Incorporated: 1955 as National Cystic Fibrosis Research Foundation
NAIC: 813212 Voluntary Health Organizations
Cystic Fibrosis Foundation, based in Bethesda, Maryland, has been dedicated since the mid-1950s to finding a cure for cystic fibrosis (CF), an inherited disease affecting the lungs and digestive tracts, so named because of the "fibrosis" (tissue scarring) of the biliary tract (with "cystic" an adjective referring to the urinary bladder or gallbladder). While not yet completely successful in its mission, the organization has spearheaded significant advances in the treatment of CF. Once the scourge of childhood, CF had caused death before a victim reached school age; a half-century later the median survival age is approaching 37. The foundation boasts 80 chapters across the United States and more than 250,000 volunteers who host fund-raising programs and events. It provides funding to 115 CF care centers, 94 of which are adult care programs. Another 54 affiliate programs also receive foundation support. In addition to treatment centers, the foundation supports more than ten research centers in its Research Development Program, provides grants to encourage independent research, and spurs drug development through grants to private companies. All told, it provides support for some 30 potential new treatments for the disease. The foundation is well respected for its efficiency, spending about 90 percent of the money it raises on care, education, and research programs. Profits made from investments in private companies are also used to fund further research.
CF Recognized As Separate Disease: 1938
While aspects of CF were recognized for many years, it was not until 1938, when Dr. Dorothy Andersen published "Cystic Fibrosis of the Pancreas and Its Relation to Celiac Disease: A Clinical and Pathological Study" in the American Journal of Diseases of Children, that CF was recognized as a separate disease. Dr. Andersen was also the first to posit that CF was a hereditary disease transmitted by a recessive gene possessed by both parents. As a result of her pioneering efforts, physicians began to specialize in the care of children with CF. One of their number was a young doctor at Columbia Presbyterian Medical Center in New York City, Dr. Paul di Sant'Agnese, who developed a key diagnostic tool that would remain in use more than half a century later, the sweat test.
During a severe heat wave in 1948 di Sant'Agnese and his colleagues first noticed that most of the people admitted to the hospital for heat prostration were CF patients. This observation was reinforced four years later by another heat wave, during which di Sant'Agnese recognized that CF children were losing electrolytes at an abnormally high rate, leading to extremely salty perspiration. Believing that this phenomenon distinguished those with CF from the general population, in 1953 he and a colleague developed a simple and painless test to detect high levels of chloride to help in the diagnosis of CF. The child's hand or foot was placed on a culture medium, a sheet of agar laced with silver nitrate and potassium chromate, laid in a flat glass dish. Normal children left only faint impressions, while CF children, whose perspiration contained ten times as much salt, left a prominent imprint on the agar. The test was about 99 percent certain and the diagnosis could be later confirmed by more advanced tests.
Foundation Formed: 1955
In 1954 di Sant'Agnese and some parents of children suffering from CF formed the Cystic Fibrosis Association in New York City. Other chapters were soon formed in Connecticut and California. Some of those concerned parents included New York mother Doris Tulcin, trucking executive Robert L. Natal of Bayside Queens, New York, and Boston physician Dr. Wynne Sharples (who had two children with CF and whose husband, Dr. Robert Denton, developed a breathing apparatus to alleviate the suffering of CF victims), all of whom, as well as other concerned parents, would play lead roles in the organization. In 1955 they and other volunteers brought the association chapters together into a new nonprofit organization, the National Cystic Fibrosis Research Foundation. "We had to do something. Children were dying and physicians didn't know what the disease was. They had no prognosis for it. There was no research. There was nothing," Tulcin recalled in an interview with the Cystic Fibrosis News Bureau years later.
At first, the organization established its headquarters in Philadelphia, Pennsylvania. In 1955 the foundation issued its first research grants to Dr. Andersen and di Sant'Agnese and Dr. Harry Shwachman. The first grant was $10,000. Fund-raising was conducted solely on the local level until September 1960 when the first national campaign was launched. A year later the foundation began its accredited care center program, opening a pair of centers. In 1962 the number of centers would increase to 30. By this time, the efforts of the foundation and CF researchers had succeeded in lengthening the median survival age of CF patients to approximately ten years.
In 1965 the trademarked term "65 Roses," the way CF is commonly referred to by children, was coined. One foundation volunteer, Mary G. Weiss, whose three young boys suffered from CF, was overheard by one of the children, four-year-old Richard, making a series of fund-raising telephone calls for the organization. He surprised her when he claimed to know what she was working for: "65 roses." What Richard misheard was easier for children to pronounce than cystic fibrosis and quickly caught on within the CF community.
