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Highlights from the CF Foundation’s North American CF Conference

November 4, 2008

More than 3,800 leading cystic fibrosis scientists and health care professionals gathered in Orlando, Fla. from October 23rd to October 25th to exchange ideas and discuss developments in cystic fibrosis research and care.

The 22nd annual North American CF Conference (NACFC) included presentations by researchers who discussed the basic science of CF, and seminars in which physicians showed that treating the symptoms of CF with ever-improving drugs is increasing the life expectancy of people with this disease.

Below are summaries highlighting some of the most exciting topics that were covered at NACFC, including:

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The CF Foundation’s Therapeutics Pipeline

“Cystic fibrosis is one of the most amazing stories in medicine today,” said Preston W. Campbell, III, M.D., executive vice president of medical affairs of the CF Foundation, during his plenary presentation, “The CFF Pipeline: The Amazing Story of Progress, Hope and Challenge.”

Click on image to hear Dr. Michael Knowles, professor of medicine at UNC at Chapel Hill discuss the diversity of drugs in the CF drug development pipeline.

Video Clip Available Dr. Michael Knowles is a professor of medicine at the University of North Carolina at Chapel Hill and discusses the diversity of drugs in the CF drug development pipeline.

In this second plenary session of the NACFC, Campbell focused on how the Foundation has moved from a discovery era—accumulating a scientific understanding of the disease—to a therapeutics era—developing life-lengthening drugs and improving quality of care.

That transition is evident in the Foundation's drug development pipeline. There are currently more than 30 potential therapies in development with a diverse approach to treating CF, from addressing the underlying cause to creating better anti-inflammatory and anti-infective drugs.

“The area involving therapies that treat the symptoms of CF has never been more exciting,” said Campbell. These therapies can add decades of life to people with CF. In addition, oral therapies that could correct the faulty protein responsible for CF are undergoing trials.

“The impossible has become possible,” said Campbell. In 2009, two pivotal clinical trials will begin involving therapies that address the basic defect. These trials, which could lead to drug approval, are the direct result of successful early-phase clinical trials.

Click on image to see the progress made in the CF Foundation's drug development pipeline since the year 2000.

Video Clip Available The CF drug development
pipeline has grown significantly since
the year 2000.

After presenting the promising data, Campbell told the audience that it is important to be realistic about the prospect of new treatments. There is no way to know what therapies will become approved drugs, he said, and warned that a drug entering into a Phase 1 clinical trial has only a 20 percent success rate.

However, Campbell noted that if the pipeline continues to produce successful therapies, a day could exist where people with CF experience no decline in lung function.

“I think we will be able to focus on maintaining health rather than treating disease,” concluded Campbell. “People with CF will live with their disease, not die of it.”

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CFTR 2008

Click on image of Dr. William Balch to hear him describe CFTR.

Video Clip Available Dr. William Balch, a professor at the Scripps Research Institute in La Jolla, Calif., explains CFTR.
More than 1,000 mutations affect how the key protein associated with CF (CFTR) is made, where it ends up in the cell and how well it functions. Throughout the NACFC, researchers presented ways of fixing the CFTR protein using a variety of approaches.

The Foundation has supported several collaborations with industry to explore the possibility of using small molecules to correct CFTR, including EPIX Pharmaceuticals, Inc., FoldRx Pharmaceuticals, Inc., Vertex Pharmaceuticals, Inc. and PTC Therapeutics Inc. This area of research generated the most exciting news at the conference.

Scientists discussed correctors, compounds that move defective CFTR to its proper place in the cell, potentiators, compounds that allow CFTR at the cell surface to function correctly, and dual-acting compounds, single compounds that enable CFTR to be made correctly and help it work better at the surface of the cell.

“These dual-acting compounds are the dream molecules that everyone would want to have,” said Philip Thomas, Ph.D., professor of physiology at the University of Texas Southwestern Medical Center in Dallas.

Scientists expressed their excitement over advances in drug discovery and development which address the basic defect, as well as advancements in the understanding of exactly how CFTR controls ion channel activity.

There is a machine in your body that is responsible for moving salt and water into the lining of the lungs, explained Thomas. Certain genetic changes in CF patients lead to a problem in a component of the machine, which breaks the machine.

