Sunday, January 17, 2010

Cystic Fibrosis History and Timeline

Note from Ronnie: I cannot guarantee the accuracy of this timeline nor the information presented as I did not write it nor do the research for it. I did however find it very interesting and wanted to share it with you guys. If you see any distorted "facts" or major discrepancies please let me know so I can be further educated on the history of CF. Thanks!

Middle Ages
- In medieval folklore, infants with salty skin, a symptom of cystic fibrosis, are considered "bewitched" because they routinely die an early death. "Woe to that child which kissed on the forehead tastes salty. He is bewitched and will soon die." This bit of folklore, handed down through the ages, is an early description of the disease now known as cystic fibrosis. This is one of the earliest references to cystic fibrosis. Folklore from the Middle Ages, from Germany and Switzerland, made this reference. The earliest documented reference in the literature is from 1606, Alonso y de los Ruyzes de Fonteca, professor of medicine in Spain, wrote that it was known that the fingers taste salty after rubbing the forehead of the bewitched child.' This is a clear reference to the fact that children with cystic fibrosis have very salty sweat, due to the abnormal protein manufactured by the cystic fibrosis gene. This protein regulates the movement of sodium and chloride from the body's cells. Sodium and chloride are two components of salt. The sweat glands of a person with cystic fibrosis secrete too much salt, and the person tastes salty. This is also a reference to the fact that in earlier days, people with cystic fibrosis did not live past infancy.

1936 - Fanconi refers to the previously nameless condition as "cystic fibrosis with bronchiectasis."

1938 - Andersen of Columbia University develops the first comprehensive description of cystic fibrosis symptoms. CF also named Mucovicidosis.

1949 - Lowe establishes that cystic fibrosis is a recessive genetic disorder.

1953 - After observing excessive dehydration of cystic fibrosis patients during a New York City heat wave, di Sant' Agnese of Columbia University formally reports to the American Pediatric Society that CF patients secrete excessive amounts of salt in their sweat. This observation leads to development of the sweat test as a diagnostic standard for cystic fibrosis.

1955 - A group of volunteers, including parents of children with CF and physicians meet to form the CF Foundation, headquartered in Philadelphia; among the group is Milton Graub, M.D., a pediatrician and father of two children with CF, who later becomes the CF Foundation’s third president and who remains active in the CF Foundation today; life expectancy for people with CF is 5 years old.

1961 - The CF Foundation establishes the first CF care centers; CF Foundation hosts first conference of CF care center directors.

1962 - Additional CF centers established, bringing total to 30. Predicted median survival increases to about 10 years.

1965 - CF Foundation begins clinical fellowship program; George Frankel, a CF Foundation founder, establishes Guidance, Action and Projection conferences, a fellowship program to bring basic scientists into the field of CF research.

1966 - CF Foundation establishes Patient Data Registry, which tracks the histories of patients treated at CF care centers.

1968 - CF Foundation hosts first National/International Medical Conference.

1972 - First national seminar on needs of young adults with CF held by CF Foundation.

1978 - Headquarters moves to Washington, D.C. to be close to the federal government and the National Institutes of Health (NIH); first nationwide fund-raising event held, called Bowl for Breath, nearly doubling the fund-raising dollars for CF. The number of Cystic Fibrosis Foundation-accredited care centers now total 125.

Early 1980's - Researchers associate organ damage caused by CF with a malfunction of the epithelial tissue.

1980 - Robert J. Beall, Ph.D. comes to the CF Foundation as medical director from NIH.

1981 - Satellite CF care centers for adults were established for the first time; life expectancy reaches 20 years.

1982 - The CF Foundation creates the Research Development Program (RDP) to encourage leading academic and scientific institutions to establish centers of excellence in CF research; Doris Tulcin, Chairman Emeritus of CF Foundation, launches a capital campaign—the first of its kind by a voluntary health agency—to raise $15 million to support the creation of the RDP; Robert K. Dresing becomes volunteer president of the CF Foundation, later becoming the first president and CEO.

1983 - Scientists show that cells lining the lungs fail to properly move chloride into the airways; the Orphan Drug Act is signed into law and provides companies with financial incentives for developing drugs for rare diseases like CF; Frank Deford, sportswriter and novelist, writes the book, Alex: The Life of a Child about his daughter who died from CF at the age of 8; Deford becomes Chairman of the CF Foundation’s Board of Trustees.

1984 - American Airlines, Outstanding Corporate Partner of the CF Foundation, hosts the first Celebrity Ski event to benefit the CF Foundation.

1985 - Life expectancy reaches 25 years.

1986 - The movie, “Alex: The Life of a Child” appears on ABC putting CF in the national spotlight; CF Foundation tops $20 million in fund-raising.

1988 - CF Foundation establishes Cystic Fibrosis Services—a specialty pharmacy—to provide availability, access and assistance with the complex insurance issues faced in obtaining CF medications.

