Comment by torben-friis
9 months ago
100% response, zero side effects?
This sounds like world changing news. Can anyone with domain expertise explain the catch, if any?
9 months ago
100% response, zero side effects?
This sounds like world changing news. Can anyone with domain expertise explain the catch, if any?
The catch is that there are thousands of promising therapies in animal models/pre-human testing. A very very tiny fraction of them will ever make it to market for a variety of both good and not-good reasons.
What's the difference between a good and not-good reason to not go to market?
Prevalence of the disease - if it only impacts 1/100 million, going to be hard to ever find sufficient patient population to test and recoup your investment.
Existing quality of treatments - if there are already efficacious drugs on the market - how sure are you that this new therapy will be best in class? Only being as good as the status quo is not an ideal competitive position. Conversely, if there is an unmet need because a disease is so lethal/debilitating, regulatory agencies can give latitude in approvals.
Likelihood patient compliance - if it is the most effective drug in the world, but requires intravenous infusion six times a day - nobody is going to adhere to that. GLP drugs are effective, but there is a needle-phobia that is preventing patients getting on board with the idea. Which is why there is an arms race for the first company to develop an oral version.
Toxicity - all chemicals are poisonous. Yet some have a lower therapeutic window than others. If you drug does what it should, but if you take 2x as much and it gives you a heart arrhythmia that is going to be a tough approval for anything but the most deadly conditions.
8 replies →
Good reasons:
* Most drug candidates just don't work
* Even among the drug candidates that do, figuring how to safely deliver them to their target is very hard (looks similar to "just doesn't work")
Bad reasons:
* It's too expensive to prove that a drug works
* It's too difficult to differentiate the patients for whom a drug works and the patients for whom it does not
* It is very hard to predict recruitment and to actually recruit patients for clinical trials
* There aren't enough people with the disorder who are also rich enough to afford treatment to justify development
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A lot of potential treatments are too easily available and can't be patented. If a big pharma company can't make massive profit from it, they won't bother bringing it to market. Consider that a not-good reason.
Other treatments may eventually prove to have too many serious negative side effects. That's a good reason to abandon them.
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In the early 2010s I had a couple friends working at a biotech startup with a moonshot cancer cure. They had amazing results in animal testing, and had raised just enough funding to do a first human trial on a terminal patient.
That patient was hit and killed by a car two days after finishing their treatment.
Along with the CEO's stubborn refusal to give up any more equity in the company, they went under, and the world will never know if that treatment worked.
From what I saw, there are very few experimental therapies that claim 100% efficacy, no side effect, no patent on complex engineering process to produce the drug.
I agree with GP that it is very notable.
I mean if it works on humans, which is not a stretch, colorectal cancer is done. It's huge.
Yes there are few that claim 100% efficacy and no side effect at this stage, but there are far, far, far fewer who make it to human availability.
I wouldn't describe it working in humans as "a stretch" per se. I'm not identifying a specific reason it shouldn't work in humans. I'm just saying that's true of thousands and thousands of really great looking treatments (per year!) that, nonetheless, end up not working in humans, or not being convincing enough to even warrant putting them in humans once.
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Certainly good news for mice.
I wonder if anyone has tried to engineer a mouse that lives forever by applying all these life enhancing mouse therapies at once.
Yes, Mathuselah Mouse Price
Very small sample size and only in mice. Most experimental treatments don't make it to the clinic, and this could be another in a long list of exciting early results that don't lead to anything. However... These results seem too good to be true, but the paper actually seems pretty good. There have been other attempts to use anaerobic bacteria to treat cancer (as solid tumors tend to be hypoxic environments), this is the first I've seen that interacts with PD-1/PD-L1 pathway. This is the most promising cancer treatment I've seen in quite a while. Maybe we can be cautiously optimistic!
It looks like they screened 45 strains of bacteria to find 9 that passed their safety tests, and then only one of those had a 100-percent response. The sample size is also small: 5/5 sounds a lot less impressive than 100%. I'd expect the true response rate to be substantially lower ("winner's curse").
The bar for an acceptable side effect profile in an FDA-approved drug would also be a lot higher than "five genetically near-identical mice did not show evidence of pathology in a single study."
I'm not saying this work is bad (skimming, it seems fine for what it is, though haven't read in detail), but it's quite preliminary if we're talking about developing a medical treatment that could eventually be deployed in humans. There's a reason it ended up in a mid-tier microbiome journal.
I hesitate to link directly to a single podcast dealer, but if you search for Dave Ricks, CEO of Eli Lilly's podcast with Stripe brothers, there's some alpha in there.
A few years ago we also had 100% effective medication with zero side effects. Now birth rates are down, mortality up, and young people misteriosly die from cancer and hearth attacks.