They're calling for volunteers for the clinical trial of my drug!

[Th1onein]

I am so excited. And scared. My work is going to be tested.

I never thought I'd get this far, but here we are! If my model is correct, I will have stopped one of the most common genetic killers of children in this country. If I'm wrong, it's back to the drawing board.

It seems almost surreal to me, at this point, to see the call for volunteers.

[MadMaddie]

1. Congrats my DU friend....and good luck....

:applause:

[Downtown Hound]

2. Wow! Good luck!

It must feel really good to have gotten this far. I understand that's not easy in that industry.

[JeffR]

3. Keeping my fingers crossed for ya!

:toast:

[Cameron27]

4. How amazing!

Congratulations and good luck.

[Old and In the Way]

5. Wow, that is really interesting.

I admire anyone whose knowledge of chemistry and biology works at this level. Good luck, maybe they'll be a Nobel Prize in your future!

[Th1onein]

10. I very much doubt any prizes are coming my way

I'm a "non-professional scientist." I got into this because my son had cystic fibrosis and I was fighting for his life. That was eleven years ago. After he died, eight years ago, I just couldn't stop studying. A group of parents on a CF support group heard about the work and they decided to support it. These people didn't just form a non-profit and raise funds; they never quit and they never let me quit. They never gave up and they never let me give up. And here we are. It just goes to show you what people, joined together for a common purpose, can do.

I don't deserve any prize. This group of people who came together in love, with a fighting spirit and a common cause, deserve all of the prizes.

[Catherine Vincent]

17. Wow! That is very commendable of you.

Congratulations! And I hope your testing is a success!

[Old and In the Way]

33. Even more amazing......I'm so sorry about your son, but I can understand

your motivation to find a cure. I truly hope your work is sucessful, it will be a gift to millions and provide you some comfort in your personal loss. What a story of inspiration. Thanks for posting about this.

[malaise]

43. Nice start to my day

:yourock:

[Horse with no Name]

54. Awesome.

A friend of mine lost her son when he was 7 to CF.
Thank you for your great work.

[KoKo]

64. Great to hear! Thanks for the news!

:applause:

[livetohike]

6. Congratulations and Way to Go!

:thumbsup: Looking forward to hearing more positive news from you on this :-).

[catnhatnh]

7. What is your disease?

I had two nephews killed by a form of MD passed only to male heirs (I forget the exact term) and since they were the first boys in two generations.....I always hoped to see that bitch whipped in my lifetime....

[gully]

16. See post 10

Cystic Fibrosis

[Th1onein]

30. Your disease was Duchenne's Muscular Dystrophy?

It is carried on the X chromosome, so most females with the disease have a functional form of the protein. Males are not so lucky. Duchenne's is a particularly cruel disease. The last I read about it, though, they were very close to doing something very radical to change the outcome for these children. I don't know that they will, though, unless some "radical" parents get together and make sure that this research is followed out to it's logical end.

[catnhatnh]

32. That was the name...

I cannot follow it too closely...I do what I can for fundraising and hope it is given proper gravity....If I give X dollars towards MD, would it not seem somehow cheap to name which form was my aim?

[Th1onein]

44. It is the deadliest form of the disease.

The other forms don't kill as rapidly as Duchenne's.

[shance]

8. That's exciting Th1*** Congratulations!

Good luck - may many blessings come your way!

[catnhatnh]

9. Oh-and congratulations...

...and fingers crossed for all of us...

[librechik]

11. Cool--what a great feeling that must be

enjoy and thanks for your dedication!

NGU!

[catnhatnh]

12. And a last thought...

...you know the odds and the statistics.You have done your best and the world wishes you well-ain't no one hoping against you. You are what this world needs whether this attempt is a success or not.There is no youthful killer that doesn't deserve and need you and others like you.So you walked to the plate and swung-I pray you hit it out of the park.If not there are other pitches and other swings....Go,Baby....

[Th1onein]

21. What an absolutely lovely thing to write, catnhatnh

I'm scared. And very tired. Eleven years of work is a long time.

But, you know, if I don't hit it out of the park this time, my butt will be back on the practice field the next morning, trying to figure out where I went wrong. I've gone too far to turn back now.

[catnhatnh]

29. Darling....

...I watched my brother-a much better man than I-and my sister-in-law (a double saint for her love of the boys and her tolerance of my brother) lose their kids-and so have you-life can kick you again but never that hard...eleven years IS a long time-guess who smiles on and blesses your work tonight-no matter the outcome???This is your Taj Mahal-a labor, an homage to your love-and love is never lost or poured down a blind hole....I pray it works, but your love is pure and perfect.God Bless those who drive too fast into a turn-because if they pull it off, they are superstars and if they don't their inspiration is special...Regards,Cat

[Canuckistanian]

13. You've been having quite a year

First your sister and now this.

