Pulmonary Medicine

Protein Assist and Chloride Channel Therapies: Phenylbutrate and Genistein

Phase I pilot study of phenylbutyrate and genistein to correct CF nasal ion transport
Children's Hospital of Philadelphia, University of Pennsylvania


Researchers have evidence, both from the laboratory and clinical trials, that phenylbutyrate appears to enable the defective protein made by the CF gene to move to the correct place in the cell. There, it has potential to function as a chloride channel. Genistein further activated the chloride channel.The two drugs will be evaluated together as a "duotherapy" for the first time. One of the study drugs, phenylbutyrate, will be given orally over a week's time, and on the first and last days of this week, the genistein will be dripped into the nose during nasal potential difference testing. Nasal potential difference testing will also occur in the midst of phenylbutyrate dosing (on day 4), and weekly for two weeks after phenylbutyrate dosing.

This is a randomized, placebo-controlled study funded through the Cystic Fibrosis Foundation Therapeutics, Inc.


Twelve adults with CF (age 18 years and older) are needed to participate. Volunteers must be homozygous for the DF508 CF mutation. Patients with a broad range of pulmonary function are eligible. A total of six visits are required, each lasting about 3 to 4 hours, over a 4 to 5 week period. Compensation for time spent in the study will be offered.

For more information, please contact:

Study coordinator
Maria L. Skotleski-Irwin

Reviewer: Jennifer Long
Date: January 21, 2004

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