Comments on: Putting all our hope in one basket. http://saltyandsweet.org/2009/06/18/putting-all-our-hope-in-one-basket/ Daily life fighting Cystic Fibrosis and Cystic Fibrosis Related Diabetes. Thu, 08 Sep 2011 19:43:29 +0000 hourly 1 http://wordpress.org/?v=3.3.1 By: Nathan http://saltyandsweet.org/2009/06/18/putting-all-our-hope-in-one-basket/comment-page-1/#comment-537 Nathan Fri, 21 Aug 2009 17:16:53 +0000 http://saltyandsweet.org/?p=470#comment-537 Hello - I'm a 32 year old with CF. I also share your frustration with treatments vs. cures, but we still have to take improvements in treatment whenever we can get them until a true cure comes. Another company called Actelion just completed a Phase II clinical trial of a drug called Miglustat (already FDA approved and marketed under the name Zavesca to treat two other diseases). Miglustat appears to help the CFTR protein make it to the cell membrane where it can function (like VX-809), and works for people who have two copies of the delta-508 mutation. The previous lab results on CF lung cell lines look very promising. The full results of the clinical trial are expected soon. So,the good news is there is at least one other horse in this race for treatments of the basic defect. Actelion website regarding the Miglustat clinical trial: http://www1.actelion.com/en/scientists/development-pipeline/phase-2/miglustat.page Or here for another story about it: http://www.bio-itworld.com/els/07/22/09/miglustat-corrects-cystic-fibrosis-cells.html Hello – I’m a 32 year old with CF. I also share your frustration with treatments vs. cures, but we still have to take improvements in treatment whenever we can get them until a true cure comes.

Another company called Actelion just completed a Phase II clinical trial of a drug called Miglustat (already FDA approved and marketed under the name Zavesca to treat two other diseases). Miglustat appears to help the CFTR protein make it to the cell membrane where it can function (like VX-809), and works for people who have two copies of the delta-508 mutation. The previous lab results on CF lung cell lines look very promising. The full results of the clinical trial are expected soon. So,the good news is there is at least one other horse in this race for treatments of the basic defect.

Actelion website regarding the Miglustat clinical trial:
http://www1.actelion.com/en/scientists/development-pipeline/phase-2/miglustat.page

Or here for another story about it:
http://www.bio-itworld.com/els/07/22/09/miglustat-corrects-cystic-fibrosis-cells.html

]]> By: Sarah Kaupp http://saltyandsweet.org/2009/06/18/putting-all-our-hope-in-one-basket/comment-page-1/#comment-495 Sarah Kaupp Tue, 30 Jun 2009 01:26:04 +0000 http://saltyandsweet.org/?p=470#comment-495 My daughter has CF and she's 21 months old. Even though I am new to the CF world, I understand your fear about the Media claiming these medications as a "cure" and what that may do to research. There are so many more mutations, the CF research HAS to move forward. Even if these meds are exactly what they are being claimed to be, our fight is still not over. There are too many of us to be ignored. My daughter has CF and she’s 21 months old. Even though I am new to the CF world, I understand your fear about the Media claiming these medications as a “cure” and what that may do to research. There are so many more mutations, the CF research HAS to move forward. Even if these meds are exactly what they are being claimed to be, our fight is still not over. There are too many of us to be ignored.

]]> By: Jesse Petersen http://saltyandsweet.org/2009/06/18/putting-all-our-hope-in-one-basket/comment-page-1/#comment-484 Jesse Petersen Fri, 26 Jun 2009 16:32:37 +0000 http://saltyandsweet.org/?p=470#comment-484 To me, the passing on isn't nearly as big of a deal as it would be to either repair lung damage over the years from infections or to not have rejection concerns from transplants. Asking my Dr. last time how I'm doing... really doing, he said, "You're 30 and haven't had a lung transplant. You're doing great." To me, the passing on isn’t nearly as big of a deal as it would be to either repair lung damage over the years from infections or to not have rejection concerns from transplants.

Asking my Dr. last time how I’m doing… really doing, he said, “You’re 30 and haven’t had a lung transplant. You’re doing great.”

]]> By: Stephanie http://saltyandsweet.org/2009/06/18/putting-all-our-hope-in-one-basket/comment-page-1/#comment-481 Stephanie Thu, 18 Jun 2009 20:27:49 +0000 http://saltyandsweet.org/?p=470#comment-481 I think a lot of people in the CF community forget that lung tissue cannot be repaired. So if a new drug does come out, it may not benefit everyone. Like myself, I have a ton of damage and the only thing that will help me is for science to figure out a way to reverse scar tissue. I think a lot of people in the CF community forget that lung tissue cannot be repaired. So if a new drug does come out, it may not benefit everyone. Like myself, I have a ton of damage and the only thing that will help me is for science to figure out a way to reverse scar tissue.

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