My paper was just published!

[Th1onein]

Yehaw!

Go to www.sharktank.org to see the abstract and link to the full text article in Medical Hypotheses.

[bluerum]

1. Congrats. I have a cousin who has cystic fibrosis.

[Th1onein]

2. Perhaps you should send your cousin to read sharktank?

We have helped a lot of people with CF. We have been instrumental in forcing changes in the treatment protocols for CF.

[ms liberty]

3. Congratulations! n/t

[Ilsa]

4. Congratulations! It is AWESOME that DUers are so smart and

well educated.

"And many more..." publications to ya! :toast:

[yellerpup]

5. Congratulations on being published

and thank you for your life saving research.

[CoffeeCat]

6. Wow! This is amazing!

Hi!

I don't know anything about CF, but your research and findings are truly groundbreaking. This was fascinating to read.

It sounds like your research will enable better treatments for CF and help untold numbers of people.

Is it your hope, that a new drug--based on your research--will be developed?

This is so cool. Thanks for sharing your great news with us. You and your research group must be so excited! It's not everyday that someone changes the world through their job, but you are.

This should be in the mainstream press, or at least publicized in science-oriented publications that are widely read by the general public (Discover, Popular Science). I bet a science reporter from the New York Times or the Washington Post would pick this up.

Awesome! :toast:

[Th1onein]

7. I'm not a scientist by trade, actually.

Science is not my job. I did this in my spare time (I don't sleep much, sometimes). I started studying because my son had CF. After he died, I couldn't stop. The research group began right after my son died, and was formed because of my research. Eight years later, here we are. We are going into clinical trials, hopefully, next month. I have a patent on the drug that we are using, which comes from papaya seeds (benzyl isothiocyanate). It worked on carriers, and we believe it will also work on patients. If it does, the treatment for CF will be changed very radically, and CF will no longer be a death sentence.

[Subdivisions]

9. That is amazing...

Congratulations!

I would very much be interested in hearing more of your overall story. It sounds very inspirational that a non-scientist mother could take on such a challenge and end up with an issue that has been subjected to the full battery of scientific review, a patent on a drug and a published work.

I am proud of you and I wish you the best as you move forward towards further testing and trials. You're dedication to advancing human knowledge of Cystic Fibrosis is surely to make a huge impact on those who suffer this tragic condition.

Salute.

[Marnieworld]

10. I am so moved by your story

And impressed and amazed. Have you considered exploring the possibility of selling the rights to your story? TV or movie rights? I don't mean to diminish your accomplishment or your struggle but I think it is a very compelling story that could be inspirational for many people. Like an Erin Brokovich story or Not Without My Daughter. Even Lifetime. Obviously you have other priorities but possibly by making the story public it can help other people in similar situations.

Anyway, Congratulations on the paper, the patent and for turning something so tragic into positive beneficial energy.

:yourock:

Peace

[Th1onein]

12. I've had people approach me about it, but

I think it's probably too boring to be anything than what it was--years of work and boring (to most people) biochemistry.

Thanks for the congrats, though!

[Straight Shooter]

13. Maybe not as "boring" as you think.

Have you ever seen the movie "Lorenzo's Oil"?

http://www.myelin.org/themovie.htm

The film is based on the true story of Augusto and Michaela Odone and their son Lorenzo. In 1984 Lorenzo came down with adrenoleukodystrophy (ALD), a rare inherited disease. Doctors said that he would lose all his functions and die within two to three years. Refusing to accept this grim verdict, the Odones set out on a mission to find a treatment for ALD and to save their child (Lorenzo survived, and is now 25 years old). In their quest for a treatment the Odones often clashed with doctors, scientists, and support groups, who were skeptical that anything could be done about ALD, much less by laypeople. Their relentless struggle tested the strength of their marriage, the depth of their beliefs, and the boundaries of conventional medicine.

The Odones haunted medical libraries, reviewed countless animal experiments, badgered researchers, questioned top doctors all over the world, and persisted until a solution came to them in a moment of inspiration. They commissioned a special type of oil from a British firm, which normalized the accumulation of very long chain fatty acids in the brain, the hallmark of ALD.

I am so sorry for the loss of your son. You have my deepest respect and admiration for honoring his memory by trying to help others. :hug:

Well-deserved congratulations for your hard work and dedication. :thumbsup:

[bryant69]

8. Congrats.

That must feel great.

[myrna minx]

11. You are amazing!

Your work is going to help so many people. What a lovely tribute to the memory of your child. :hug:

[Generic Other]

14. Impressive

Congrats! :)

[Celebration]

15. wow what a story

Truly, I am so sorry about your son. I can understand where you got your inspiration. Anecdotally, can you tell us that the proposed treatment works well? Is it taken in the form of diet changes, or are supplements involved?