A clinical fellowship program was sponsored by the foundation in 1965 as a way to bring basic scientists into CF research. It was also late in 1965 that the foundation laid plans for a five-year $1.5 million coordinated research program, intended to speed up the discovery of CF's cause and a way to control the disease. In order to help in this effort, a year later the foundation created a data registry to track the histories for all patients at its accredited care centers. Five years would not be enough time to complete the research mission, however. While this important work was carried on, the foundation also made progress in its educational effort. In 1968 it hosted its first National-International Medical Conference. The first national seminar held by the foundation to address the needs of young adults with CF was held in 1972.
Move to Washington, D.C., in 1978
The foundation in 1978 moved its headquarters from Philadelphia to the Washington, D.C., area, a more advantageous location given that it was the seat of federal power as well as the home of the National Institutes of Health, a major provider of research funding. It was also in 1978 that the foundation conducted its first national fund-raising event, Bowl for Breath, a bowling tournament. It was highly successful and became a fund-raising staple for local chapters of the organization. Some of that money would be put to use in increasing the number of foundation accredited care centers to 125 by the end of the year.
The early 1980s brought a number of changes to the foundation. In 1981 the first satellite care centers were established for adults, needed because the life expectancy for CF sufferers had reached 20 years. At the start of the decade the Research Development Program was established, creating a network of university research centers. To support the program Doris Tulcin launched the first capital program ever conducted by a voluntary health organization, the goal to raise $15 million. Private enterprise was encouraged to become involved as well, provided with financial incentives to develop drugs for CF and other less common diseases from the Orphan Drug Act signed into law in 1983. Scientists were also making progress in their research during this period. In 1983 they were able to demonstrate that cells lining the lungs of CF patients did not properly move chloride into the airways. Moreover, CF was receiving more national attention due to well-known sportswriter and novelist Frank Deford, who wrote a book about his daughter, who died of CF at the age of eight. Deford was named chairman of the foundation's board of trustees and used his celebrity status to raise the profile of CF.
Working closely with Deford was the foundation's first chief executive officer, Robert K. Dresing, a successful businessman who owned three Ethan Allen furniture stores in Ohio and Florida. The father of a son with CF, Dresing became the volunteer president in 1982 and two years later became CEO, applying his business experience to the running of the organization. "I brought a bottom-line mentality to an organization that had never been run like a for-profit group." Among the changes he made were incentive-based pay and centralized management.
CF Services Established: 1988
By the start of the second-half of the 1980s, the life expectancy of CF reached 25 years. The foundation enjoyed improved success in its fund-raising efforts, building donations to the $20 million mark in 1986, due in part to a made-for-television movie based on Deford's book that ran that year on the ABC network, Alex: The Life of a Child. The extra funds allowed the foundation in 1988 to establish CF Services, Inc., a national pharmacy that made CF drugs and treatments available across the country. The most significant event of this period, and arguably the turning point in the history of the foundation, was the 1989 discovery of the cystic fibrosis gene, along with its protein product, by scientists supported by the foundation: Francis Collins, John Riordan, and Lap-Chee Tsui. For the first time, rather than merely treat the symptoms of CF, researchers could replicate a healthy version of the gene to seek out the root cause of the disease in order to speed the creation of drugs and gene therapies.
In 1990 researchers achieved "proof of concept" in a lab dish, showing that gene therapy for CF was possible. Three years later a gene therapy trial began in CF patients and demonstrated correction in CF cells for treating nasal passages. Also in 1993 the Food and Drug Administration (FDA) approved the first biotech drug for CF, Pulmozyme, which breaks down the thick mucus deposited in lungs caused by CF.
The foundation underwent a major reorganization in 1993 that transferred some of its assets to a private company headed by Dresing, who would resign as the foundation's CEO and take about half of the foundation's employees to run the offshoot. Included were the foundation's healthcare service, direct-mail fund-raising operation, and mail-order pharmacy business. Dresing agreed to run these operations on an audited, no-profit basis for the benefit of the foundation, but was free to make a profit from other health-related ventures. Some trustees and outside observers questioned whether Dresing was receiving a sweetheart deal, or whether the foundation would be better served by considering other third-party companies to run the units. Deford, however, dismissed these criticisms, telling the Washington Post, "If you are happy with the people you have, then I don't think you should go through the motions of going outside." He added, "Do you really think we could get anybody to do this for free or at cost? He's not making any money out of this." By making the switch, the foundation also hoped it would be able to better focus its resources to finding a cure for CF.
