B.cepacia

Caleb's clinic just called and said that his last culture which was about a month ago grew B. Cepacia!!! They sent it to a few different places to confirm before calling me... They said out of the 5 different types this was the better of them.......


I would rather him not have any! I feel like I am going to puke! I thought this was something that they cultured when they were older.... NOT at 7!


Melissa

Pray for Stellan

If you get a moment please say a prayer for baby stellan and his family. He is in the NICU having heart problems (SVT). They are having a hard time getting his heart rate back to normal. His button is to the right.

Until next time.........

Sorry!

Sorry... I really didnt fall off the face of the earth! Having computer issues is no fun! That on top of sick kids and meds galore......... You know that drill. Wyatt has still been having a rough time! He just finished his 4th antibiotic for his on-going ear infection issue. Its just so weird. He has never had a ear infection until about 2.5 months ago and its been down hill ever since. I brought him for a re-check today and his ped said the left looked good but the right was red and full of fluid! She is going to give him 2 weeks and check again. If its still full of fluid or it gets infected again she is going to recommend tubes. She said the right ear is so bad she isnt sure he can fully *hear* out of it. I guess id rather go with the tubes than keep dealing with a miserable kid constantly on antibiotics! Caleb is doing good (knock on wood). He had a cold a few weeks back but with some antibiotics and increased treatments we were able to jiggle it out of him :) Dylan is also over his cold so hopefully they are all on the mend. Its been nice around here lately its so nice to open the windows and air out!


Until next time

VX-809

CF Foundation Announces Initiation of Phase 2 Trial of VX-809
March 25, 2009 The Cystic Fibrosis Foundation announced today the initiation of a Phase 2a clinical trial for VX-809, a compound designed to treat the basic defect in cystic fibrosis—a faulty gene and its protein product, called CFTR. Developed by Vertex Pharmaceuticals Incorporated, in collaboration with the Foundation, VX-809 is known as a “corrector” and aims to move defective CFTR protein to its proper place in the cell. The protein is then poised to move chloride out of the cell and potentially allow it to function as a healthy cell would. The Phase 2a trial is designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with CF. In addition to assessing safety, the trial will also evaluate whether VX-809 affects key measures of how well the protein is functioning. The trial is expected to enroll approximately 90 patients with the Delta F508 mutation of CF, the most common mutation in CF patients. "Today, CF treatment involves the use of multiple drugs that address the symptoms of CF, however no therapy currently exists that targets the underlying cause of this disease,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “We are encouraged by Vertex’s rapid progress in advancing two novel therapies aimed at correcting the basic defect in CF. We believe that VX-770, which is expected to enter registration studies in the first half of this year, and VX-809, which today entered Phase 2 clinical development, have the potential to play a significant role in changing the future course of this disease for people with CF.” "The advancement of VX-809 into Phase 2 development demonstrates our commitment to improve care for patients with CF," said Freda Lewis-Hall, M.D., executive vice president, medicines development, and chief medical officer of Vertex. "This Phase 2a trial is designed primarily to provide a safety, tolerability and pharmacokinetic evaluation for VX-809. Any additional signals observed in this trial, including changes in sweat chloride and nasal potential difference, that indicate VX-809 has an effect on measures of CFTR function may be highly informative and encouraging for planning future clinical trials with VX-809."
VX-809 was discovered as part of a collaboration between Vertex Pharmaceuticals and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Foundation. Initiated in 1998, the Foundation has invested $76 million in CF research and development efforts with Vertex.
Read Vertex's press release.