1989 - Francis Collins, M.D., Ph.D., John Riordian, M.D., and Lap-Chee Tsui, Ph.D. discover the CF gene with support from the CF Foundation’s RDP; CF Foundation hosts first GREAT STRIDES walk-a-thon, now a $20 million+ annual fund-raising event. A Team led by Tsui and Riordan of the Hospital for Sick Children in Toronto discover the gene responsible for CF and name its protein product cystic fibrosis transmembrane conductance regulator (CFTR). The gene is mapped to chromosome 7q.

1990 - CF researchers achieve test tube “proof of concept” for gene therapy.

1993 - FDA approves Pulmozyme, the first biotech drug designed for CF, which breaks down thick CF mucus following studies in CF care center network; first gene therapy tested in person with CF—shows gene therapy corrects CF cells in nasal passages; CF Foundation and NIH establish nine gene therapy centers for CF studies; Beall becomes president and CEO of the CF Foundation; The Boomer Esiason Foundation redirects its mission to support the CF Foundation when Esiason's son, Gunnar, is diagnosed with CF.

1996 - The Seattle Breath of Life Gala, which has raised more than $24 million since its inception in 1984, becomes the CF Foundation’s first “million dollar event,” raising $1,002,500 in one night; CF Foundation forms public policy alliance, a grassroots group of volunteers who inform policy decision makers.

1997 - FDA approves TOBI, an inhaled antibiotic, after CF Foundation-supported studies showed safety and efficacy; CF Foundation establishes the Therapeutics Development Program (TDP) with seven centers in the Therapeutics Development Network (TDN); CF Foundation forms Adult Task Force to address specific issues facing adults with CF.

1999 - Bill and Melinda Gates Foundation gives the CF Foundation a $20 million grant for drug discovery; Cam C. Cooper is named Chairman of the CF Foundation’s Board of Trustees.

2000 - CF Foundation-supported researchers complete map of Pseudomonas aeruginosa genome; CF Foundation establishes CF National Bioinformatics Center to help spur discovery of new treatments; CF Foundation awards contract to Aurora Biosciences for up to $46.9 million for CF drug discovery (the largest grant of its kind by a nonprofit organization); CF Foundation establishes Cystic Fibrosis Foundation Therapeutics, Inc. to govern CF Foundation’s drug discovery and evaluation efforts.

2001 - Tom Marsico, CEO of Marsico Capital Management, LLC, commits $25 million over several years in support of the TDP; CF Foundation makes first multi-million award for drug development with Altus® Pharmaceuticals, Inc. to develop TheraClec TM Total, a potentially more efficient pancreatic enzyme.

2002 - Results of CF Foundation-supported clinical trial on azithromycin show that the drug is effective in improving lung function in people with CF; the TDN expands to 18 centers.

2003 - Targeted Genetics, Inc., with CF Foundation support, begins a Phase II CF gene therapy trial that represents the largest and most advanced gene therapy trial for CF to date; CF Foundation tops $150 million in fund-raising; CF Foundation launches Volunteer Leadership Initiative, to bring 2,003 new volunteers to the CF Foundation in 2003.

2004 - Results from Inspire Pharmaceutical’s CF Foundation-supported clinical trial of INS37217 to restore ion transport demonstrate both safety and improvement in lung function in people with CF; pipeline of CF therapeutics in development includes nearly two-dozen potential therapies; CF Foundation recommends nationwide newborn screening for CF at North American CF Conference, which now includes nearly 3,000 attendees; life expectancy increases to the mid-30s.

2005 - Predicted median age of survival increases to nearly 37 years.

2006 - Cystic Fibrosis Foundation Therapeutics-supported studies in Australia and UNC show that hypertonic saline helps clear CF mucus and becomes a therapeutic option.

2006 - The first compound for CF, discovered through high-throughput screening at Vertex Pharmaceuticals, begins clinical trials.

2006 - Inspire Pharmaceuticals begins Phase 3 clinical trials of its innovative denufosol drug to hydrate and clear CF mucus.

2007 - VX-770, a potentiator candidate developed by Vertex Pharmaceuticals, entered Phase 2 testing. This drug may restore the function of the CFTR protein. Vertex also is working to develop corrector candidates, which aim to improve the function of the CFTR.

**The original post was taken from**

3 people had something to say...:

Teeda said...

I need to thank my CF doctor for having me on hypertonic saline in 2001 and Zithromax in 2000. He's a smart dude.

Andrea said...

My dad and I knew about all the early stuff. Very interesting.

On a different note, I have spent waaaaaaay too long watching your many youtube videos. Yes, watching a dressing change of a PICC line still makes me sick to my stomach (I can't watch my own!) and yes the Vest + Mandi CPT made me cough.

Thanks. :]

Anonymous said...

That statement (in the middle ages)about the salty child was interesting. When my daughter, Teresa, was a baby (she was born in 1963) my grandmother made a statement more than once about her skin tasting very salty. Teresa was diagnosed when she was almost three (after my girlfriend read an article in the newspaper about symptoms of CF). Before that, our doctor thought all of Teresa's problems were due to her being premature. Due to this she had lots of lung and heart damage so only lived to 10 years old. Her younger sister (born 1965) was lucky and had treatment from the age of one year and she is still living at 45 and doing fairly well.