CFF News

FDA Advisory Panel Rules in Favor of AZLI

As we enter the holiday season, I am pleased to share with you some very positive news. Yesterday, a potential CF drug called aztreonam lysine for inhalation was recommended for approval by an advisory panel to the U.S. Food and Drug Administration (FDA).

Aztreonam lysine for inhalation (AZLI) is being developed by Gilead Sciences, Inc with support from Cystic Fibrosis Foundation Therapeutics (CFFT). The drug would offer a much-needed antibiotic alternative for cystic fibrosis patients who battle recurrent lung infections and often develop resistance to existing antibiotics.

In the fall of 2007, results of the final Phase 3 trials were presented to the FDA in the form of a New Drug Application. At that time, unfortunately, a division of the FDA decided not to issue an approval letter for a number of reasons, which we believed were not sufficient to withhold approval. Working with Gilead we communicated with the FDA urging reconsideration.

Finally, in the spring of this year, the FDA reluctantly agreed to convene an advisory committee to review and provide non-binding recommendations to the staff. As you will see in the two links below, the committee voted 15−2 in favor of approving the New Drug Application! This is a major milestone vote for our patients in many ways.

The FDA staff has 60 days to make their ultimate decision, but with a 15−2 vote, it will be difficult for them to go against the panel's decision. More importantly, a strong message was sent by the committee to the FDA staff that in the case of CF, where few options are available, the bar for approval needs to be modified when the safety of the drug is not an issue. Clearly this was critical for numerous sponsors of new drugs to hear (many of whom were in attendance).

In a meeting of this kind, the sponsor (Gilead) presents its data, the FDA states its concerns, and then a public hearing is held. We organized this hearing and we had three compelling presentations by Drs. Bruce Marshall (CF Foundation) and Patrick Flume (Medical University of South Carolina), as well as by Beth Sufian, an adult with CF who manages our CF Legal Hotline. Their messages were powerful, and stressed the crucial need for new antibiotics against this insidious disease. Beth brought the audience to tears as she described the impact of this drug on her life and her future. She challenged the committee on why this drug was not yet approved in United States, while it was already approved in Canada and Europe. Dr. Frank Accurso likewise reinforced these points when he spoke on behalf of Gilead. They all did a superb job in telling our story.

What is exciting is, if − or better yet, when − this antibiotic is approved, it will be the first drug whose initial funding came from the CF Foundation's Therapeutics Development Program that receives FDA approval. Even more remarkable is that our drug development pipeline has dozens of promising potential drugs, which could dramatically improve and extend the lives of those with CF.

While we still need approval, yesterday's vote was a major milestone. It is because of you that we can make this kind of progress. Thank you for all you are doing to help advance our mission.

Best wishes for the holiday - this decision should make a happier holiday for all those affected by CF.

Sincerely,

The CF Foundation

What a Wonderful Christmas!

I can't even begin to express how wonderful this Christmas has been. It very well could be the best Christmas I have ever had. On Christmas Eve we attended a Cantata and Bell Choir at the Baptist Church and then midnight mass at St. Mary's. I was so overwhelmed with joy that I wanted to shout out to everyone MERRY CHRISTMAS!!! What a thrill to be able to stay up late enough to attend midnight mass. Our priest, Fr. Greg, made the mass so special and ended by playing his accordion to Silent Night, what fun!

We always celebrate Christmas Eve with Scott's family, so we spent the afternoon at Mom Matthews' house where we had a wonderful dinner and just enjoyed time together. Scott's grandmother who was in the hospital the last couple of weeks was home and able to be with us which made it even more special. She turned 90 on Saturday, what a blessing she is to our family!

Christmas morning was spent at our house with my parents. What fun to see what Santa brought and to enjoy the morning together. Scott and I fixed a big Christmas breakfast and then we hung out playing Wii for the afternoon. That night we went to my sister-in-law, Leslie's, house for dinner.

What a perfect Christmas! We have received so many miracles and blessings and I felt them so deeply these past few days. God is so good and generous!

All my love! Nancy

Remember the True Meaning of Christmas

I can't begin to tell you how many times I have heard people say, "I can't wait until Christmas is OVER!" What a sad thing to say! Why do we make ourselves so overwhelmed this time of year that instead of enjoying this magical season we wish it was over?

