Antisense Drug May Prevent Asthma Symptoms

Preclinical studies of an inhaled antisense oligonucleotide (EPI-2010) suggest that it can prevent the body from manufacturing proteins that cause the symptoms of asthma. A phase I study is now under way.

EPI-2010 targets the adenosine A1 receptor, a unique mediator of asthma that is produced in excess in virtually all asthma patients. The A1 receptor appears to be involved in three effects of asthma that contribute to a patient's breathing difficulties: inflammation, bronchoconstriction, and surfactant depletion. By blocking the messenger RNA of the A1 receptor gene, EPI-2010 may be able to prevent all three deleterious effects of asthma.

The developer, EpiGenesis Pharmaceuticals Inc. (Cranbury, NJ, USA), has discovered that respirable antisense oligonucleotides are well suited for respiratory ailments. The company's researchers found that surfactant, a natural, lipid-rich film that lines the lung, effectively absorbs and distributes inhaled oligonucleotides, providing better results than other methods of administration. In animals models, the compound was found effective for nearly seven days and appeared to be broken down within the lung, causing no systemic toxicity.

"We eagerly await the results of the clinical trial,” said Jonathan Nyce, Ph.D., chairman and CEO of EpiGenesis. "We feel strongly that we are on the threshold of major progress against asthma--a disease that afflicts more than 100 million people worldwide.”