Pcdf Annual Letter Nov 2009 Rev

Dear Supporters of the PCD Foundation: As another year concludes and another one begins, it is a fitting time to reflect on what we are thankful for, what we have achieved and the exceptional people who helped us get there. At the PCD Foundation, we are very grateful for the PCD community, the families, healthcare providers and researchers who are working every day to support one another and to find the cure for PCD. 2009 has been a very busy year for PCD. Here are some highlights: • • •

The PCDF partnered with the Genetic Disorders of Mucociliary Clearance Consortium (GDMCC) to refund this crucial research program for five more years The first usable mouse model of PCD is now available. This development will help expedite clinical trials for PCD Early successful genetic therapy tests on PCD in the laboratory have provided proof of potential for human therapy aimed at correcting the basic defect(s) in PCD

As important as these developments are, we still have before us a very complex puzzle from which many pieces are missing. These gaps can only be filled by the imagination, discoveries, and insights provided through the efforts of everyone committed to changing the course of PCD in this world. That is why the theme for the PCD Foundation in 2010 will be “Putting the Pieces Together”. We are going to invest in research, expanded outreach to patients and medical professionals, education & advocacy efforts to find the pieces to the PCD puzzle – to greatly improve the lives and opportunities of those affected, and to put an end to PCD one day in the not so distant future. ”To know even one life has breathed easier because you have lived - that is to have succeeded,” – Ralph Waldo Emerson

For most of us, breathing just happens. For some, it is a daily battle and race against the clock to lessen the damage of chronic lung disease. To take our momentum to the next level, it is critical that we seek the financial assistance of interested individuals to help us put those pieces together. This letter explains a little bit about PCD and how you can help us in 2010. What is PCD? Primary Ciliary Dyskinesia (PCD) is an inherited disorder of the structure and/or function of cilia. PCD affects a specific type of cilia that rely on coordinated motion to clean organ systems in the respiratory tract (lungs and sinuses), ears, reproductive organs and ventricles of the brain. People with PCD are born sick and face a lifetime of infections and daily therapy to prevent infections. The severity of the disease runs the gamut. For some PCD patients, the disease may progress rapidly causing early disability or even death. Others can live full and productive lives, but are never free from living with a chronic, progressive, life-altering condition. Why are Cilia so Important to Everyone? Everyone has cilia! These microscopic projections that resemble hair are found in nearly every cell of the body. We rely on cilia daily for crucial mechanical and sensory functions. When ciliary motion is impaired, organs are prone to repeated infections and inflammation. Only recently have

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scientists begun to recognize the foundational role of cilia in the development of a healthy human Sbeing. In fact, processes now known to be linked to cilia include cancer, obesity, diabetes, Solving the Puzzle of PCD, page 2. oncogenesis (cancer formation) and formation of tumors (fibrotic liver, spleen, kidney and breast disease) and skeletal and connective tissue formation (Marfan-like syndromes). Why is Your Support for PCD So Important? PCD patients are among a population of more than 25 million Americans who suffer from almost 7,000 separate rare and orphan disorders. A disease is rare if fewer than 200,000 people in the United States have it. While the National Institutes of Health budget rings in at $30 billion, only a small fraction benefits rare diseases like PCD. Drug companies are spending more money than ever on research and development, but few invest in research to cure rare diseases because they perceive there is little money to be made based on the relatively small target patient populations. This perception needs to change in the case of PCD. Adequate mucociliary clearance is an important feature of lung health for all people. Disorders of mucociliary clearance including asthma and chronic obstructive pulmonary disease (COPD) affect millions of people. PCD research provides invaluable insights into mucociliary clearance, insights that can be adapted to help millions of individuals with respiratory disease. Additionally, there is increasing evidence of overlap in disorders that affect moving cilia and those that affect sensing cilia. Sensing or non-motile ciliary function has now been implicated in a large and diverse set of human diseases, including polycystic kidney disease, spina bifida, tumor formation, skeletal disorders and a number of very rare syndromes. Together, these diseases are called ciliopathies and research into PCD and other ciliopathies could potentially provide important information for the treatment of a wide variety of human diseases. How You Can Help Us “Put the Pieces Together” in 2010 As a tax-exempt organization, we rely on individual donations to fund our programs (detailed program descriptions are available from the PCD Foundation). Statistically, non-profits receive 80% of their funding from private donors. This can be a challenge for groups like the PCD Foundation that serve a small population. However, to accomplish the goals of the Foundation we need the assistance of every PCD family and of their circle of friends. As people who care about individuals with PCD, we are a small group in a unique position to provide answers through research that could potentially help many people, in addition to improving the quality of life for people with PCD. The biggest threat to our continued progress is lack of funding to operate our important programs. The PCD Foundation is a 501(c)(3) tax-exempt organization and all donations made to the PCD Foundation are deductible on your federal tax return. Please consider including the PCD Foundation in your end of year giving plans. It will be money well spent! Every donation makes a difference, regardless of size. Thank you in advance for your commitment to support the efforts of the PCD Foundation to continue making a difference in the lives of the PCD community and beyond. Sincerely, John Carson PhD, President

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Michele Manion, Executive Director

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Do You Hold the Missing Piece? Help us Solve the

Puzzle of PCD!

Please return this page with your tax-deductible donation! Checks can be made to the PCD Foundation and mailed to: PCD Foundation 10137 Portland Ave South Minneapolis, MN 55420-5050 If you would prefer to make a donation by credit card, you can use our secure PayPal online donation option: http://www.pcdfoundation.org/paypalcontribution.htm.

Tax receipts will be provided. □ YES! I want to help fund research and education in PCD! Enclosed is my taxdeductible donation in the amount of $________. Please send thank you note and tax receipt to: Name: ________________________________________________________ Address 1: ______________________________________________________ Address 2: ______________________________________________________ City: ___________________________________________________________ State or Province: __________________ Zip/Postal Code: ________________ Please add me to The Beat e-newsletter list: _____________________________

PLEASE SHARE THIS LETTER WITH YOUR FRIENDS AND FAMILY!

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