Please don't wish away Christmas. Feel free to cut back on the list of things you feel you must do, or lower the amount you feel expected to spend, heck, even eliminate the cards, but don't wish Christmas away.

Try to remember that Christmas is not about gifts, cookies, parties, and spending, Christmas is about the celebration of the birth of our Lord. It is the time when God blessed us by sending His only son to be born as a human and live among us, to begin the journey toward His ultimate death on the cross to save us from our sins. This is a time when we should be just enjoying each other. Laughing together, sitting quietly by the fire, caroling, playing, and rejoicing.

I can honestly say I have never felt as joyous as I do right now. Every event we attend, every gift that I give, every cookie that I've baked has been full of fun, love, and appreciation for the life I have been given. When you start feeling that overwhelmed feeling, stop, take a breath, and remember what Christmas is REALLY all about!

May God bless you all! Nancy

We celebrated Christmas with Scott's Family on Saturday, what a great day!

Wellsphere Health Blogger Awards 2009

Hi Nancy,

CONGRATULATIONS on your success in the People's Health Blogger Awards Competition — your readers have spoken, and you've been chosen as the Top Blogger in the Cystic Fibrosis community. What a great tribute to you, and a wonderful recognition for your blog!

The competition was a huge success, with hundreds of the best health bloggers competing and thousands of votes cast. These awards are recognition by your readers for the contribution you are making through your blog, so you should feel very proud of your achievement!

Cystic Fibrosis

Congratulations again, and keep up the great work with your blog in the coming year!

Cheers, Hua--David HuaDirector of Blogger Networks

Hannah's Holiday Performances

Hannah's Duet at her Theatre Performance

Performance at Tops

All Went Well in Pittsburgh!

Dear friends,

What a crazy couple of days. We headed to Pittsburgh on Monday so I could have a chest X-ray, blood work, and repeat my pulmonary function tests. Are you ready for this? My lung functions were at 102%!!!!! Can you even believe it, I am actually above average. So, in one year's time my lung functions have risen from about 24% to 102%, God is SO GOOD!!!

We were able to visit Cammi and her family for the last time in Pittsburgh, they will be heading home to California over the weekend. I will miss her terribly, but am so glad she is finally able to go home. And, we finally remembered to get some pictures together. We have gotten so caught up in our visits the last couple of trips that we totally forgot to get pictures, how frustrating!










I had my bronch yesterday morning and all went well. They did not have to do the ballooning, which was wonderful! So despite the fact that they used general anesthetic, we were on the road headed home by 11:30 giving me plenty of time to prepare for Hannah's theatre concert. I'll post some pictures of that later.



I got another wonderful piece of news yesterday, my friend Bill who recently had his double lung transplant was discharged from the hospital yesterday. What a Merry Christmas this will be for them! Please continue to pray for his recovery, he still has some issues they are trying to work out.

Finally, thank you all so much for your votes for the Wellsphere Health Blogger awards. As of last night my blog was in 5th place. The final results won't be released until Friday, but I certainly couldn't have moved into the top 10 without all of you! Thank you, Thank you, Thank you!!!!!

My parents fly in tomorrow, so I have lots to do. Hope you are getting your hearts ready for this most blessed season of all! Love you! Nancy

Health Blogger Award

Dear friends,

Today is the last day to vote for the Wellsphere Health Blogger awards. If you haven't voted yet, PLEASE take a moment and click on the vote for me link to the right, I would so appreciate your support!

We are headed to Pittsburgh today for another bronch. This one will be just like the last, they will use general anesthetic and intubate me so they can balloon the lung opening that has been closing up. As you know, I had some difficulties after the last one, so please pray that all goes well tomorrow!

Thank you all so much! Nancy

I wanted to share the card my sister-in-law gave me for my 1 year re-birthday:
" Just living is not enough said the bumblebee."

and her very insightful inner words:

"Last year you were just living. This year - you are Alive!

Make sure you aren't Just Living this year!

Hannah's Performance at Tops

Hannah's theater group performed at our local Tops today for Toys for Tots. They did an amazing job! I will post some video later. Hannah and I had a nice visit with Santa and Mrs. Claus. What a great way to get in the holiday spirit!

Hannah & her cousin Jacinda

Hannah & her friends Haley & Anna

Today is my One Year Re-Birth Day!

