Great Strides T-Shirts and Other Official Business

We're going to order our Great Strides T-shirts soon and I'm so excited. Last year was such a whirlwind, as the twins were only a few weeks old at the time of the walk and we were still learning all about this thing called CF. I'm trying to figure out how many shirts to order, and here's where I need your help. We have decided that everyone who is signed up to walk and is raising money for us will get a complimentary t-shirt . We would also like to offer t-shirts to your family members or friends if they will be walking with you so that we can be identified as one unified team for Drew. We understand that not everyone is able to raise money, so we are going to offer the t-shirts to walkers who do not raise any money for a small fee (probably about $10 to cover the cost of the shirt) and all kids will get a shirt for free (while supplies last).

I am trying to figure out how many shirts I need to order, so I'm asking that you register yourself and your spouse/children/neighbors/friends or whoever will be attending the walk, regardless of if they plan to raise $ or not, so that I know how many shirts I will need. It would mean the world to us to have the support of all of our family and friends and neighbors and friends of friends and everyone who cares about us and about Drew, to have you there with us the day of the walk. For all of my Philadelphia friends & family, the other benefit to registering to walk is that all walkers registered by April 1 will receive free admission to the zoo the day of the walk (the walk actually takes place inside the zoo). If you do not register by April 1, you will have to pay $15 admission to the zoo for the day. This goes for all of your family members, including all children over the age of 1. So it might seem silly, but please register anyone who will be joining you on walk day so that you can get into the zoo for free and you can get a team t-shirt from us.

Here are the links to the cities we are walking in. There is no obligation to raise money (although we'd love for you to!) if you officially register as a walker. It will simply help me to gauge how many t-shirts I need to order. I'm ordering on April 1, so please register before then.

Cincinnati, OH - May 21
Philadephia, PA - May 21
Honolulu, HI - May 28
New London, CT - May 14
Charleston, SC - May 14
San Francisco - May 21

Next on the list, we're going back to Tobi. It was our decision and I think we made the right one since there's really no downside. Tobi only treats pseudomonas, and the last culture that was done on Drew was taken while he was still on the Tobi, but it came back negative for pseudomonas. That was good news, sort of, because it told us the Tobi was working to get rid of that bacteria. However, pseudomonas apparently is really good at hiding in your lower airways and doesn't always show up on oral cultures. The docs and us were both a little concerned that we would stop the Tobi and not cycle it (its usually given on a 28 day on/off cycle on an ongoing basis for people who are colonized with the bacteria) and the bacteria would be secretly living way deep down in his lower airways doing the damage that pseudomonas does. So we decided to do a second cycle of Tobi which will start on April 2, twenty-eight days after we ended the last 28 days run, and will end a couple of weeks before his next bronchoscopy. This is probably a lot of confusing jib-jab, but basically we're going to err on the side of caution and treat aggressively trying to eradicate the miserableness that is pseudomonas, lest it haunt us on an ongoing basis. I also should note that its not terribly common for CF kids to get as many bronchoscopy's as Drew gets. He mostly gets them to see how his airway abnormalities (tracheal malacia and bronchial malacia) are changing and hopefully improving, and how much they are really hurting his ability to appropriately clear his airways. Hopefully, as he gets older and outgrows these malacia problems, the bronch's will become less and less. By then though there will hopefully be a cure, which leads me right into my next topic...

There was another article giving some more media coverage of the new CF drug which will hopefully help Drew and thousands of other people. Here's what Business Week had to say about VX-770:

Vertex's Treatment for Cystic Fibrosis

Its VX-770 is the first pill designed to repair an inherited flaw based on the discovery of the gene mutation that causes the disease

By Meg Tirrell

Then in December 2009, Schaller started taking VX-770, an experimental drug from Vertex Pharmaceuticals (VRTX). Now the self-described exercise enthusiast says she can run 10 to 12 miles with barely any breathing problems. "This past year has been the coolest of my life," says Schaller, who runs the Rock CF Foundation, a nonprofit near Detroit that helps fund research on the disease, which afflicts 30,000 Americans. "It was a whole new feeling that I hadn't felt since I was a little kid."For the better part of two decades, Emily Schaller could barely take a deep breath without lapsing into coughing fits. At the age of 18 months, Schaller was diagnosed with cystic fibrosis, a hereditary disease that chokes the airways with sticky mucus, making victims susceptible to infections and other problems that often kill them before they turn 40. Schaller, now 29, says doctors told her parents, "Enjoy her, because she probably won't live long enough to graduate from high school." By age 13, she was suffering the full effects of the disease, which required her to take various medications and wear a vest that vibrates to loosen her congested lungs.

