Want to see something very cool? Not political

[Th1onein]

This is a snippet from an email from a mom of a child with cystic fibrosis who is currently using my drug. It's anecdotal, to be sure, but this is going on across the board with these kids who are using this drug (most with their doc's permission, I hope). Here, the mother is talking about the reduction of pancreatic enzymes necessary for digesting fats in these children. The drug works systemically for this disease:

"I feel like a naughty drug toting momma because her dosage has reduced by 2/3 at least it's quite amazing.. I don't know what to make of it. She seems v v well on it, it's extraordinary. My Dad - ex-pharma exec - wonders why the drug companies don't want to trial it and get a "use patent" if they can't patent the substance itself. Any ideas?"

Sometimes, I can't believe I'm responsible for something like this. I know I've worked very, very hard, and I've lost my own child to the disease, but it still shocks me that I accomplished this.

[A HERETIC I AM]

1. Are you a chemist for a Pharmaceutical firm?

You said "My drug". Does that mean this was a product of your research?

If so, you should be very proud and congratulations.

The depth and breadth of professional talent on DU never ceases to amaze me.

[Th1onein]

2. I'm the mother of a child who died of cystic fibrosis.

I'm not a professional. I studied the disease until I figured it out and then I came up with a drug to fix it. I did it in my spare time. It took me about nine years.

I am proud, of course. Sad, too, in a way. And, despite the long years of work, still surprised that it works so well. I've always had an ability to focus and apply myself, and to learn, but I just didn't think I would come up with the correct answer with the first drug. Beginner's luck, huh?

[unkachuck]

5. my hat's off to you, Th1onein....

....you're absolutely amazing....congratulations on a job well-done....you deserve a big, thumbs up!

[Th1onein]

6. Thanks!

I don't know why I'm still shocked over it working so well.

[RedCappedBandit]

24. Wow.. that is amazing. Cheers. nt

[yellowdogintexas]

3. a drug which reduces the # of enzyme pills a CF kid has to take? Am I reading this right?

I am very tired,but I just have to say.


CHEERS to YOU!

How does it work? I should think someone would want to trial it!

had two dear dear friends whose children suffered from this disease. My hat is off to you

[Th1onein]

4. It doesn't just reduce the amount of enzymes...

It works systemically, causing the cells to replace the mutated protein with another protein that actually works. Once it "kicks in," it is like the child "recovers" from the disease, in all affected systems. But, of course, it is not a cure--they have to take it for the rest of their lives.

[KC]

7. Just wondering

What it is ?

[Th1onein]

8. Benzyl isothiocyanate

Comes from papaya seeds. It's an oil. I have the use patent on the entire class of isothiocyanates for the treatment of cystic fibrosis.

[miles 2 go]

14. I hope you get very rich off of it

and are able to use some of that money to do even more astounding things. WOW, I am truly amazed at you. Way to go. If heaven exists, your child is beaming.

[Th1onein]

19. I assigned my patent to the foundation.

The only thing that I asked for was to be able to name the drug after my son. I did the work to honor him; no other reason.

[miles 2 go]

27. WOW again I say WOW

You are incredible. I hope the foundation makes billions on your patent. You certainly did honor him. You should feel very proud.

[A HERETIC I AM]

9. Well, that's astounding

I agree with the poster above - sounds like this is something that should be put through a trial process. You never know, you might have the cure right around the corner. Stranger things have happened.

Damn. Closest thing i ever came to any kind of cure is my anti-hangover, stomach rinsing technique for a drinking binge!


Again, you should be very proud. Congratulations. Efforts such as yours that have a positive impact on people reassures me of the idea that humanity tends to be inherently innovative and decent.

[Th1onein]

10. I learned a lot about the big pharmas, too.

This system is set up to either ignore you, or to take your work from you. The research world is a dirty, dog eat dog place.

[sam sarrha]

12. people at the FDA make more as "CONSULTANTS" to Pharma than their wages at FDA

[sam sarrha]

11. your story is wonderful..please elaborate... i know about the 'Pharma" control, i was suicidal from

hideous pain from rheumatoid arthritis.. i could not even feed myself, , or dress,i was 35 years old, i'm 59 now and no problems, i could barely walk if i could stand, knuckles in my hands were breaking backward.. and i couldn't straighten them out with my other hand..because i couldn't close it.

doing research on my own i ran into Dirk Pearson's book Life Extension. in which was information on the amino acids Arginine/Ornithine. there is an A/O cycle.. later if you are interested.. they are essential in the production of T-cell Receptor Inhibitors and growth hormones*.. it is the T-cells that attack the joints and cause allot of the inflammation of RA

after taking 2 to 4 grams of Arginine75%/Ornithine25% for a couple weeks my symptoms vanished. this has helped a lot of people i have shared it with. but since amino acids are non patentable and dirt cheap doctors cant tell you about it. i now only take once a week, the pains come back after about a month of not taking it..

apparently the word is out enough that even Walmart carries it in 75/25%.. http://www.vrp.com carries bulk vitamins, minerals, enzymes etc.. i get my supplements at Vitamin Shoppe http://www.vitaminshoppe.com their prices are good for my area

....there are a lot of good alternate treatments out there for lots of things, let us know more about yours.. i collect and share information



*Handbook of Physics and Chemistry

[Th1onein]

15. I'm not sure what you want me to tell you.

CF is a genetic disease. It is caused by the mutation of a gene responsible for the expression of a protein that sits in the membrane of epithelial cells, which line organs, such as the lungs, the pancreas, the kidneys, etc. It's supposed to transport compounds out of the cell and for a long, long time, scientists thought that that important compound was chloride ions, but they couldn't figure out why the lack of that transport would cause the disease. The day that my son died a paper was published showing that that protein transported, along with chloride ions, another compound, called glutathione. Glutathione is known for binding with other compounds, particularly toxins, and being transported as conjugates out of the cell. It turns out, that was what that protein was responsible for. The lack of this transport was what caused the disease, not so much the lack of the chloride ion transport.