Sing to the Mountains

Sing to the Sea

Raise your voices

Lift your hearts

THIS is the Day the Lord has made

Let us rejoice and be glad!

Today IS the day the Lord has made; He saved me from death and raised me back to life. He has blessed me beyond all comprehension and I Glorify His name!

God bless my donor's family on this anniversary of her death, may they some day understand the miracles she helped to create and may she know total peace and happiness with God in heaven above!

Thank you to those of you who have called, written, and sent cards. A special thanks to Blake and Britney Blomquist, Sharon Wiley, and my mom for donating gifts to Toys for Tots in celebration of this joyous day!

A special Happy Re-Birth Day to my dear friend Jamie who is celebrating her 2nd year post-transplant!!!!

One of the Band's songs

Be sure to pause the music player before you watch this.

Hannah's First Band Concert

We enjoyed a wonderful concert last night at Hannah's school. The elementary band director, Mr. Crane did a wonderful job with his 90+ 5th and 6th grade band members. It is so nice to see how all of the practicing has paid off!

Hannah and her friend Courtney before the concert

It's Beginning to Look A Lot Like Christmas!

Wow, how do I describe the way I feel right now. I was able to decorate my own house for the first time in several years. I will be hosting Christmas at my house, I can sing along with the Christmas carols on the radio, I was able to shop in actual stores, not only online, for Christmas presents, I will be able to make cookies, attend concerts, and enjoy my family without the worries of illness or death clouding my vision. All of this is so incredible! As I approach this momentous anniversary, I feel nothing but happiness, wonder, and extreme thankfulness. Christmas IS for miracles!

crafts we made this weekend
Sweet Pea thinks she might like to fit in a special box for wrapping.

My love to you all! Nancy

Help Me Celebrate my One Year Re-Birth Day!

Dear friends,

My one year Re-Birth Day is quickly approaching. On December 10th I will celebrate one year with new lungs. It is so hard to believe it has been a year. What a roller coaster! I have so much to celebrate this year and I was hoping you would join me in the celebration.

You may remember last Christmas I donated toys in honor of many of my family members to Toys for Tots. This is a very important thing for me, knowing that every child will wake up Christmas morning with something special under the tree. I have already made my purchases, and would like to ask you to do the same. Would you honor my miracle of new lungs and my one year anniversary by donating a new toy to Toys for Tots for me? If you are willing, please let me know what you donated and I will share all of the gifts you purchased in my honor next week on my special day.

Please join me in making Christmas a little more special for children in need.

God bless you all! Nancy

Give the Gift of Hope with Holiday DVDs

Holiday Classics to Benefit the Cystic Fibrosis Foundation!

The Cystic Fibrosis Foundation and CVS Pharmacy has worked together this holiday season to bring you Classic Holiday DVDs and give back the CF Foundation!

Here's how it works: now through December 25th, buy one of three holiday classics from Warner Home Videos -- "How the Grinch Stole Christmas," " The Year Without A Santa Claus," and "Jack Frost" - at any of the more than 6,900 CVS/pharmacy stores nationwide and $5 dollars of the purchase will benefit the Cystic Fibrosis Foundation.

The videos are in a specially designed floor display in the holiday aisle of each store, but in some cases are included in a permanent DVD rack located near the checkout counters. All three of the above titles have been labeled with a sticker to identify them as part of this promotion.

Special Visitors

We had some special visitors on Sunday. Our friends the Shuckras came for a visit. Amy and I were close friends from childhood and she was one of my maids of honor in my wedding. The last time they visited Olean, Hannah was a newborn. Over the years we have re-connected during the summers in Jackson, but the last time we saw them was in 2006.

Jeff's parents live in Rochester, so this year they spent Thanksgiving there and since it is only a 2 hour drive, we got the pleasure of a visit. I couldn't get over how much their boys, Jack and Ben have grown. We had such a nice visit. Hannah had a great time entertaining the boys, so the adults could catch up.

Thank you, Amy and Jeff, for taking time away from your family to visit us. What a wonderful day we had with you!

Hannah with the Shuckras and Blomquists summer 2006

2009
Hannah (10), Jack (7), & Ben (5)

Amy & Me

Our Families!

All my love! Nancy

P.S. Amy brought me a great new cook book, so be looking for some new recipes on my cooking for fun with Nancy blog.