Karma works in stange ways....

Congrats!
:toast:

[SlavesandBulldozers]

14. wicked.

congratulations are already in order no matter the outcome. I admire your focus and drive and wish you all the best. You certainly deserve it!

[benddem]

15. the fact it is going to go to clinical trial

is amazing..and many congratulations. I will keep my fingers crossed for you and all the cystic fibrosis kids now and in the future. :toast: :grouphug:

[lumberjack_jeff]

18. I just read Sex, Drugs and DNA

Apparently the percentage of researchers who EVER make a drug which actually goes to clinical trial is very small.

Congratulations!

[stevedeshazer]

19. Wonderful

:hug:

The world needs more of that.

[mmonk]

20. Pulling for your success.

My fingers are crossed.

[HeeBGBz]

22. Well, WTG, Th1onein, I wish you the best possibilities!

"I want a new drug
One that does what it should
One that wont make me feel too bad
One that wont make me feel too good"

[Lars39]

23. I remember your story!

Just reading it was mind boggling! I hope all goes very, very well. :hug:

[G Hawes]

24. Best of luck to you, Th1onein

And great big kudos to you on your work, your passion, and your perserverance.

[Control-Z]

25. Oh, wow!

That is an enormous accomplishment.

It must have taken amazing strength - to work so hard - to persevere.

I wish you the very best in those trials.

[driver8]

26. A big congratulations to you !!

That is really amazing! What a story...

[Kurovski]

27. Best of luck, and thank you!

Recommended.

[dave502d]

28. Good luck and thank you. n/t

[sandnsea]

31. Wow!! Congratulations!

Is there another "Lorenzo's Oil" in the works perhaps?? You have a fascinating and inspiring story. I sure hope the medicine is successful and helps lots of kids. Congratulations again!

:applause:

[TygrBright]

34. Wow. Just... Wow.

Thank you.

People like you give me hope. I remember reading about Jonas Salk when I was a youngster, and how he refused to patent his vaccine because he wanted every kid to be able to live free of the horror of polio. There were still kids who had survived polio in school with me; it was a very real threat.

I read "The Microbe Hunters," and thought that there was nothing more exciting and heroic than the men and women in laboratories, working to free humanity from the fear and suffering of terrible diseases.

But by the time I grew up, it was all about "money pills"-- Big Pharma had consolidated everything into a machine diligently searching for profits, working on yet another cure for acid indigestion or male pattern baldness or weight gain or erectile dysfunction. "Researchers" would make their latest "discoveries" by making one minute molecular change or addition to some compound so they could take out a new patent. Drug companies were refusing to manufacture and distribute life-saving vaccines because there wasn't enough profit in it. Holding dying AIDS patients and health care systems in poverty-stricken countries hostage for vast profits on hypercostly medicines.

It seemed like there were no more of the white-coated lab warriors tackling the toughest, cruelest diseases because they BELIEVED in a cure, believed in the importance of the work, even if it wouldn't make a huge profit.

Thank you for restoring my faith. My prayers are with you and the clinicians managing the trials.

appreciatively,
Bright

[Bridget Burke]

49. The "white coated lab warriors" never went away.

But the "suits" decide what deserves funding. Most of the time, that is.

[shadowknows69]

35. If this succeeds

would you consider designing a drug that eliminates the genetic predisoposition to become a republican?

[Diane R]

36. Bless you.

I know a wonderful little girl with CF who desperately needs a cure. And, I know a girl who died when she was only 20 years old.

Crossing all my fingers, toes and eyeballs that this trial will work. But even if it doesn't, you've helped everyone move closer to a cure.

[Th1onein]

45. We should know the results in three or four months.

We had a carrier (a parent) who took it upon himself to test this drug on himself and the results were amazing. It is, without a doubt, one of the most satisfying feelings in the world, to go from a theory on paper, to the lab, and to see it bear out. You can be as sure of your theory as is humanly possible and still be absolutely shocked at the results of testing it, especially when the outcome is so dramatic.

I don't know if it will work in CF patients, themselves, though. Or even if it manages to change one of the major pathological indices in this disease, if it will change the overall pathology of the disease. It is going to be difficult to wait and see. Thank God I have my day job to keep me busy.

[DeepModem Mom]

37. My daughter lost a very close friend, an extraordinary girl...

she'd known since early childhood to CF not too long ago. I pray your efforts succeed, and I thank you.

