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Experimental asthma drug helps untreatable patients in study

Asthma suffers who aren't helped by existing medications may have reason to breathe a sigh of relief.

An experimental drug called dupilumab was shown to reduce asthma attacks and helped boost lung function for people with moderate to severe asthma who can't control their symptoms with conventional medicine like inhalers.

"Overall, these are the most exciting data we've seen in asthma in 20 years," Dr. Sally Wenzel, lead investigator for the study and director of the Asthma Institute at the University of Pittsburgh in Pa., told Reuters.

The research was presented May 21 at the American Thoracic Society 2013 International Conference in Philadelphia, and was also published that day in the New England Journal of Medicine.

About 25 million Americans suffer from asthma,a chronic lung disease that inflames the airways, leading to wheezing, coughing, chest tightness and shortness of breath, the National Institutes of Health reports. About 7 million U.S. asthma sufferers are children.

Dupilumab is an experimental drug developed from Sanofi and Regeneron Pharmaceuticals, the sponsors of the new study. It is an injectable drug known as a "monoclonal antibody," which is designed to trigger an immune system response. This drug targets two inflammatory compounds -- or cytokines -- called interleukin-4 and interleukin-13, which lead to an immune response that triggers asthma symptoms.

Some monoclonal antibody treatments are used in cancer care.


The experimental trial involved 104 patients between 16 and 65 years of age who all had moderate to severe asthma that could not be adequately controlled by inhaled glucocorticosteroids (ICS) and long-acting beta agonists (LABA), medications often prescribed to treat asthma. About 20 percent of patients who use these two drugs in combination still have problems controlling their asthma, according to background information in the study.

Patients received either the injectable drug or a placebo in addition to sticking to their twice-daily asthma medications over a 12-week period, and were followed up with for another eight weeks after. At week four of the study, they were told to stop taking the LABA, and they were weaned off the ICS between weeks six and nine.

The researchers saw an 87 percent reduction in asthma attacks was seen in those who received dupilumab when compared with the placebo group. Patients in the dupilumab group also showed significant improvements in lung function, peak flow measures and reductions of asthma symptoms, even once they stopped using their inhalers.

"Intriguingly, dupilumab showed substantial efficacy in objective and patient-reported endpoints when added to ICS and LABA and when those therapies were discontinued," Wenzel said in a statement.

More side effects were reported in the dupilumab group, including injection site reactions, nausea, headache and swelling of the nasal passages, but the side effects were not considered severe.

In an accompanying editorial in the same journal issue, Dr. Michael E. Weschler, director of the asthma program at National Jewish Health in Denver, writes that at first glance the 87 percent drop in asthma attacks is "outstanding." But, he said upon closer examination, there was no significant difference in asthma attacks between the dupilumab group and placebo group during the weeks when both groups were taking their asthma medications.

Given that, he's not sure if the drug will be advantageous in a "real world setting."

"Longer studies comparing dupilumab with placebo as add-on therapy to inhaled glucocorticoids and LABAs, without withdrawal, are necessary to show the clinical usefulness and durability of potential benefits of this drug," Weschler wrote.