Leland Hein

Working with the Cystic Fibrosis Foundation (CFF) and the foundation’s research and treatment successes supported by CFF’s fundraising efforts is the thing that provides me the greatest satisfaction in life. www.cff.org

I first became aware of Cystic Fibrosis (CF) twenty-five years ago the only therapies available to those with CF were a limited set of antibiotics and artificial enzymes used to counter pancreatic insufficiency caused by the disease. These therapies were all that the CF doctors had in their tool kits. While these treatments were certainly better than no tools at all, these treatments only mitigated the damage caused by CF and only treated the symptoms of the disease. The life of a person with CF at the time was a constant battle with uncomfortable daily physical therapy, inhaled and oral antibiotics, and handfuls of artificial enzyme pills every time they ate. Two-week hospitalizations for intravenous antibiotic therapy every 6 to 18 months were the norm. In 1999 a person born with CF was more likely to pass away prior to graduating high school than wear a cap and gown, let alone live through adulthood.

The outlook for someone with CF today is much better. Thanks to the generosity of the foundation’s supporters and CF Foundation funded research, not only have the set of therapies used to treat the symptoms of the disease expanded and improved, but there is also now a highly effective therapy, Trikafta, available for about 90% of the CF patient population. Trikafta works at the cellular level to correct the basic cellular defect at the root of the disease. Because of Trikafta many CF patients have seen their Pulmonary Function Tests (PFT) score as much as double in just two-weeks; big difference between 35 and 70%. Many of the younger patients whose lung function had not begun to decline are now staying level because of Trikafta.

Even with supplemental digestive enzymes and diets with double or more the calories usually recommended, many CF patients have serious troubles keeping their weight up to a healthy level because of pancreatic insufficiency. Trikafta is effective enough that some CF patients report that for the first time in their lives that they now have to watch what they eat to prevent gaining too much weight.

However, the struggle to treat CF is not over! Approximately 10% of the CF population cannot be helped with Trikafta because they lack the specific gene mutations that Trikafta treats. The treatment of those within this 10% are still limited to treatment of the symptoms of cystic fibrosis and they are still in need of help.

Moreover, despite Trikafta being a “miracle” drug it does not cure the disease and has some at-times unpleasant side effects. Trikafta, along with the host of other CF specific therapies can always be improved and expanded. The CF Foundation continues to expand its research and the ways it can help the CF population.

I am happy to provide an update on my 36-year-old son with CF, Steven. Because of the treatments developed by the CF Foundation, Steven continues to do well in both his private and professional life.

Steven has been on what is without exaggeration the lifesaving Trikafta since it was approved. The effects of the medication were dramatic, and the root cause of the disease is now being successfully treated. Health wise, Steven continues to do well.

Steven is a wildlife biologist with U.S. Fish & Wildlife Service (USFWS). He is assigned to the Anchorage, Alaska USFWS office, though he works remotely from upstate New York, where he lives with his girlfriend/partner who is a UN diplomat. Steven’s area of interest is polar bears, sea otters, and walruses and the protection of these species’ habitat. Polar bears live in such remote areas and the walruses most live offshore, so living in NY is not a problem. None of this would have been possible without the lifesaving work of the Cystic Fibrosis Foundation.

When I write “lifesaving”, there is no hyperbola in my statement. Without the CF Foundation developed medication Trikafta, digestive therapies, and a host of other research funded by the CF Foundation, Steven and many other of those with CF would not be with us today. However, there still work to be done. There are still the 10% that Trikafta cannot help and Trikafta can be improved upon. The CF Foundation’s drug development pipeline is a thing of great hope. https://apps.cff.org/trials/pipeline

Please help me exceed my fundraising goal by donating to my Great Strides fundraising campaign. Your gift will help add tomorrows to the lives of people with cystic fibrosis by supporting life-saving research and medical progress. Your gift is 100-percent tax deductible. Please give me a call or e-mail me if you have any questions. 410-917-3954/ leland.hein@gmail.com

The Cystic Fibrosis Foundation continues to need your support. Cyber security restrictions will not allow me to imbed links into my contribution to the Great Strides website. Refer to WWW.CFF.org for additional information about cystic fibrosis, the CF Foundation, and the complete CF Foundation Drug Development Pipeline that lists past and future research successes funded by the supporters of the CF Foundation.

+ Intro to CF: https://www.cff.org/intro-cf

+ Drug Development Pipeline: https://www.cff.org/research-clinical-trials#drug-development-pipeline-clinical-trials

With your help, we can make CF = Cure Found!

Thank you,

Lee Hein
leland.hein@gmail.com

410-917-3954