Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Study shows new medication effectively treats underlying cause of cystic fibrosis

03.11.2011
Final stage clinical trial shows significantly improved lung function in subset of CF patients

A new study has confirmed that the drug, ivacaftor (VX-770), significantly improves lung function in some people with cystic fibrosis (CF). The results of the phase III clinical trial study, "A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation," led by Bonnie W. Ramsey, MD of Seattle Children's Research Institute and the University of Washington, were published today in the New England Journal of Medicine.

Ivacaftor, also known as VX-770, was developed by Vertex Pharmaceuticals with financial support from the Cystic Fibrosis Foundation. The oral medicine targets the defective protein produced by the gene mutation called G551D that causes CF. Researchers found that patients carrying G551D – approximately four per cent of all CF patients – who were treated with VX-770 showed a 17 per cent relative improvement in lung function that was sustained over the course of 48 weeks.

Additionally, patients with G551D treated with VX-770 showed improvements in other areas critically important to the health of people with CF. Study participants experienced significant reductions in sweat chloride levels indicating an improvement in the body's ability to carry salt in and out of cells – a process which when defective leads to CF. They also experienced decreased respiratory distress symptoms and improved weight gain. Those who received VX-770 gained on average seven pounds compared to those in the placebo group who gained approximately one pound. This is significant because many people with CF have difficulty gaining and maintaining weight due to reduced lung function and chronic infection.

"Our study shows that we are now able to improve the quality of life for cystic fibrosis patients with the G551D mutation with the administration of VX-770," said Dr. Ramsey, director of the Center for Clinical and Translational Research at Seattle Children's Research Institute and endowed chair in Cystic Fibrosis (CF) in the Department of Pediatrics at the University of Washington School of Medicine.

Dr. Ramsey and co-investigators evaluated lung function in patients 12 years or older who carry at least one copy of the G551D mutation. The study included 161 patients at multiple healthcare centers who received at least one dose of VX-770 or placebo. The study is the third and final in a series designed to assess VX-770's effectiveness and safety before it is approved for public use.

Approximately 30,000 children and adults in the United States and 70,000 people worldwide have CF. The disease is caused by a defective gene that affects many parts of the body, but is especially harmful to the lungs and pancreas. The gene mutation causes very thick, sticky mucus to clog the lungs and often cause life-threatening infections. In the 1950s, few children with CF lived beyond age five. Today, advances in research and medical treatments have made it possible for people with CF to live well into their 30s, 40s and beyond.

Co-investigators of this study in the Washington region are: Moira Aitken, MD, professor of medicine in the Division of Pulmonary and Critical Care Medicine at the University of Washington Medical Center and Ronald Gibson, MD, PhD, director of the Cystic Fibrosis Clinical Center at Seattle Children's Hospital and professor of pediatrics at the University of Washington School of Medicine.

Other co-investigators of the study include: Jane Davies, MD, MB, ChB from the Respiratory Biomedical Research Unit, Royal Brompton and Harefield National Health Service Foundation Trust, London; N. Gerard McElvaney, MD from the Royal College of Surgeons and Beaumont Hospital, both in Dublin; Elizabeth Tullis, MD from St. Michael's Hospital, University of Toronto; Scott C. Bell, MB, BS, MD from the Department of Thoracic Medicine, Prince Charles Hospital and Queensland Children's Medical Research Institute, University of Queensland in Brisbane, Australia; Pavel Dřevínek, MD from the Department of Pediatrics, 2nd Medical School, Charles University, Prague, Czech Republic; Matthias Griese, MD from Dr von Haunersches Kinderspital, University of Munich, Munich, Germany; Edward F. McKone, MD, from St. Vincent's University Hospital in Dublin; Claire E. Wainwright, MD, MB, BS from St. Vincent's University Hospital in Dublin and the Queensland Children's Medical Research Institute, University of Queensland in Brisbane, Australia; Michael W. Konstan, MD from Case Western Reserve University School of Medicine, Rainbow Babies and Children's Hospital, Cleveland; Richard Moss, MD from Stanford University School of Medicine, Stanford, CA; Felix Ratjen, MD, PhD from the University of Toronto Department of Pediatrics and Hospital for Sick Children in Toronto; Isabelle Sermet-Gaudelus, MD, PhD from the Centre d'Investigation Clinique–Hôpital Necker, Paris; Steven M. Rowe, MD, MSPH from the University of Alabama at Birmingham, Birmingham; Qunming Dong, PhD, Sally Rodriguez, PhD, Karl Yen, MD and Claudia Ordoñez, MD from Vertex Pharmaceuticals, Cambridge, MA; and J. Stuart Elborn, MD from the Centre for Infection and Immunity, Queens University Belfast, Belfast, United Kingdom.

About Seattle Children's Research Institute

At the forefront of pediatric medical research, Seattle Children's Research Institute is setting new standards in pediatric care and finding new cures for childhood diseases. Internationally recognized scientists and physicians at the Research Institute are advancing new discoveries in cancer, genetics, immunology, pathology, infectious disease, injury prevention and bioethics. With Seattle Children's Hospital and Seattle Children's Hospital Foundation, the Research Institute brings together the best minds in pediatric research to provide patients with the best care possible. Children's serves as the primary teaching, clinical and research site for the Department of Pediatrics at the University of Washington School of Medicine, which consistently ranks as one of the best pediatric departments in the country. For more information, visit http://www.seattlechildrens.org/research.