Vertex's drug is in the third and final stage of testing required for U.S. Food and Drug Administration approval. In a study released in February, Vertex reported that the drug increased breathing function by 10.5 percent over a year, reduced patients' need for antibiotics, and led to an average weight gain of seven pounds. "We were euphoric," says Robert Beall, chief executive officer of the Cystic Fibrosis Foundation in Bethesda, Md., which helped fund development of the Vertex drug. The results "suggest we've already added five years of life to these patients."

The CF gene, discovered in 1989, was one of the first found using new gene-hunting techniques that have led to better understanding of the biology of dozens of illnesses. The genetic research is helping scientists close in on treatments for cancer, diabetes, and other disorders that target glitches that occur during an individual's lifetime. VX-770 is the first pill designed to repair an inherited flaw based on the discovery of the mutation that causes the disease.

Cystic fibrosis is caused by mutations to a gene that, when working properly, creates proteins to help transport fluid in and out of cells. When the gene is defective and the proteins don't work, chloride, a component of salt, can't travel freely across cell membranes. That limits the flow of fluid outside the cell, causing mucus there to dry up and clog airways, allowing bacteria to gather.

VX-770, designed for a mutation carried by 4 to 5 percent of CF patients, enhances the function of the protein, creating a gateway that allows chloride to pass through the cell wall. About 90 percent of people with the disease, though, also suffer from a thornier problem: The protein never makes it to the cell wall. So Vertex is developing a second drug—VX-809—that helps transport the protein to the membrane. "To have optimal benefit, you need two compounds," says Eric Olson, head of Vertex's cystic fibrosis unit. "One to get the protein to the surface, and one to boost its activity once it's out there."

VX-809 is still in a so-called Phase II test as is the combined treatment. While VX-770 could win approval as early as 2012, any formula with both drugs likely won't be available for several years. "The combination therapy down the line is going to be the real driver," says Brian Skorney, an analyst with brokerage ThinkEquity in New York. Such a drug, he says, could eventually generate $1 billion a year in sales. On Feb. 23, when Vertex reported the VX-770 data, the company's Nasdaq-traded shares jumped 15 percent.

The medicines are the fruit of more than a decade of research. After the CF gene was discovered, scientists initially pursued gene therapy, trying to introduce a normal gene into cells to replace the faulty one. When that didn't work, the focus shifted to medicines such as VX-770 that could simply help defective proteins function properly. The Cystic Fibrosis Foundation asked drugmakers if they would screen compounds in their chemical libraries to determine whether one might affect the protein that helps move fluids in and out of cells.

Aurora Biosciences in San Diego agreed to test thousands of chemicals, eventually yielding compounds that led to the Vertex therapies. Vertex bought Aurora in 2001 for about $600 million and maintained the relationship with the CF Foundation, which supplied $75 million in funding for the program. "Discovering the genetic cause of a disease can lead you toward a therapy you never would have thought of otherwise," says Francis Collins, one of the CF gene's discoverers and now director of the National Institutes of Health.

The cystic fibrosis program, once an uncertain part of Vertex's future after the Aurora acquisition, has transformed the company into a diversified drugmaker, says Phil Nadeau, an analyst with Cowen & Co. in New York. Vertex is awaiting regulatory approval for Telaprevir, a medicine for hepatitis C that Nadeau estimates could draw more than $4 billion in annual revenue. "Now people see [Vertex] as more diversified," Nadeau says. "VX-770 is a second pillar the company can stand on."

The bottom line: If approved, Vertex's new cystic fibrosis pill will be the first treatment developed from genetic research for an inherited mutation.

If you missed my post on why this drug is good news for us, CLICK HERE to check it out.

That's about all the energy I have for this evening. It was a great week in our house. The nice weather allowed us to get outside for many hours on many occasions this week which was literally a breath of fresh air. Hope you'll sign up to walk with us!