientific.
These people will die anyway and most of them prefer to die fighting or at least knowing that someone might benefit(i.e their children) if a treatment is found.
It is both the human and scientific thing to do for them.
I feel like you should ask the people themselves what they want. These experimental drugs may save their lives and will help with future medicinal breakthroughs. Assisted suicide is another topic and imo should be allowed, but the two aren't mutually exclusive when it comes to legality.
Well what I mean is that if things go horribly wrong, the experimental patient should have the right to pull their own plug. It should be in place ahead of time considering the unknowns. Would you really want to do a treatment if it could leave you in a forced miserable existence for the next 40 years without any escape?
That situation is an extreme hypothetical and I'd probably take the experimental treatment along with painkillers/cannabis if there's any miserable side-effects. It depends on the person and their tolerance, I'd assume most people would prefer to live and be with their loved-ones. Humans are pretty resilient and they adapt. Nothing is stopping a person from committing suicide on their own if they aren't able to cope in the future.
Let’s say you have a life-threatening illness. all legal treatments have failed. there is a potentially life-saving treatment that has passed FDA safety trials but is still in efficacy trials and has not been approved for any legal use. You’re fine with just throwing up your hands and dying, because you’re not allowed to make that decision?
Let's say I'm a startup with a new idea for a treatment. I don't really have the funding to actually go through the proper testing and I decide to use humans to test my new ideas. All of the patients in my trial die a horrible death (much worse than going through cancer) in the process. In fact, there was no chance it would have ever worked.
Are you okay with these consequences? Regulations are in place for a reason. While I do think we need to loosen some of the regulations in the US, having no regulations, like what is describe in this article, will bring forth companies that have no business selling drug treatments and literally getting away with murder.
You set up a strawman and destroy it without answering any of my questions; this seems like arguing in bad faith.
Endeavoring to be more constructive and respond in good faith: No, I am not OK with those consequences, and the ludicrous strawman argument you raise bears zero relation to the entirely realistic hypothetical that I described.
I hope you are never stricken with a life-threatening illness that cannot be treated with our artificially limited set of legally available interventions.
"You set up a strawman and destroy it without answering any of my questions; this seems like arguing in bad faith."
I gave you a scenario that is very possible without some sort of regulations. We have regulations in place because experimental drugs can kill people without proper oversight.
China is not known for having a good track record in terms of regulations or caring about the life of a human being. Many people still won't buy baby formula to this day because of the lead scare a few years back.
"I hope you are never stricken with a life-threatening illness that cannot be treated with our artificially limited set of legally available interventions."
You are too emotional to have an actual discussion about this.
Yes. Your will be facing multiple lawsuit and go down if it found out that you knew that there was no chance it would have ever work. Maybe facing criminal charge too.
It might not work because it hasn't been completely tested. The human is the test and people may die during the testing process.
Why should anyone go to jail over this? The startup was just testing it out and the person was willing to take the risk. They might not have even known that it would kill the patient.
When a patient dies, the family will most likely try to sue the company that created the treatment. What company would ever take a risk like this?
The bright side is that the human testers in China will lead to better care in the rest of the world.
What's to stop a hospital charging 1000s for a treatment they have no reason to think will work.
"Hey lets do a study to see if cheese prevents cancer. We'll do a thorough 20 year study involving 10,000 participants each paying $100,000pa. Obviously double blind to keep it fair." A Hospital administrator.
You're offering an emotional argument, that to me is even more reason not to have a profit motive like this. People will pay any price, for any thin sliver of hope. We should be making sure that isn't taken advantage of.
Would you please stop breaking the site guidelines by adding generic flamebait and taking HN threads into nationalistic battle? I don't want to ban you, but we've had to ask you this repeatedly.
This was ~5 years ago so things QA/QC for cell therapy has advanced since then, but its still evolving. Would be interested to know how these hospitals handle this