Approval of Second CF Drug: 1997
A second CF drug, TOBI, an inhaled antibiotic, received FDA approval in 1997. In that same year, the foundation established the Therapeutics Development Program in order to shepherd potential CF drugs through the development and regulatory approval process. Seven specialized clinical research centers were opened to form the Therapeutics Development Network. On another front, the foundation created the Adult Task Force to address issues faced by an increasing number of adults with CF due to the increasing age of survival.
Although a decade passed since the identification of the CF gene, progress on the development of drug treatments was not as great as the foundation had hoped. Rather than awarding small grants to academic researchers, by the start of the new century a greater emphasis was placed on encouraging private companies to become involved. Because the market for CF drugs was small, financial incentives were necessary.
In the 1990s the foundation had enjoyed a successful partnership with the development of TOBI. After funding the early FDA trials, it was able to sell the rights for $17 million in cash to PathoGenesis, which benefited because TOBI soon became its top-selling product. This experience led to the foundation adopting a new business model and the 2000 creation of Cystic Fibrosis Foundation Therapeutics to oversee drug discovery and development funding. In addition, Cystic Fibrosis National Bioinformatics Center was established to further spur drug and treatment development. While it was legal for it to invest in private companies, the foundation preferred to provide funding for development and trials and then take royalties. (Later investments would ask for multiples on the investment in lieu of royalties.)
By the spring of 2001 the foundation gave money to 11 companies, both established and start-ups, including Genzyme, MoliChem Medicines, Inologic, Altus Biologics, Structural GenomiX, Copernicus Gene System, and Genaera Corp. It also provided seed money to PathoGenesis, Inspire Pharmaceutics, and SciClone Pharmaceuticals. Another $46.9 million was pledged to Aurora Biosciences in May 2000, contingent on the discovery of three drug candidates and meeting certain milestones over a five-year period.
Basic science was not neglected, however. In 2000 researchers funded by the foundation completed the genetic map of Pseudomonas aeruginosa bacterium, the most common cause of CF lung infections. The private companies backed by the foundation would soon enjoy successes as well. In 2003 Structural GenomiX scientists were able to determine the three-dimensional structure of a portion of the CF protein product, thus clearing the way for many more drug discovery possibilities. A year later there were about two dozen CF therapies in various stages of development. Some of the more promising ones proved to be disappointments: Boehringer Ingelheim's anti-inflammatory drug was pulled during Phase II trials in July 2004; Amelubant failed in trials later in the year; and in 2005 Targeted Genetics' aerosol gene treatment was terminated during Phase II trials. Others took their place in the pipeline, though.
The median age for survival for CF patients approached 37 years in 2005, a tribute to the efforts of the foundation over the course of 50 years. More drugs entering advanced clinical trials for FDA approval and new investments in companies to introduce more drug candidates into the pipeline promised to increase the survival age. Better diagnostic tools also offered hope, because early intervention could prevent lung deterioration and add decades to a patient's life. The day was likely approaching that the Cystic Fibrosis Foundation would reach its ultimate goal: putting itself out of business. On that day, with a cure found, another scourge of childhood, like polio, would begin to become a mere memory.
Principal Subsidiaries
CF Services Pharmacy; Cystic Fibrosis Foundation Therapeutics; Cystic Fibrosis National Bioinformatics Center.
Further Reading
Angier, Natalie, "New Advances Offer Cystic Fibrosis Victims Heartening Prospects," New York Times, August 28, 1990, p. C3.
"Breath of Fresh Air for Cystic Fibrosis Drug Pipeline," Pharmaceutical Approvals Monthly, December 1, 2007.
"Cystic Fibrosis Foundation Marks 50 Years in the Fight Against Cystic Fibrosis," Medical News Today, January 26, 2005.
"Cystic Fibrosis Unit Sets 5-Year Research Study," New York Times, December 5, 1965.
Madden, Stephen, "Sweet Charity," Fortune, February 13, 1989, p. 113.
Moukheiber, Zina, "Drug Money," Forbes, April 2, 2001, p. 80.
"Robert L. Natal, 57, Trucking Executive," New York Times, April 26, 1967.
Spayd, Liz, "Charity's Reorganization Questioned," Washington Post, October 23, 1993, p. A1.
"Warning Sweat," Time, August 5, 1957.
— Ed Dinger
