One Month Out: Great Strides

May is a huge month for raising funds for the Cystic Fibrosis Foundation. It's during this month that the CFF hosts its single largest event; Great Strides. Great Strides as it's so literally states is when the foundation takes it's great stride to help fund a cure. I know many think, "yup, another way to give over-paid executives more money in their pockets" but I can first hand tell you how completely untrue that is for this foundation.

Over the past 30 years, as I can only speak for those that I've lived, the foundation has been the sole organization to place research dollars in the appropriate hands to raise the average life expectancy from 12-15 years of age to 41. In 30 years. CF was taught in medical school as a childhood disease, because most often they didn't reach teenage years or adulthood.

In addition, to prolonging the life expectancy here's a lovely list of a few milestones:

• 1982 The Foundation creates the Research Development Program, a network of research centers at leading universities and medical schools nationwide.
• 1988 The Foundation launches the Cystic Fibrosis Services Pharmacy.
• 1989 A team of Foundation-supported scientists discovers the defective CF gene and its protein product (CFTR), opening the door to understanding the disease at its most basic level.
• 1990 CF researchers achieve “proof of concept” that gene therapy (in the lab dish) is possible.
• 1993 Landmark gene therapy trial begins in people with CF.
• 1993 The Food and Drug Administration (FDA) approves Pulmozyme^®, which is proven to thin the tenacious, sticky mucus in the lungs and is the first drug developed specifically for CF. The time taken to develop Pulmozyme is less than half of the industry average.
• 1997 The Foundation establishes the Therapeutics Development Program.
• 1997 The FDA approves TOBI^®, the first aerosolized antibiotic designed for CF, which is proven to reduce hospital stays and improve lung function.
• 1998 Specialized clinical research centers are designated as the Foundation’s Therapeutics Development Network.
• 2000 Cystic Fibrosis Foundation Therapeutics (CFFT), a nonprofit research affiliate of the Foundation, is established to govern drug discovery and development efforts.
• 2000 Foundation-supported scientists map the entire genetic structure of the most common cause of CF lung infections — the Pseudomonas aeruginosa bacterium. Researchers can identify the function of specific genes and find ways to turn off the bad ones.
• 2002 A Foundation-supported study shows azithromycin improves CF lung health.
• 2003 Foundation-supported scientists at Structural GenomiX Inc., determine the three-dimensional structure of a portion of the CFTR protein, opening the door to more drug discovery opportunities.
• 2004 Foundation-supported studies in Australia and at the University of North Carolina show that hypertonic saline helps clear CF mucus. It is proven to improve lung function and reduce hospital stays, and becomes a therapeutic option.
• 2006 VX-770, an oral drug in development by Vertex Pharmaceuticals Inc., with support from the Foundation, enters clinical trials. VX-770 is one of the first compounds to attack the root cause of CF, and works at the cellular level to open chloride channels that do not function correctly in people with the disease.
• 2007 Vertex selects a second potential drug, VX-809, for development. Like VX-770, VX-809 addresses the underlying cause of CF, but it works by helping the defective CF protein move to its proper place in the cell.
• 2008 The Foundation and Vertex achieve a “proof of concept,” showing that it is possible to treat the root cause of CF. During Phase 2 studies of VX-770, trial participants, all of whom carry the G551D mutation of CF, show unprecedented improvements in key signs of the disease.
• 2010 The FDA approves a new inhaled antibiotic, Cayston^®(aztreonam for inhalation solution), to treat CF lung infections. Developed by Gilead Sciences Inc., Cayston offers a much-needed antibiotic alternative for CF patients who battle recurrent infections and develop resistance to existing antibiotics.
• 2011 The Foundation announces that Phase 3 clinical trials of ivacaftor (formerly VX-770) showed profound results. Those receiving the drug demonstrated the highest increase on a lung function test seen in any clinical trial of a CF drug. Vertex submits a New Drug Application to the FDA for ivacaftor under the trade name Kalydeco™.
• 2012 The FDA approves ivacaftor for people with the G551D mutation of CF ages 6 and older. The drug is the first to address the underlying cause of CF and opens exciting new doors to research and development that may lead to a cure for all people living with the disease.
• 2013 Vertex begins two large international Phase 3 trials of ivacaftor in combination with lumacaftor (formerly VX-809) in people with two copies of the most common CF mutation, F508del.
• 2014 The FDA approves ivacaftor as a single therapy to treat people ages 6 and older with one of eight additional rare CF mutations, and the drug continues to be evaluated in more patient groups.
• 2014 Results from Phase 3 studies of ivacaftor in combination with lumacaftor showed significant improvement in lung function and other key measures of CF in people with two copies of the F508del mutation of CF, ages 12 and older. Vertex has submitted a New Drug Application to the FDA, with possible approval in 2015.
• 2014 The Foundation maintains a robust pipeline of potential therapies that target the disease from every angle. The more drugs in the pipeline, the greater the odds of producing successful therapies and a cure for CF.

Pretty amazing accomplishments over the past 30 years. Over the next month I'd like to bring some knowledge about WHAT the foundation has actually accomplished with those donated funds.

~Doodlin'