Experimental drugs.

A recent article in New Scientist made me wonder what other people's thoughts on this and similar instances were.

I'll try and keep this generic, it has wider applications than just the specific case mentioned.

News spreads that a simple chemical (not patentable) has shown some promise in animal trials of curing or mediating a specific terminal condition. No drugs companies are prepared to run trials because the compound is not patentable. Some people get hold of the drug and try it anyway - mixed results some good, some negative and some with side effects. News spreads further.

Should you do this - in any case? in limited cases? never?

pros - it might work, you might get - longer life expectancy, relief, hope ...
cons - unknown side effects, dashed hopes, you make it harder for other people to know by not being part of a trial.

Should you volenteer for being part of a trial. What if it can't be organised quickly enough.

plenty of questions. Any thoughts? or related reading materials?

I suppose it depends on who is providing oversight for the trial, etc. Is the trial initiated by some larger institution with IRB involvement? Who are the "people" that are initiating use distinct from the drug companies? (I know you are trying to keep it sufficiently vague for the sake of conversation, but these are factors I would consider before consenting to my participation).

'the people' initiating use are patients/sufferers of the disease. Often convential medicinal approaches have not worked in their cases, or there is no treatment available. Any hope of any treatment often seems good enough "if it might work" to them.

Oversight of the trials is one of the questions - who will pay? If you are lucky you can get perseude some pHd students to organise something.....

Isn't this how a lot of hoo-ha alternative medicine gets started, like laetrile (vitamin B17)? Is it really true that conventional pharmaceutical researchers will not try out drugs that aren't patentable? Can't they still patent the process or some other part?

"Is it really true that conventional pharmaceutical researchers will not try out drugs that aren't patentable?"

Yes it is true - the individual researchers may want to, but considering the vast expense of full trials for a registered drug, pharmaceutical companies do not. The only way they can re-coup trial costs is to have an exclusive market for x years, before the prices are undercut by competing generic products. So they at least claim.

"Can't they still patent the process or some other part?" Depends. If tis a natural extract they could patent the extraction method, or modify the compound slightly, but for many possabilities this is also not an option - or at least not as profitabel an option as progressing with their own unique drugs.

I hadn't thought about the alternative medicine market, but you are right the issue is substantially the same.

So what are the 'ethics' of 'alternative' medicine in all its forms.

Message 5: reading_fox -- So what are the 'ethics' of 'alternative' medicine in all its forms.


That depends. When the parents of a child with leukemia (95% curable now if ALL, with many fewer side effects) disdain mainstream medicine and take their child to Mexico for Laetrile or whatever the quacks there are selling, that should be illegal.

When Christian Scientists shun medical treatments for their children with life-threatening illnesses, that should be illegal.

When people go on Oprah and similar shows and claim they cured their own breast cancer by avoiding allopathic (mainstream) physicians and using "whatever" instead, that should be illegal.

When a person, even a child, who is undergoing the best medical treatment available for a life-threatening illness, wants to supplement that with electrolized water from the ancient stream of Thebes or 100-year-old dessicated forest mushrooms and those don't interfere with what he/she is already getting, then I say go for it.

I admit to a bias for allopathic medicine and blame it on my religion (Jewish) that believes physicians are G-d :).

Do you still feel that way when - according to someone I know who used to work for a pharma company - most drugs are only effective for 25% of the patients who take it!

We can quibble over whether that is really 25% or closer to 50% but, medicine just does not work on some (large) proportion of people.

"don't interfere with what he/she is already getting" One of the points above is that until you have run the statistical significant double blind trials, you don't know whether or it interfers. Considering the variations in metabolism, even then you probably wouldn't know for absolute certain.

By the way - i do actually agree with you on all your points, but I have doubts which is why I started this thread.

Message 7: reading_fox -- You're right when you say medicine doesn't work for everyone, though I think 25% or 50% is probably too low. For example, if statistics show that ALL leukemia in children is 95% curable today, the established treatments used must work for 95% of those patients.

My youngest daughter had primary bone cancer when she was 12 (15 years ago) and nothing could have persuaded me to leave the country in search of some "holy grail". She went through a living hell during treatments and was not able to complete the protocol (all children with cancer are put in clinical trials) because the chemo was curing her and killing her simultaneously. There was a new anti-nausea drug that just came out about then called Zofran. It's the gold standard in preventing nausea and tests showed it worked on 99% of patients. Not her. The only way the doctors could keep her from continually throwing up was to sedate her for days at a time. And she still ended up on a feeding tube. She lived in the hospital for almost the entire ten months because the treatments made her so sick and so susceptible to other ailments. But I never once considered stopping the drugs and following the siren call of some pied piper who claimed he had all the answers the doctors didn't or that non-patentable "natural" compounds would cure her.

Physician themselves and their loved ones also get cancer. Don't you think if there were something better out there, they would use it for them?

Frankly, an unorganized human-phase drug trial that probably lacks oversight is dangerous and irresponsible. The fact that people are drawn to this trial by some type of news means they are likely to over-report reductions in negative symptoms and under-report side effects, and those combined with other factors could invalidate any controls built into the study. The reason drugs take so long to get onto the market is because these scientific processes are built into the system to prevent useless or deadly drugs from making it to patients. They still do, but often because the process was rushed in some way.

As for doubting that drug companies will lose money to generics, it's irrefutable that they will. Brand prices drop as soon as generics are available. Your argument with drug companies seems, really, to be about making product decisions based on profit rather than on potential loss or gain of life.

As for the statistics of how much of the population a drug works for, it really misses the point. Drugs are only designed to work on a portion of the population with a particular disease state. A prescription antihistamine would have had no effect on me last month. Today, it would be is the most wonderful drug ever. Drug research is only part of the scientific process for tackling diseases. The first part is actually identifying that there is a disease. Later, a physician must diagnose the disease state correctly and try and treat it with the medications available. But, there are other variables that the medical team, as scientists, must work around. If I had strep throat, penicillins would probably work well enough. They would kill my partner, however, as he's allergic to the whole drug class. Maalox or Mylanta would work well for my upset stomach, unless I have ulcers that require antibiotics and PPIs. All of those drugs have been approved to treat certain diseases in most people, but there are often factors that prohibit their use or make them ineffective in a substantial portion of the population.

I've gone on enough, so I won't start on "alternative," "natural," or "homeopathic" medicine. :)

"Drugs are only designed to work on a portion of the population with a particular disease state. A prescription antihistamine would have had no effect on me last month. Today, it would be is the most wonderful drug ever"

I'm not sure I understand what you are trying to say here? Last month you weren't alergic and hence an antihistamine wouldn't have helped? or last month you were alergic but that antihistamine didn't help, but now it does?

The latter is the point I was trying to make - In the UK there are 2 common non-prescription antihistamines, (loratadine and certazine (?) chloride ). My partnet finds that only loratadine works, the other has no effect at all. I find the opposite, for the same sort of pollen allergy. There are people for whom neither works!

"but there are often factors that prohibit their use or make them ineffective in a substantial portion of the population"

Yes - unrecognised factors, try this for 2 weeks, if it doesn't work we'll try something else. Not exactly the most reassuring words ever spoken by a doctor. When you get to the end of the offical list of "something elses" what do/should you do, if you hear about something that might work?