The mutated protein belongs to a class of proteins, that transport compounds out of the cell. I found out that one of those proteins, that is usually induced in multidrug resistant cancer cells, is functionally redundant to the mutated protein. If that protein could be made to be expressed at a high level, then it could substitute for the mutated protein, and the pathology would stop. But you can't give these patients chemotoxins on a daily basis for the rest of their lives, so I had to find something that would do the same thing, but that wouldn't be toxic in the doses that were necessary to induce the expression of the redundant protein. I looked at the enzymes responsible for combining glutathione and toxins so they could be transported together out of the cell, and I looked at the compounds that increased the expression of these enzymes and I found the isothiocyanates. It was simply a matter of choosing the least toxic and the most efficacious, of the isothiocyanates, at that point.

This is bunching all of the work together into two paragraphs, but the whole thing took about nine years to figure out. While I was doing this, a bunch of parents of CF kids got together and formed a non-profit foundation to support my research, and we are in the process of getting this compound tested in these kids. Anyone who has followed this work, though, can get the compound from a chemical company, and many parents have done that. Especially those in the EU, where doctors seem to be a lot more open-minded and will actually read the research that their patients bring to them. These are the children that are taking this drug.

In the States, it is much harder. Especially for CF, which is rigidly tied to the big pharmas. I actually had a doctor from a large research university here ask me if he could test my drug. I was elated, but my patent attorney heard about it and told me that was a nono because they have contracts with the big pharmas and if they do the trials, they have a right to your intellectual property. I wouldn't mind that, except that I know that the big pharmas stand to lose a lot of money on their own drugs for this disease, whose patents have not run their course and they would likely bury this, until those patents have expired.

[some guy]

13. Congratulations!

:hug:

As you know, I've been following your posts on this (at least the ones I happen to see) and so continue to be supportive and impressed iwth your efforts and success.

You TOTALLY rock!

I am so happy for you, and hope the good news continues for you.

:bounce: :bounce: :bounce:

:loveya:

:D

[Th1onein]

17. Thanks, Some guy!

I am so pleasantly surprised by the results that we're getting. I knew that I had "done my homework," but I didn't expect to see such dramatic results so quickly, and at such low doses. Low doses, as you can imagine, means very little chance of toxicity. It is quite wonderful, what is happening.

[some guy]

25. What

other concerns do you see as potential hurdles in this treatment? I had asked before about toxicity, and you thought that wouldn't be a problem because the dosages were low, so you seem to be correct on that. Is there potential for the patient to build up a tolerance for the compound such that it becomes less effective? Long term concerns of any type?

From the description I read of what you did, and how the compound works, it doesn't seem to me that are any potential down sides, but I really only barely understand (or at least, think I understand) what the compound is doing, so I'm curious as to what you think long term.

:hug:

[Th1onein]

28. The process of acquiring tolerance is what it is based upon.

You know, "tolerance" requires a change made in the cell? I mean, something new has to happen in order for tolerance to be built up. Your cell has to start making a new protein, that can move the compound that you've built the tolerance to, out of the cell; and that's usually what happens, when you are said to build up a tolerance to a drug (either to your cells, or to the bacterial cells that are attacking your system). The thing here, though, is that it is that very tolerance that we are utilizing. We are utilizing the Type II detoxification process to induce the redundant protein. So, if anything, you're going to need less of the drug, as time goes on, to achieve the same effect, not more.

It looks like this drug will make a big splash in the EU, because research is not as rigidly controlled by the big pharmas over there like we are here. Once that happens and hits the airwaves, so to speak (thank God for the internet), they'll look pretty bad in the States if they don't pick it up. The only place I see for myself, after that, is helping the process along. I know quite a bit about this little world I have immersed myself in for so many years; they are going to need that.

[crispini]

16. K&R

:hug:

[FloridaJudy]

18. What an inspiring story!

You have every right to be proud.

:yourock:

[BlackVelvet04]

20. If you need a new project.....

how about figuring out interstitial cystitis and coming up with a real treatment that works.

[Th1onein]

21. I have been searching for something else to study....

but, you know, when you've learned a lot about a specific system, it's hard to go to a brand new one, and lungs are kind of my thing. I am very curious about pulmonary fibrosis, but until I get this drug launched, I'm kind of tied up still.

[BlackVelvet04]

23. Oh, and congratulations on your success

and the potential of helping so many people. I admire your tenaciousness.

I understand what you are saying about changing systems.

Unfortunately interstitial cystitis doesn't get a lot of research money and millions of Americans suffer from the condition. It's extremely painful and there is no cure and no really good treatments for the condition.

[myrna minx]

22. Congratulations!

I know you've been down a long road. You should be proud of what you've accomplished. :applause:

[kas125]

26. Wow, that is amazing

It must be very gratifying to know that your drug is really helping people. I hope that some drug company will test and manufacture your drug. Congratulations on your success.

[Richard Steele]

29. K&R

[applegrove]

30. Wow. You changed the world for many people. Saved lives. It is truly amazing what

one person can do. Just amazing!

[eppur_se_muova]

31. Have you ever edited a wiki entry? "Isothiocyanate" could use a little help ...

http://en.wikipedia.org/wiki/Isothiocyanate

and it might help get your message out.

[Th1onein]

33. I don't know how to do that.

I'm sure it would get word out about this new treatment, but I just don't know how to do it. Do you? Can you tell me how?

[eppur_se_muova]

34. Sorry, no. I'd do it myself if I could. I don't believe it's hard, just haven't gotten into it. nt

[flashl]

32. Continue the great work.