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New imaging method tracks cystic fibrosis drug Special

By Tim Sandle     Apr 12, 2017 in Science
A new imaging technique called hyperpolarized helium MRI scanning could aid in the development of improved therapies for lung diseases like cystic fibrosis.
Cystic fibrosis is an inherited condition (a genetic disease) whereby the lungs and digestive system can become clogged with thick, sticky mucus. The condition can also affect the pancreas, liver, kidneys, and intestine. For those with the condition, long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. In the northern hemisphere the disease affects about one out of every 3,000 newborns.
There is currently no cure for cystic currently has no cure. However, a relatively new drug, approved by the U.S. Food and Drug Administration (FDA), treats the underlying cause of the disease. However, the drug’s effectiveness for each individual is unknown. This is the motivation behind the development of the new imaging technique.
The new method comes from the Missouri School of Medicine. Scientists have developed an imaging technique using a specific form of helium. This is to measure the drug’s effectiveness. Researchers hope the finding will lead to improved therapies for cystic fibrosis as well as for other lung conditions. Explaining the drug, principal scientist Dr. Talissa Altes told Digital Journal: "The drug ‘ivacaftor’ targets this defective protein, but to what extent it is successful is not well understood. Our study sought to use a new way of imaging the lung to understand how well the drug is working in patients with a specific gene mutation known as G551D-CFTR."
The current way to assess lung function is via a test called spirometry. With this patients blow through a tube and the results are tracked over time (a paper by Antonella Chesca and Tim Sandle explains more about the technique). For pediatric patients computerized tomography scans can be used (because younger people have difficulty with the spirometry test); however these methods only provide structural images of the lungs without showing gas or airflow.
With the new approach, the researchers used a gas called helium-3. This acts as a contrast agent for use with magnetic resonance imaging to visualize lung function. To verify the technique a study was run. One group of patients were given the ivacaftor drug or a placebo over four weeks (a test for short-term effectiveness). In a second study patients were administered ivacaftor for 48 weeks to determine long-term effectiveness. The patients performed a spirometry test and underwent MRI imaging using hyperpolarized helium, at periodic intervals to assess the effectiveness of the treatment.
It was found that those given the drug ivacaftor experienced a significant increase in lung improvement over the short and long term. This was seen most clearly with the helium-3 gas used as a contrast agent in conjunction with the MRI scan. The findings are published in the Journal of Cystic Fibrosis, with the research paper titled "Use of Hyperpolarized Helium-3 MRI to Assess Response to Ivacaftor Treatment in Patients with Cystic Fibrosis."
More about Cystic fibrosis, Imaging, Drug, Lungs, Medicine
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