What is your next project?

[Th1onein]

18. We've only done a carrier test so far

In cystics and in carriers (CF is an autosomal recessive disease, so both parents have to carry the gene and give a copy of it to their child for the child to have the disease), there is an imbalance in the fatty acids DHA and AA. AA is high; DHA is low. We took blood for a baseline, tested the levels of fatty acids, then administered 5mg of benzyl isothiocyanate for one week, tested again, no change. Then we tried 10mg of benzyl isothiocyanate, one week, another blood test, and voila! The levels of DHA had risen and the levels of AA went down, both quite radically. So, in one of the major indicators of pathology in the disease, benzyl isothiocyanate made quite a difference. Our next trials will be in the patients, themselves.

[Celebration]

19. How about DHA?

Can that be given by itself? Obviously I understand the need for trials. But have some with cystic fibrosis, based on your information, on their own radically changed their diets? If so, is there any anecdotal information on that? Even if the patients need more benzyl isothiocyanate than their parents, presumably a level could be hit that would work.

We are so proud of you.

[Th1onein]

20. Changing your diet won't work

You can't get enough of benzyl isothiocyanate into you by just changing your diet. And increasing the amount of DHA you ingest doesn't work, either. The fatty acid imbalance is part of the problem in CF, and it certainly contributes to the increased inflammatory cascade in the disease, but it is not the root of the problem. There are many other things happening that contribute to other pathologies in these patients. You can restore levels of DHA to normal, which will decrease AA levels, but this does nothing to address the lack of cell-mediated immunity and a plethora of other pathologies seen in these patients. The simple fact is that you must have a CFTR protein in that membrane, or next best, another protein that is redundant to the CFTR protein (mutation of the CFTR protein and subsequent degradation of it, before it can "traffic" to the membrane is the cause of the disease). Benzyl isothiocyanate induces the expression of that redundant protein: multi-drug resistant associated protein (MRP) and that protein can and does function just as well as the CFTR protein functions. Supplementing DHA solves one problem; replacing the misfolded protein with a functionally redundant protein solves them all.

The research group was formed on the internet. People who had children with CF came from a support group for that purpose and decided to support my work by raising funds and contributing ideas. This was a grass roots effort to educate ourselves and find something that worked, because mainstream science was failing us, and failing our children. We were tired of waiting on people who cared mostly about making a buck, so we did it ourselves.

Unfortunately, along the way, we found that some of our work was being usurped (ours is a public forum) and claimed by others. Science, in turned out, was a cut throat world; very competitive. So, we began to keep a lot of our conclusions 'under our hats.' At least until we could publish and get a patent on the compound for it's use to treat CF. So...some people have used some isothiocyanates (not benzyl ITC, though) and found that they do help a bit, but they only used them to the degree that they could find them in food, which is not a high enough dosage to cause the induction of the redundant protein.

We have the patent on this compound, and we are currently raising funds to test it's efficacy and safety in CF patients. We are contracting out that study. In fact, if you would like to donate to that cause, you can do so at the sharktank website: www.sharktank.org

Before my son died, I promised him I would stop this disease. I have worked these past eight years toward that purpose. I have had many wonderful people working along with me, towards that purpose. If this drug works, I will have kept that promise, but I am sure that I will take up some other study, since it would be a shame to waste all of the knowledge that I have gleaned from this quest.

The bigger thing, though, is this: What we did; what this group, starting out small--less than 50 people on the net, and now close to three hundred strong--did, just everyday people, from all walks of life; this is a message to anyone and everyone in the same position: Together, you can do anything.

[katinmn]

16. Great work! Bookmarking for follow up.

:)

[rasputin1952]

17. Congratulations...and I hope and pray that a cure or at least an

"arrestor" come of this and other research. CF is a terrible disease and is often just 'swept under the rug'...Let's all hope progress will made and cure is in the near future.

[bleever]

21. Congratulations!

Very impressive work. Best of luck with the clinical trials.

[EFerrari]

22. Congrats! It's a great feeling, isn't it?

Enjoy!

:toast:

[ismnotwasm]

23. Excellent!

I work on a transplant unit, and while we don't "do" the lungs--they go to ICU, we get transplanted cystic fibrosis patients fairly regularly-- in for complications or occasional rejection

These patients are living longer with better quality of life than before. It's a heartbreaking disease and I have a lot of respect for the research being done in it.

Congratulations!

[Th1onein]

24. We were trying to get a transplant for my son when he died....

He didn't want one, but it was his only hope. Unfortunately, he died before it could happen.

Hopefully, if this new therapy works, it will obviate the need for lung transplants in these patients. We should know if it's going to work within the next couple of months.