Vertex drugs and pancreatic function

[zeeannie]

Regarding these drugs, particularly Kalydeco in combo with VX-809, and Kalydeco in combo with VX-661, I understand that they improve lung function. One drug acting as a potentiator, the other a corrector allowing NaCl to reach the surface of the cell and correcting the problem of sticky mucus. Is it safe to assume that it's affecting all cells, therefore all systems affected by sticky mucus will benefit? Specifically the pancreas. Assuming there is no damage to the organ or the duct, will blockages clear and function return to normal? Has anyone in trials experienced this?

 

[rillopy]

I gained about 5 pounds over the 4 weeks of Phase 2 VX-809/Kalydeco. No idea if it was improved pancreatic function, or because I was burning fewer calories on airway clearance.

 

[Printer]

I was told, by the leading adult CF-GI researcher in the US, that there seems to be no benefit, to the pancreas, from Kalydeco.

Bill

 

[knowitall]

Bill no one cares what your leading GI moron said.

The reason Kalydeco fixes or is perceived to fix pancreatic function is because most of our pancreas (maybe no printer since he is "100%" PI) still secrete exocrine hormones and enzymes, but the pancreas also makes acid at the wrong times as well as bicarbonate becaue the CFTR interfaces with the SLC48A gene that is responsible for bicarbonate ion transport. So when the CFTR is now fixed the SLC4A8 can now resume normal function and the aicd/bicarb issues resolve to an extent and don't fuck up the enzymes our body IS making.

 

[OJL5-12-12]

It must do something.. Of those who are prescribed Kalydeco now for g551d many have been able to reduce enzymes and iv read of some who have stopped enzymes altogether!!

 

[Printer]

I would love that, if it is true.

Bill

 

[Lanna429]

g551d

I was in the double blind study for Kalydeco. And am prescribed it now. I was able to cut back on my enzymes and if I miss a dose of enzymes it is not a disaster right away. I can go almost a day w/o them (not on purpose. It just happens sometimes) I take creon 24. 4-6 with meals. Rarely with a snack.
I have
DF508 & G551D CFRD

 

[Chrissyb]

I can't wait for it to come out for my jean I'm really happy about it

 

[dasjsmum]

Hi There

Its been while since I posted here, but I can tell you that Kalydeco absolutely does improve digestion. My son is almost 34 and been pancreatic insufficent since he was 5mths old. He has now been on Kalydeco for 3.5 years (he was in the Ph3 trial for 770 & G551D and was on the actual drug throughout).

Within two weeks of the trial starting he had put on quite afew kilos (cant remember how many now). He stopped taking enzymes during the trial - even though he wasnt supposed to, he felt he no longer needed them. He hasnt taken Creon now since that time, and is great. So sorry Bill, but he isnt the only one either.

The problem is that they werent actually testing for pancreatic function during the trial, and I dont think they've targeted that specifically since, concentrating more on FEV1 improvement and sodium chloride reduction. If they had tested for pancreatic function during the trial I have no doubt it would show improvement in function. We were always told that the pancreas was scarred, however I think it was just blocked

Just to add, my son (age 34) takes NO enzymes and hasnt for about three years, and that's since needing them from when he was 5mths of age...the only times he takes any enzymes is if he has a very fat laden meal.

 

[zeeannie]

That's great news dasjsmum! It seems logical that the same principal would apply to any system affected as long as damage is minimal and function is possible. That's what I'm hoping with my daughter. She's likely only blocked and not scarred, so pancreatic function may resume for her as well.

 

[LittleLab4CF]

Knowitall,

Don't be too quick to dismiss Printer's answers. The leading CFGI researcher you would deride as a "moron" has advanced the science of treating GI problems in CF patients especially the pancreas's function. Bill and I amongst many others met this polymath who was encountering many undiagnosed CF patient with undiagnosed or under reported pancreatitis. This eventually took Dr. Freedman mostly out of general practice into the specialty of CFGI medicine.

He has quantified enzyme replacement scientifically for most pancreatic function scenarios and every conceivable CF GI issue has been or is being studied anew with his insightful and brilliant team. His contributions have eliminated nearly all the guess work or titrating enzymes, food, motility aids and effective therapies for DIOS and such.

Whether he is correct or wrong in his estimated opinion concerning Kalydeco and pancreatic function improvement, he's never to be easily dismissed. Were Dr. Freedman to answer this question in detail, a lot would go into his answer beyond a short statement. The nut of it all probably distills down to Printer's summary. The point is we are working with a small, widly desparate group that has been tested for a few years only and my guess is the pulmonary improvement hogged the analytical resources. A researcher quantifies results scientifically so possibly pancreatic improvement measures are statistically insignificant. When patients value any perceptable improvement, a little is so deeply encouraging in the currency of hope.

I don't understand your contribution. In an opening, inflammatory sentence you attacked the messenger and used vulgar conflation to discredit an extraordinary doctor, healer and a decent man. Is this an expression of your morbid fear of talent? Or do you maybe need to get a refuned for that anger management course? Although you may have been trying to insult Printer, you may have found a skew in Dr. Freedman's direct experience. I don't think he sees minors often if at all. I have never asked what age range he handles either so it's a guess.

LL

 

[kmhbeauty]

"knowitall" thinks he knows everything, and "thinks" that his opinions are superior above all others. His comments make me laugh, all you can do is pity him. So sick of reading your negative comments towards everything!

 

[Printer]

LL

When you look at the profile of this author of self inflated wisdom, you will find that he hasn't finished High School. When one is this ignorant of the facts it is easy to be a self proclaimed expert on all subjects known to mankind.

Bill