Established in 2000, Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) is the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation. CFFT supports and governs activities related to cystic fibrosis (CF) drug discovery through the many stages of drug development and clinical evaluation.

The CF Foundation provides support to fund CFFT's operations, specifically the Therapeutics Development Program. Sound investment by the Foundation in cutting edge science has built an extensive base of knowledge about this disease. Some of these ideas have already led to innovative new therapies now in the Drug Development Pipeline.

In fact, one way to look at the wide-ranging and diverse science supported by the CF Foundation and its affiliate, CFFT, is to think that each study could be a step toward a new CF therapy. Molecular biologists, cell physiologists and immunologists, for example, all ask the same question — How does this study lead to a potential CF therapy or the cure? Seeking the answers drives the research advances forward to improve the lives of individuals with CF.

Therapeutics Development Program

To bridge the gap between what has been learned in the laboratory and the evolution of new therapies, the Therapeutics Development Program was created. This model initiative has the infrastructure in place to support a virtual “pipeline” of CF therapeutics development from the discovery phase through several stages of clinical evaluation.

Despite the increasing age of survival, people with CF continue to need new medications to reduce the effects of their disease until the cure is found. Therefore, the CF Foundation and its affiliate, CFFT, strive to maximize the number of innovative drugs being developed because, on average, only one in five compounds will successfully make it through clinical trials all the way to the patient.

Increasing the number of promising compounds increases the odds of success! Such investment by a voluntary health organization is unprecedented and has already served as a model for others to follow.

How the Program Works

Through the Therapeutics Development Program, CFFT offers matching research awards to scientists, as well as access to a specialized network of CF clinical research centers. These awards provide support for the drug discovery phase through several stages of evaluation to complete the full-length drug development pipeline.

This is a win-win equation — the Therapeutics Development Program provides companies with a powerful new opportunity to have investment capital during the early phases of drug research. And, it ensures the availability of new potential compounds for clinical investigation for the CF community.

Current estimates suggest that it costs more than $800 million to move a drug from its concept stage to the market place. There is a critical need to help provide support to pharmaceutical and biotechnology companies that conduct drug discovery and early-stage clinical evaluation studies in small population diseases such as CF.

Even with incentives, such as the Orphan Drug Tax Credit (that encourages investment in orphan diseases like CF, which affect patient populations of less than 200,000), the fact remains that pharmaceutical companies must first secure the financial resources to invest in these diseases.

Further, with increasing demands being placed on pharmaceutical and biotechnology companies, especially the small “start-ups,” investors are often hesitant about making major capital investments for orphan disease-classified drugs. The Therapeutics Development Program attracts researchers to the CF drug development process and shows a level of commitment unrivaled by any other voluntary health organization.

Opportunities Abound

As the understanding of the science of CF increases, there is a correlated growth in the number of opportunities to discover and develop new potentially lifesaving therapies. Today, there are early phase trials underway in CF gene therapy, protein-assist therapy, as well as studies testing anti-infective drugs, and anti-inflammatory drugs. With the increasing number of potential drugs and an innovative network of specialized clinical trial centers to evaluate them, the future for those battling CF has never been brighter.

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