Why Does It Take So Long For Drugs To Enter Common Use?

Factual Questions
1

While browsing a few days ago I came across a small article about an anti-cancer compound called HAMLET (Human Alpha-lactalbumin Made LEthal to Tumours). It’s derived from a compound in human breast milk, is hypothesised to kill cancer cells in several different ways, has virtually no side-effects and, best of all, it shows a very high selectivity, meaning it picks off cancer cells and leaves healthy ones alone.

HAMLET has been through numerous and animal trials and, while I’ve not read all of them, has performed wonderfully in every study I’ve managed to dig up. It caused tumor shrinkage in 100% of patients in a double blind study of its efficacy on Papilloma lesions. It increased survival time in rat studies of Glioblastoma (one of the most lethal and hard to treat malignancies), and a recent clinical study of HAMLET in advanced bladder cancer showed tumour shrinkage without any damage to healthy cells. In vitro studies have shown preliminary success in HAMLET treatment of carcinomas of the lung, throat, kidney, colon, prostate, and ovaries, as well as melanomas, and leukemias.

But here’s my question. HAMLET is not a new substance. We’ve known about it for over 20 years. Indeed, the Papilloma study was done in 2004, as was the glioblastoma study. A recent interview with a lead researcher (name escapes me) stated that “optimistically” HAMLET will be ready within the next five to ten years.

My question is basically “Why”? Why is it that, when we have no shortage of toxic cancer treatments which are only proven to extend survival by a few months, why have scientists basically been sitting on what sounds to me like an absolute wonder drug, that’s passed every hurdle it’s faced, that’s proven to be safe, and which, in the case of glioblastoma patients especially, would be used on patients with very little to lose. You’d think every pharmaceutical company on earth would just be throwing boat after boat of money at this thing, yet until this week I can find no mention of it outside the archives of pubmed. Maybe I don’t know where to look, but I can find literally NOTHING negative about this stuff.

I know it’s asking a lot to expect anyone here to know about the progress of this specific drug, but it seems the pharmaceutical industry always moves at a glacial pace. Speaking more generally now, what kind of impediments do drug manufacturers face which present such colossal delays in bringing their drugs to market?

2

Username directly answers question.

Also, drug name answers question:
To be, or not to be: that is the question:
Whether 'tis nobler in the mind to suffer
The slings and arrows of outrageous fortune,
Or to take arms against a sea of troubles,
And by opposing end them. To die: to sleep;
No more; and by a sleep to say we end
The heart-ache, and the thousand natural shocks
That flesh is heir to, 'tis a consummation
Devoutly to be wish’d.

3

More seriously, after a bit of Googling it looks to me as though this stuff is not really a drug at all. It kills cancer cells in vitro (i.e., outside the body) but, as essentially a protein, it would be extremely difficult if not impossible to administer internally (where most cancers live). If you took it orally it would just get digested long before it got to the cancer; if you injected it one wold expect a strong immune reaction, and very likely death from anaphylactic shock. This is not just something yo could pop as a pill or as a shot. So far as I can tell, research is continuing into how it could be used therapeutically, but it is not going to be easy

4

this reads more of spam for product than as a question - way too many keywords and phrases in there.

5

I don’t think it is a product, and Doctor_Why_Bother has been around since June.

6

How long he’s been here is irrelevant to the comment.

Still reads like spam for the it - it is apparently a ‘real thing’ - His question is simple enough without talking about how great ‘hamlet’ is.
It takes time for meds to get approved - there’s good reason for the process.

7

If my question reads like spam for the product, it’s only because everything I’ve read about it suggests it’s amazing. For instance, while I’m well aware it takes time for meds to get approved, and that there are good reasons for this, one of those reasons is typically that it takes a long time to ascertain whether the drug is safe. But we’ve knows for years that HAMLET has an excellent safety profile. What’s more, it will add ten inches to manhood and sexxxy ladies from RUSSIA will love you very big…

Sorry. Not sure what happened there. Anyway, moving on.

It’s passed in vivo tests as well. Here’s one they did on 9 bladder cancer patients. 8 of the 9 saw dramatic tumour shrinkage:

The thing I don’t get is that I’ve seen stories in the press about drugs with a far lower success rate than 89% being touted as “miracle cures”, but I’ve heard zip about this. Even if it’s nowhere near as good as the study suggests, even if it only buys you a month, why not use it anyway? In patients with terminal bladder cancer, what is there to really lose?

That sounds interesting. I think in the bladder trial anove they injected it directly int the tumour. Obviously, that wouldn’t work for everybody. I’m pretty clueless about most of this stuff. Would you mind elaborating on how it could give people such a bad reaction? Cheers.

8

P.S. - Cheers for the vote of confidence. Next time you want easy access to cheap
Pharmaceuticals from Asia, just gimme a shout and and I’ll hook you up.

But only if you forward this to ten friends :slight_smile:

9

There’s your problem. It’s a natural substance, and therefore cannot be patented. This greatly lowers the profit potential, which is all that the drug manufacturers care about.

10

The point is that, as far as I can see, it would be likely to provoke an immune reaction if it got into the bloodstream. Most normal drugs, you get them into the bloodstream one way or another, and that carries them to wherever they need to be. I do not think that will work in this case. The in vivo trials you mention all (from my fairly hasty skimming) seem to have been on essentially external tumors, and even the bladder is external in the relevant sense, that is, you can get to tumor growing in it without breaking the skin or entering the blood. Most tumors, are much harder to get at.

Injecting directly into the tumor does not necessarily solve your problem. If the tumor has a blood supply, then stuff you inject into it is likely to get into the general blood system, and provoke that nasty immune response. If it does not, well, it isn’t a very nasty tumor anyway, it hasn’t metastasized and can probably be easily removed with surgery.

I am not saying there is necessarily no possible way around these problems, but I would not expect it to be easy, and I am not surprised that it is taking time to figure it all out.

And, of course, they may just be unable to make up their minds what to do about HAMLET’s problems. :slight_smile:

I doubt that is the issue. They have patented natural products before, even people’s genes. They can also patent ways of isolating and purifying natural products, or making them in quantity with biotech. Anyway, from what I can make out this is a modified natural product.

11

Here is a list of one class of “natural” proteins currently being manufactured and sold by drug companies.

12

Actually, it mentions tumor shrinkage or change in character of tumor (whatever that means). The abstract does not mention what type of bladder tumor was involved (invasive or non-invasive), or indicate that surgery did not have to take place to remove it.

We already have a well-defined and useful drug for bladder cancer that is instilled into the bladder (BCG) which has marked anti-tumor effects and is used mostly to prevent tumor recurrence. It is also a “natural” product (a form of inactivated tuberculosis) which is thought to work by stimulating an immune response that whacks out tumor cells.

There are many, many drugs (“natural” or “unnatural”) that look very good in cell culture or limited case studies, but prove ineffective and/or toxic on closer examination. I am also highly skeptical of any drug that claims to be wildly effective against cancer but has no side effects. :dubious::dubious::dubious:

13

So - your real question is not “why does it take so long for drugs (generic) to be in common use” but “Why isn’t this wonder drug ‘hamlet’ on the shelf and in everybody’s cereal”.

Which is a very different question -

14

Because we know that it probably works, but we don’t really know how it works. The FDA generally doesn’t approve NDAs unless the underlying mechanism is explained.

15

Well, I’m interested in both really. Apologies if that wasn’t clear.

Good answers though guys, so thanks for that.