Liprotamase

CF Foundation Finds Collaborator to Ensure Development of Important Pancreatic Enzyme
March 25, 2009
The CF Foundation announced today it will collaborate with a pharmaceutical company to ensure development of an important porcine-free pancreatic enzyme replacement therapy.
In a Foundation investment worth up to $3 million, Alnara Pharmaceuticals Inc., a Massachusetts-based pharmaceutical company, will complete the ongoing Phase III long-term safety study of liprotamase. Following completion of the study, Alnara will complete clinical and regulatory activities needed to file a New Drug Application with the U.S. Food and Drug Administration. The company is committed to bringing this important product to patients with CF.
Liprotamase was previously in development with Altus Pharmaceuticals Inc. and was known as ALTU-135 and Trizytek.
“The Cystic Fibrosis Foundation is excited to enter into a licensing agreement with Alnara for the development of liprotamase, which has the potential to be the first porcine-free pancreatic enzyme therapy for CF patients with pancreatic insufficiency,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We look forward to working with Alnara to quickly finish the development of liprotamase and bring this promising new treatment option to patients with cystic fibrosis.”
The Development History of Liprotamase
Liprotamase, which has been in development since 2002, was originally discovered and tested by Altus Pharmaceuticals. Unfortunately, due to financial difficulties, Altus discontinued development in January 2009. Unwilling to let this important therapy languish, the Foundation retrieved rights to the potential drug from Altus, maintained its clinical progress and searched for a company to see the enzyme through the approval process.
The Foundation selected Alnara because of its unique experience with the pancreatic enzyme therapies: two key members of management were involved in developing the original product at Altus. Alnara also plans to develop a pediatric liquid form of the pancreatic enzyme.
Cystic Fibrosis and Pancreatic Insufficiency
Pancreatic insufficiency occurs in approximately 90 percent of people with cystic fibrosis and is treated with pancreatic enzymes that aid digestion and improve growth. Liprotamase is unique and important because, unlike other pancreatic enzymes currently available, it is derived from non-porcine sources.
In addition, liprotamase offers potential advantages over the current standard of care. Today, people with CF must take multiple capsules—sometimes four or five—with every meal or snack. In contrast, with liprotamase, patients can take one small capsule with every meal or snack.
Read Alnara’s press release.

Its late........

It is too late right now so part 2 will be posted tomorrow!

About Us........

My Name is Melissa and I have been married to jim for almost 10 yrs.....

We have a son dylan who is 1o and does NOT have cystic fibrosis.......

Our Middle son Caleb was diagnosed with Cystic fibrosis at 7 months old....... 7 yrs ago today....

I remember our wonderful ped. saying she just wanted to rule it out, after caleb had been sick off and on for several months................

I also remember my mom telling me that there was no way he had cf..... and when kids did have it they had to be pounded on several times a day.................

I remember the day caleb had his sweat test the person that did it said out of all the tests she had done no one had ever tested positive................

I remember the day caleb had his sweat test we had just gotten home (only 40 mins after the first test) I was called to come back in for another sweat test................

I knew in MY HEART SOMETHING WAS WRONG!!!

I brought caleb back in and they re-assured me that I was only there due to their error!

Another hr passed and the Ped's office called me and wanted me to come in ASAP to talk about the test results.....................

I demanded to hear the results over the phone!! I knew something was wrong and didnt want to drive an hr worrying......

After a few mins of hesitation they told me that calebs sweat test came back POSITIVE for Cystic fibroisis..........

It had been a long time since I saw my mom and gram cry.......................

Expecially MY mom!

We were told a few facts and pretty much sent on our way.....................

Later that evening I got a call from calebs ped (who was on vacation)

She was stunned with the results.........

She just couldnt believe it.................

No one could..................

Caleb was started on meds which he just wouldnt take................. NO matter what!!!!!!!!

A few weeks later he still wasnt getting better and he was rolling his eyes.............

It was found that he had a very low sodium level and was immediately transported to barbara bush childrens hospital.....................

He spent a week there getting his levels back to normal................... and for me it was learning more about his diagnosis.............

It took him a few yrs to get past the gag reflux in order for him to take his enzymes!

For him it came down to swallowing a piece of Hubba Bubba bubble gum!!!

So far he has only been in the hospital that one time.............

He is now 7 1/2 and on the following...........

2 creon 20.....

2 puffs of flovent twice daily

albuterol twice daily

azithromicin M/W/F

Cefzil twice daily

Pulmozyme once daily

ADEK vitamins...

culturelle

Chest PT twice daily

prevacid once a day

2-3 pediasure daily

High calorie diet.........................................


Until next time...............

Melissa