Click on image of Dr. Philip Thomas to hear him discuss dual-acting compounds.

Video Clip Available Dr. Philip Thomas, from
the University of Texas in Dallas, discusses dual-acting compounds.

“To repair any broken machine, you have to understand what the machine does and what part of the machine is broken,” said Thomas. That understanding “gives us the chance to make treatments that only work on CFTR. That’s important because it reduces the chance of side effects of these potential drugs.”

“I think the course of this disease is going to be impacted by the emergence of these molecules in several years,” Thomas added. “The clinical trial data that’s emerging is extremely promising. The data is really breathtaking.”

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Quality Improvement

Improving the quality of care for people with CF was a key focus throughout NACFC. Care centers involved in the sixth cycle of the Foundation’s Learning and Leadership Collaborative (LLC), an intensive quality improvement program, met before the conference kick-off to share progress toward achieving their quality improvement (QI) goals, learn from each other and make plans for implementing new ideas at their own care centers.

Click on image of Dr. Kraynack and hear what he has to say about the importance of quality improvement.

Video Clip Available Dr. Kraynack, who is the CF center director at the Akron Children's Hospital in Ohio talks about the importance of a quality improvement program.

Center staff members who had implemented QI programs at their own care centers shared with attendees from other centers several keys to successful quality improvement efforts, including the importance of:
  • Creating a core team comprised of physicians, nurses, people with CF and their families
    and other key leaders;
  • Promoting communication among the CF healthcare provider, people with CF and
    their families;
  • Setting goals and measuring progress towards those goals; and
  • Providing patient education to increase adherence and improve outcomes.

“It’s important to build a core quality improvement team,” said Nathan Kraynack, M.D., CF center director at Akron Children’s Hospital in Ohio. “When you build as a team you can really accomplish a great deal. When you realize the team is there to help with a common goal, QI really begins to work.”

In addition to its LLC program, the Foundation has implemented mentorship programs as a way to help train caregivers new to CF. The first mentorship program, spearheaded by Amanda Leonard, a pediatric registered dietician who works with children with CF and families at Johns Hopkins Children’s Center, was adopted by healthcare providers at centers across the nation.

Click on image of Amanda Leonard to hear more about the mentorship program and how it has made an impact on overall CF care.

Video Clip Available Amanda Leonard, R.D., a pediatric dietitian at the Johns
Hopkins Children's Center, explains the mentorship
program and how it has made an impact on overall CF care.

“We identified CF mentors and matched them with an apprentice at a CF care center with similar demographics,” said Leonard. “At the end of the mentorship program, the dietitians had more confidence delivering care, which makes for better care overall.”

The mentorship program has been so successful that it has expanded to include social workers, respiratory therapists and research coordinators. Mentoring programs for clinical coordinators and pharmacists are in development.

Overall, 77 care centers have gone through the Foundation’s QI and LLC programs and approximately 16,500 people with CF have benefited. Through the Foundation’s QI Initiative, care centers have learned specific ways to make small changes that add to big improvements in the health of people with CF.

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Adult Care

In 1985, adults with CF made up only 28 percent of the CF population. Today, adults represent more than 45 percent of people with CF. “There have been substantial changes in the adult population,” said Richard Simon, M.D., adult CF program director at the University of Michigan. “Patients are not only living longer, but they’re living healthier.”

Click on image of Dr. Simon to hear him talk about the therapeutic needs of adults with CF.

Video Clip Available Dr. Simon, who is the adult CF program director at the University of Michigan, elaborates on the therapeutic needs of CF adults.
Simon stressed that “adults are not merely big children with CF.” They are patients with a unique set of concerns and health challenges. As this population of patients grows rapidly, the Foundation is implementing programs that will specifically address these challenges.

In a NACFC short course about strengthening adult programs, the Foundation’s Program for Adult Care Excellence (PACE) was discussed as an excellent example of strengthening adult CF care programs.

PACE, which kicked off earlier this year, aims to recruit new doctors who are already established at medical centers to the field of CF. PACE provides partial funding to physicians so they can become better trained and better skilled at treating adults with CF.

During the short course on strengthening adult programs, James Yankaskas, M.D., co-director of the University of North Carolina adult CF program at Chapel Hill, noted “we’re getting pretty close to being able to control this disease.”

As CF is controlled and people with the disease live longer, they need to be cared for by specially trained doctors at accredited CF adult care centers.

Click on image of Dr. Yankaskas to hear him discuss challenges for adult CF care.

Video Clip Available Dr. Yankaskas is the
co-director of the UNC adult CF
program at Chapel Hill and
discusses adult CF care.
Currently, 96 adult care centers exist throughout the United States and 32 PACE grants have been awarded to expand the population of adult care specialists.

Adults with CF deal with an additional set of challenges, reiterated Yankaskas. Several complications that are relatively uncommon in pediatrics occur in adults. For example, adults may be faced with diabetes associated with CF, osteoporosis and depression. In addition, reproductive issues become an important consideration.

Women with CF who want to have children can generally do so without significant effects to their health, but infertility in men with CF is a significant problem.

By 2011, Yankaskas believes that more than 50 percent of people with CF will be adults. To keep this population thriving, the Foundation and its Care Center Network will continue to make clinical improvements in adult care by updating and revising clinical care guidelines, pushing QI efforts and emphasizing the importance of therapies in improving and maintaining health.

In the third and final plenary session, Simon who presented “Healthy Aging with Improved Adult Care” looked even further ahead and noted that the CF medical community will “need to determine what the needs of an elderly CF population would be. Who will take care of the elderly CF population? Perhaps 10 years from now there will be a short course at the 32nd annual NACFC entitled ‘Strengthening Geriatric CF Programs.’”

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Drug Development News

Two of the most advanced therapeutic programs currently addressing the basic defect in CF are from Vertex Pharmaceuticals, Inc. and PTC Therapeutics. The latest data from both programs were presented at NACFC and represent key advancements for CF therapies. In addition, Inspire Pharmaceuticals, Inc., which is developing a drug that bypasses the basic defect, also presented new data at the conference.

  • Vertex Pharmaceuticals, Inc.
    Dr.'s Beall and Accurso discuss VX-770 trial results.
VX-770, an investigational oral drug aimed at treating the basic defect in cystic fibrosis, showed positive results in a 28-day, Phase 2 clinical trial.
The study was carried out in patients who carry the G551D mutation of CF.

Frank Accurso, M.D., director of the CF care center at the Children’s Hospital in Denver, Colo. and principal investigator of the VX-770 clinical trials, presented the most recent clinical trial data for VX-770. The potential drug significantly reduced sweat chloride levels and improved lung function of patients who took the drug over 28 days.

“Data from the Phase 2 trial of VX-770 provide evidence that a small molecule can address the basic defect in cystic fibrosis, and suggest that modulation of CFTR may play an important role in CF therapy,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation.

  • PTC Therapeutics, Inc.

Data from a Phase 2 extension study of PTC124, an investigational oral drug for the treatment of cystic fibrosis caused by nonsense mutations (mutations that prevent a full-length protein from being made), were presented at the conference.

Eitan Kerem, M.D., principal investigator and head of the Department of Pediatrics and Cystic Fibrosis Center from the Hadassah University Hospital in Israel, summarized the Phase 2a data, as well as results from a Phase 2 extension study. He reported that patients who took the drug showed significant improvement in CFTR activity. A Phase 2b study is expected to begin in 2009.

“The encouraging data from the Phase 2a studies of PTC124 indicate that it may become a very important drug for the treatment of CF in the 10 percent of patients who carry nonsense mutations. These exciting results lead us to believe we are on the right path to finding a drug that will treat the basic defect in CF,” said Beall.

  • Inspire Pharmaceuticals, Inc.

Denufosol, an inhaled therapy being developed by Inspire for the treatment of CF, is designed to improve chloride transport in the airways and enhance mucus hydration and clearance. In the Phase 3 clinical trial called TIGER-1, the drug significantly improved lung function in patients.

Accurso, who is also principal investigator of the denufosol clinical trials, presented additional data from TIGER-1 at NACFC. The new data indicate that patients who took denufosol for an additional 24 weeks during the open-label extension experienced additional improvement in lung function.

“These are very exciting, promising data suggesting that denufosol can make a difference for CF patients, particularly since the therapy could benefit all types of patients regardless of their specific genetic mutation,” said Accurso.

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