About UW Medicine

UW Medicine trains health professionals and medical scientists, conducts research to improve health and prevent disease worldwide, and provides primary and specialty care to patients throughout Seattle/King County and the WWAMI (Washington, Wyoming, Alaska, Montana and Idaho) region. UW Medicine includes: Harborview Medical Center, UW Medical Center, Northwest Hospital & Medical Center, Valley Medical Center, UW Neighborhood Clinics, UW School of Medicine, UW Physicians, and Airlift Northwest. UW Medicine also shares in the ownership and governance of the Seattle Cancer Care Alliance with Seattle Children's Hospital and Fred Hutchinson Cancer Research Center and shares in ownership of Children's University Medical Group with Seattle Children's Hospital. UW Medicine has major academic and service affiliations with Seattle Children's Hospital, Fred Hutchinson Cancer Research Center, the Veteran's Affairs Puget Sound Health Care System in Seattle, and the VA Hospital in Boise, Idaho. The UW School of Medicine has been ranked #1 in the nation in primary-care training for the past 18 years by US News & World Report. It is the top public institution for receipt of biomedical research funding from the National Institutes of Health (NIH) and second among all institutions for NIH funding, public and private. UW Medicine's 2,000 full-time faculty and nearly 5,000 volunteer and part-time faculty include four Nobel Laureates, 33 members of the National Academy of Sciences, and 33 members of the Institute of Medicine. For more information, visit UW Medicine.org. Follow us on Twitter - @UWMedicineNews

Media Contacts:
Louise Maxwell
Seattle Children's Research Institute
Email: louise.maxwell@seattlechildrens.org
Phone: 206-987-5210
Clare Hagerty
UW Medicine
Email: clareh@u.washington.edu
Phone: 206-685-1323

Louise Maxwell | EurekAlert!
Further information:
http://www.seattlechildrens.org

More articles from Studies and Analyses:

nachricht WAKE-UP provides new treatment option for stroke patients | International study led by UKE
17.05.2018 | Universitätsklinikum Hamburg-Eppendorf

nachricht First form of therapy for childhood dementia CLN2 developed
25.04.2018 | Universitätsklinikum Hamburg-Eppendorf

All articles from Studies and Analyses >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Molecular switch will facilitate the development of pioneering electro-optical devices

A research team led by physicists at the Technical University of Munich (TUM) has developed molecular nanoswitches that can be toggled between two structurally different states using an applied voltage. They can serve as the basis for a pioneering class of devices that could replace silicon-based components with organic molecules.

The development of new electronic technologies drives the incessant reduction of functional component sizes. In the context of an international collaborative...

Im Focus: LZH showcases laser material processing of tomorrow at the LASYS 2018

At the LASYS 2018, from June 5th to 7th, the Laser Zentrum Hannover e.V. (LZH) will be showcasing processes for the laser material processing of tomorrow in hall 4 at stand 4E75. With blown bomb shells the LZH will present first results of a research project on civil security.

At this year's LASYS, the LZH will exhibit light-based processes such as cutting, welding, ablation and structuring as well as additive manufacturing for...

Im Focus: Self-illuminating pixels for a new display generation

There are videos on the internet that can make one marvel at technology. For example, a smartphone is casually bent around the arm or a thin-film display is rolled in all directions and with almost every diameter. From the user's point of view, this looks fantastic. From a professional point of view, however, the question arises: Is that already possible?

At Display Week 2018, scientists from the Fraunhofer Institute for Applied Polymer Research IAP will be demonstrating today’s technological possibilities and...

Im Focus: Explanation for puzzling quantum oscillations has been found

So-called quantum many-body scars allow quantum systems to stay out of equilibrium much longer, explaining experiment | Study published in Nature Physics

Recently, researchers from Harvard and MIT succeeded in trapping a record 53 atoms and individually controlling their quantum state, realizing what is called a...

Im Focus: Dozens of binaries from Milky Way's globular clusters could be detectable by LISA

Next-generation gravitational wave detector in space will complement LIGO on Earth

The historic first detection of gravitational waves from colliding black holes far outside our galaxy opened a new window to understanding the universe. A...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

Save the date: Forum European Neuroscience – 07-11 July 2018 in Berlin, Germany

02.05.2018 | Event News

Invitation to the upcoming "Current Topics in Bioinformatics: Big Data in Genomics and Medicine"

13.04.2018 | Event News

Unique scope of UV LED technologies and applications presented in Berlin: ICULTA-2018

12.04.2018 | Event News

 
Latest News

When corals eat plastics

24.05.2018 | Ecology, The Environment and Conservation

Surgery involving ultrasound energy found to treat high blood pressure

24.05.2018 | Medical Engineering

First chip-scale broadband optical system that can sense molecules in the mid-IR

24.05.2018 | Physics and Astronomy

VideoLinks
Science & Research
Overview of more VideoLinks >>>