Study shows new medication effectively treats underlying cause of cystic fibrosis

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.

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UCSF researchers identify promising new treatment for childhood leukemia

An experimental drug lessens symptoms of a rare form of childhood leukemia and offers significant insight into the cellular development of the disease, according to findings from a new UCSF study. The mouse model research could spearhead the development of new leukemia therapies and paves the way for future clinical trials in humans.

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Protein involved in cystic fibrosis also plays role in emphysema, chronic lung disease

Contact: Ekaterina Pesheva
epeshev1@jhmi.edu
410-516-4996
Johns Hopkins Medical Institutions

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Novel drug offers hope for early intervention in cystic fibrosis patients

Cystic fibrosis (CF) patients with normal to mildly impaired lung function may benefit from a new investigational drug designed to help prevent formation of the sticky mucus that is a hallmark of the disease, according to researchers involved in a phase 3 clinical trial of the drug. Called denufosol, the investigational medication can be given early in the CF disease process, and may help delay the progression of lung disease in these patients, the researchers found.

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Novel approach shows promise for cystic fibrosis, say UAB researchers

Birmingham, Ala. – An investigational drug targeting a defective protein that causes cystic fibrosis has been shown to improve lung function in a small study of CF patients, according to findings published Nov. 18, 2010, in the New England Journal of Medicine. The investigational drug, VX-770, appeared to improve function of what is known as CFTR–the faulty protein responsible for CF. It is among the first compounds being developed for CF that specifically targets the root cause of cystic fibrosis.

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Common strain of bacteria found in patients with cystic fibrosis in Canada

A common transmissible strain of the bacteria Pseudomonas aeruginosa has been identified among cystic fibrosis (CF) patients in Canada, suggesting that cross-infection has occurred widely between CF centers in the United Kingdom and Canada, according to a study in the November 17 issue of JAMA. Infection with this strain among Canadian CF patients has been associated with an increased risk of death or lung transplantation.

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Arsenic early in treatment improves survival for leukemia patients

WINSTON-SALEM, N.C. – Thursday, Nov. 11, 2010 – Arsenic, a toxic compound with a reputation as a good tool for committing homicide, has a significant positive effect on the survival of patients with acute promyelocytic leukemia (APL), when administered after standard initial treatment, according to a new, multi-center study led by a researcher at Wake Forest University Baptist Medical Center.

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Growth defects in cystic fibrosis may start before birth

A new study using a pig model of cystic fibrosis (CF) suggests that low levels of a growth promoting hormone at or before birth may contribute to growth defects in patients with CF.

The study, led by University of Iowa researchers and published online the week of Nov. 8 in the Early Edition of the Proceedings of the National Academy of Sciences, could help predict the severity of the disease in patients and may lead to new therapies for growth defects in people with CF.

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1 step closer to a drug treatment for cystic fibrosis, MU professor says

Study recognized for significance and importance in the world’s most common genetic disease

COLUMBIA, Mo. – A University of Missouri researcher believes his latest work moves scientists closer to a cure for cystic fibrosis, one of the world’s most common fatal genetic diseases.

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Research identifies a new bacterial foe in cystic fibrosis

Exacerbations in cystic fibrosis (CF) may be linked to chronic infection with a bacterium called Stenotrophomonas maltophilia, which was previously thought to simply colonize the CF lung. The finding that chronic infection with S. maltophilia is independently linked with an increased risk of exacerbations gives clinicians and researchers a new potential measure of the health status of CF patients, as well as a new potential target in fighting their disease.

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