[pitohui]

38. go you and good luck

there would be no need for tests if we knew everything in advance

you are trying, that's what counts, take it a day at a time

[BlooInBloo]

39. How exciting! Congratulations!!! Good luck!

[RevolutionStartsNow]

40. Amazing story.

Beautiful. Your compassion and determination is so admirable.

Keep pushing!

[KT2000]

41. This is great!!

All I can say is
:bounce: :yourock: :applause:

[lostnfound]

42. We are excited for you!!!

How proud you should be. I deeply hope it is a big success.

[AzDar]

46. That is fantastic! All the best....n/t

[knitter4democracy]

47. That's great!!!

You'll find out so much more in the testing phase. You'll see how it really works in real life, and you just can't duplicate that in a lab. I'm so happy for you! That's a drug that we need.

[myrna minx]

48. Your extraordinary work has truly honored your

child's memory.

[IdaBriggs]

50. Yeah! You Rock! Congratulations!

:yourock: :woohoo: :bounce: :toast: :yourock: :woohoo: :bounce: :toast: :yourock: :woohoo: :bounce: :toast: :yourock: :woohoo: :bounce: :toast: :yourock: :woohoo: :bounce: :toast: :yourock: :woohoo: :bounce: :toast:

[Richard Steele]

51. I hope it tests well, Th1onein! nm

[Avalux]

52. Phase I trials?

I'm assuming you've already done animal models with good results. I'd be interested in knowing more about your compound. :hi:

[Th1onein]

53. We didn't have to do an animal model

It is a compound that comes from a food, so we didn't have to do that. We are testing directly on the patients, themselves. The compound is benzyl isothiocyanate, which comes from papaya seeds.

[civildisoBDence]

56. Regardless of the outcome of the clinincal trials,

you've forwarded the science on CF.

Congratulations--may more be in the offing!

Newsprism

[Avalux]

57. Thanks for the info.

I wish you the best of luck - please keep us posted on your progress. :hug:

[Fleshdancer]

55. WOW! please please PLEASE keep us updated!

may I ask what genetic killer you're refering to?

on edit: nevermind...I read more of the thread. :D Your story has me tearing up. :hug:

[Mnemosyne]

58. How exciting Th1onein! My nephew died at 8 months of

global genetic defects. I know how much this means.

:yourock:

[brook]

59. what a compelling

story. To read what you have chosen to do after such a tragedy as losing your precious child is awe-inspiring.
May the deities bless you and this trial.

[wiley]

60. This compound has been studied in animals and humans for at least 30 years

Herbs and fruits and vegetables that contain this compound have been used by healers for centuries. The US Army Survival Manual mentions severasl sources of this compound for certain illnesses (mainly helminths). Please explain what you mean by your model. Is it based on eating papaya seeds over a certain period of time, or a pill taken x number of times a day, or an on one week off several months infusion of benzyl isothiocyanate.

Would you please provide a link to either a protocol description of the clinical trial you are talking about that is recruiting, or at least the Informed Consent for this study? Is the protocol rationalization based on BITC as a mechanism-based inactivator of various forms of P450s? Just a little bit more info, please? Published papers?

[Th1onein]

62. I don't have the protocol on this computer; sorry.

And I'm not sure that it is available to the public yet. The IRB has it, though. As well as the informed consent. No, BITC is not used to inactivate any form of P450. It is used to induce a functionally redundant protein (MRP-1) to the CFTR protein, which is mutated in this disease.

You can look up the abstract on PubMed, by searching "Melanie Childers." It's the only abstract that pops up under those search terms.

[wiley]

65. Thank You. Very Exciting. I pray it works

If so, it could not only address CF, but perhaps serve as a prototype for addressing similar concerns related to drug resistance (not a shabby idea considering MRSA and resistance that develops to otherwise effective drugs), esophageal and lung and or breast cancer, AIDS, etc. I look forward to seeing a protocol perhaps in the future, but first and foremost hope that the first person who takes this drug in whatever formulation leads to a second person and to the completion of a successful Phase I study. That's all and everything that we can hope for. I wish for so many kids that this was a therapy available right now, but we know how research works and why it must be done. Even a small successful proof of concept trial with mixed results could blow the whole field wide open and lead to better treatments for those coming up. Good Luck to us all. May you never tire or lose hope in a despondent hour.

[nicknameless]

61. Awesome, Th1onein!

I am so sorry you lost a child to this.
Thank you for never giving up.
Please keep us posted.

[mrreowwr_kittty]

63. Well done!

I'm so happy for you. I remember when you posted about your sister passing. I'm glad for this positive development in your life and career and I wish you the best of luck. :hug: