Cort, this is a great blog. I am very impressed with this one. It is well-written. It reveals the obstacles, medical research system mismatched to a multi-system syndrome.
You started it with the main message in the first paragraph.
I see some of the thoughts come from Newsweek, but you do add some of the thoughts of how this problem affects CFS and the response to that.
From "Lorenzo's Oil":
Lorenzo's mom: "Our son has complied with this diet yet his levels of his saturated C24 and C26 have risen for two months."
Another parent in the audience: "That's exactly what happened to Jay Bird."
Lorenzo's Mom: "Perhaps, before talking about cook books, we should ask ourselves if the diet is working at all."
Another mom: "Damn right we should."
ALD patient group leader: "This is not the way we do things here."
Lorenzo's Father: "Excuse me, there are two families with this paradox, maybe there are more."
Lorenzo's Mom: "Shouldn't we open it for discussion?"
Another parent: "My boy's levels are rising."
Lorenzo's Father: "Now three families. Maybe we can have a show of hands and maybe we will see how many families there are."
ALD patient group leader: "You don't understand, this is a formal pilot study. It has to run the full 6 months. We're not scientists. We don't take it upon ourselves to interpret experiments. That's the solemn responsibility of the doctors."
Another parent: "Come on Ellard, let's at least have a show of hands."
Leader's wife: "No, no, that would be misleading. Now the medical folks call this kind of evidence 'anecdotal'."
Another parent: "What's wrong with a show of hands?"
Leader's wife: "It wouldn't have any meaning."
Recipe book lady: "Excuse me, may I say something. The only way doctors can get useful results is a strict protocol and statistical samples in a control group."
Leader: "And a proper time frame. Doctors have to be very careful. Clinical trials must undergo a tremendous amount of scrutiny."
Leaders wife: "This is the way medical science works. That's the only way scientists can get the information that they need."
Lorenzo's Mom: "So what you're saying is our children are in the service of medical science. How very foolish of me, I always assumed the medical science was in the service of the sufferer."
Leader's wife: "Yes, thank you. Now I think it's time to get back to our agenda."
Lorenzo's Mom: "Why? So we can discuss further salvaging our marriages and managing our grief? What about the children? No one here is discussing the children."
The Lorenzos later set up The Myelin Project, for research in many illnesses. The approach is different: To attain its objectives, The Myelin Project relies on three major strategies: prompting researchers to work as a team and coordinating their research efforts, promoting interaction between researchers and laypeople, and rapid financing of practically oriented experiments.
There has been a shift in Pharma research over the past 10 years. As the article stated, the big companies have stopped churning out drugs which are only effective in a minority of diseases they are designed to treat.
The head of GSK research really said only what most people in the pharma industry already knew. His remedy to start matching genetic profiles to drugs opens up a whole new field which appears to have been enthusiastically accepted in research circles.
However, they appear to be finding the research much harder when the mixture of genes interact in ways far more complicated than they at first expected and there are also more disease pathways being found.
This is probably why they are spending a lot more money on trying to crack genetic codes of disease, which would be essential before they could design more effective drugs.
They know however that when they do crack some of these codes and have effective drugs they will be sitting on a goldmine. Just imagine a drug that was not only effective against one form of cancer but all forms of cancer.
The Head of GSK research would not have trashed his drugs the way he did if he did not think this was the way forward for future research.
This doesnt help the likes of us though in the short term with a combination of hormonal, immunological and neurological problems. Our best hope is that some of the smaller Biotech drug research companies will come up with something, despite the shortage of funds now available from venture capital sources for these sort of companies.
In the meantime we must keep up the pressure on governments for more research funding.
The Myelin Project relies on three major strategies: prompting researchers to work as a team and coordinating their research efforts, promoting interaction between researchers and laypeople, and rapid financing of practically oriented experiments.
It seems like more and more of this is happening - mostly in the private foundations. We're kind of trying that approach here as well - with the Patient Data Repository/Treatment Review program that will enable patients to give inputs of drug and therapy effectiveness = thus revealing patterns that would never show up in the medical literature.
Thanks for the explanation liverock - hopefully over time digging deeper will pay off for all of us.