From Bench to Bedside
Since Dr. Dorothy Andersen recognized cystic fibrosis as a disease in 1938, researchers have been trying to understand what causes CF and its complications and to find a cure. Families of people with CF realized early on that funding medical research would be essential to bring an end to the disease and created the National Cystic Fibrosis Research Foundation in 1955.
Although the name has changed, the mission of the Cystic Fibrosis Foundation remains the same. By pursuing a wide range of promising strategies, the Foundation has built a robust pipeline of approved and potential therapies that tackle CF from all angles, including drugs targeting the underlying cause of the disease. Nearly every drug recommended for CF was made possible because of CF Foundation support.
The Foundation supports the best research on both national and international fronts and from bench to bedside -- funding work on potential therapies in the laboratory and facilitating clinical trials to inform specialized care at CF Foundation-accredited care centers. This research includes:
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Basic science, which is fundamental bench research in the laboratory that provides the basis for improving our understanding of CF at molecular, cellular, and tissue levels
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Clinical research, in which basic science research findings are “translated” into potential diagnostic tools, procedures, and drugs that are then tested to see if they can improve the health of people with CF
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Real-world research, which examines how “real-world” situations and human behavior affect CF treatments and care
Basic Science
Funding basic science research underpins our understanding of how CF affects the body. Scientists focus on explaining the mechanisms -- or the essential processes -- involved in causing the disease and its complications, such as CF-related diabetes.
The Foundation supports leading scientists at universities around the world to study everything from the structure of the cystic fibrosis transmembrane conductance regulator (CFTR) protein to the challenges associated with editing the CFTR gene. In addition, the Foundation supports research and development programs -- or research hubs -- that focus on critical scientific problems and train the next generation of CF scientists. Every year, many of the labs that receive grants present their research findings at the annual CF Foundation Research Conference.
The CF Foundation operates its own research laboratory dedicated exclusively to advancing CF therapies. The Cystic Fibrosis Foundation Therapeutics Lab capitalizes on emerging scientific discoveries related to CFTR modulators, nonsense mutations, gene editing, and stem cell research. It develops drug-screening tools and technologies identified by scientists and makes them available to other researchers and companies. In addition, the lab team trains external researchers all over the world in the specialized techniques needed to develop CF therapies.
Sound investments by the Foundation in cutting-edge science have helped build an extensive base of knowledge about the disease. Some of these ideas have led to innovative new therapies in the Foundation's Drug Development Pipeline that have made a significant difference in the health and quality of life of tens of thousands of people who have CF.
Clinical Research
Because pharmaceutical companies have historically been reluctant to make major investments in developing drugs for rare diseases like CF, the Foundation has provided funding to encourage biotech and pharmaceutical companies to focus on CF and help advance potential therapies that would be unlikely to move beyond the lab and into clinical trials. The Therapeutics Development Program provides funding during the early phase of drug discovery and development, when the risk for failure is greatest and companies are the most hesitant to invest. These efforts have resulted in CF drugs, such as dornase alfa (Pulmozyme®), inhaled tobramycin (TOBI®), and ivacaftor (Kalydeco®).
The Foundation also funds clinical researchers who analyze the most promising research findings to develop potential new treatments. For example, the Foundation is supporting drug discovery efforts to screen compounds to see if they can “read through” the premature stop signals caused by nonsense mutations. If so, these compounds will be developed further into potential drugs and then tested in animals to help determine safety. This type of pre-clinical work -- before a drug can be tested in people -- is essential in drug development.
If the animal studies are successful, clinical researchers then set up clinical trials to test these potential therapies in people. To speed up the testing of these drugs, the Foundation provides funding to the Therapeutics Development Network (TDN), a network of 90 Foundation-accredited care centers with specialists in CF clinical research who recruit study participants and conduct clinical trials. (Our Drug Development Pipeline shows drugs that the Foundation has either helped financially or assisted through the TDN.)
In addition, the Foundation has been working with the U.S. Food and Drug Administration (FDA) to develop strategies to test new therapies even when only a small number of people with CF are affected. These strategies are especially needed to help people with CF who have very rare mutations.
Real-World Research
Real-world research takes into account the realities of daily life and human behavior and how they can affect the way treatments work and the way care is provided. At the CF Foundation, we conduct different types of real-world research, including:
- Observational
- Post-approval
- Comparative effectiveness
- Behavioral studies
Observational studies conducted with data from the CF Foundation's Patient Registry help us understand CF by showing trends in survival and complications among people with CF who volunteer their information and are seen at CF Foundation-accredited care centers. The studies also allow us to track the effectiveness of therapies. Unlike interventional studies where potential treatments are tested, observational studies do not involve any changes to a treatment plan and are designed for observing individuals in their normal day-to-day settings, while measuring the effects or outcomes of those treatments.
Another example of real-world research supported by the Foundation is the post-approval study. After a drug has been approved by the FDA, researchers will continue to monitor the drug for safety and effectiveness in what is known as a Phase 4 study. These studies look at the long-term effects of the therapy to further refine the safety, efficacy, or usage of the treatment as people use it in their day-to-day or “real-world” settings.
The Foundation also funds comparative effectiveness research on FDA-approved therapies to collect data on which ones help people with CF the most. These research findings are used to inform best practices at CF care centers nationwide. For example, in the STOP 2 trial, researchers are trying to determine the ideal amount of time to administer IV antibiotics to treat pulmonary exacerbations.
Behavioral studies are designed to identify how factors, such as self-management, mental health, and social support, are associated with health outcomes. Understanding these factors help us develop ways to improve therapies and how they are used. Researchers can observe how people take their medications or follow a daily care plan to see how it affects their health. Behavioral studies can be observational, where there is no change in how or what people do with regard to their treatment plan, or interventional, where researchers monitor a change in behavior to determine if it has an impact on the health of people with CF.
At the CF Foundation, the Success with Therapies Research Consortium (STRC) is conducting behavioral studies to identify new tools and practices to help care teams partner with people with CF to more effectively manage their disease.
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Information contained on this site does not cover all possible uses, actions, precautions, side effects, or interactions. This site is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.
FDA-approved drug information is available at dailymed.nlm.nih.gov/dailymed.