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 TU Dresden biologists examine sperm quality on the basis of their metabolism
29.11.2019 | Technische Universität Dresden

nachricht Approaching the perception of touch in the brain
27.11.2019 | Max Planck Institute for Human Cognitive and Brain Sciences

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: Cheers! Maxwell's electromagnetism extended to smaller scales

More than one hundred and fifty years have passed since the publication of James Clerk Maxwell's "A Dynamical Theory of the Electromagnetic Field" (1865). What would our lives be without this publication?

It is difficult to imagine, as this treatise revolutionized our fundamental understanding of electric fields, magnetic fields, and light. The twenty original...

Im Focus: Highly charged ion paves the way towards new physics

In a joint experimental and theoretical work performed at the Heidelberg Max Planck Institute for Nuclear Physics, an international team of physicists detected for the first time an orbital crossing in the highly charged ion Pr⁹⁺. Optical spectra were recorded employing an electron beam ion trap and analysed with the aid of atomic structure calculations. A proposed nHz-wide transition has been identified and its energy was determined with high precision. Theory predicts a very high sensitivity to new physics and extremely low susceptibility to external perturbations for this “clock line” making it a unique candidate for proposed precision studies.

Laser spectroscopy of neutral atoms and singly charged ions has reached astonishing precision by merit of a chain of technological advances during the past...

Im Focus: Ultrafast stimulated emission microscopy of single nanocrystals in Science

The ability to investigate the dynamics of single particle at the nano-scale and femtosecond level remained an unfathomed dream for years. It was not until the dawn of the 21st century that nanotechnology and femtoscience gradually merged together and the first ultrafast microscopy of individual quantum dots (QDs) and molecules was accomplished.

Ultrafast microscopy studies entirely rely on detecting nanoparticles or single molecules with luminescence techniques, which require efficient emitters to...

Im Focus: How to induce magnetism in graphene

Graphene, a two-dimensional structure made of carbon, is a material with excellent mechanical, electronic and optical properties. However, it did not seem suitable for magnetic applications. Together with international partners, Empa researchers have now succeeded in synthesizing a unique nanographene predicted in the 1970s, which conclusively demonstrates that carbon in very specific forms has magnetic properties that could permit future spintronic applications. The results have just been published in the renowned journal Nature Nanotechnology.

Depending on the shape and orientation of their edges, graphene nanostructures (also known as nanographenes) can have very different properties – for example,...

Im Focus: Electronic map reveals 'rules of the road' in superconductor

Band structure map exposes iron selenide's enigmatic electronic signature

Using a clever technique that causes unruly crystals of iron selenide to snap into alignment, Rice University physicists have drawn a detailed map that reveals...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

The Future of Work

03.12.2019 | Event News

First International Conference on Agrophotovoltaics in August 2020

15.11.2019 | Event News

Laser Symposium on Electromobility in Aachen: trends for the mobility revolution

15.11.2019 | Event News

 
Latest News

Weizmann physicists image electrons flowing like water

12.12.2019 | Physics and Astronomy

Revealing the physics of the Sun with Parker Solar Probe

12.12.2019 | Physics and Astronomy

New technique to determine protein structures may solve biomedical puzzles

12.12.2019 | Life Sciences

VideoLinks
Science & Research
Overview of more VideoLinks >>>