As more treatment options for cystic fibrosis have become available, researchers have found a way to match each patient with their best individual treatment option.
A team from the University of North Carolina (UNC) have developed a new test based on small balls of fluid found on the inside of the nose that predicts the best treatment for each cystic fibrosis patient.
“Any given drug may not be the drug that works best for a given patient, because there’s so much variation from person to person,” said Dr. Jennifer Guimbellot, assistant professor of pediatrics at the University of Alabama at Birmingham, who conducted the research at UNC, in a statement.
“We still have a long way to go to get a really optimized therapy for most cystic fibrosis patients and the only way we can do that is to have a model like ours, where we can take cells from each individual patient and test them with each individual drug to find out which one is the best match.”
Cystic fibrosis occurs when people do not have a functioning version of the CFTR gene. However, the condition, which involves the buildup of thick, sticky mucus in the lungs, is tied to more than 2,000 genetic mutations, making it difficult for doctors to prescribe the correct treatment for each patient.
Recent years have brought hope in the form of several drugs known as CFTR modulators that counteract the effects of mutations in certain cystic fibrosis-linked genes.
The new drugs tend to get to the root cause of the disease by helping the body’s cells maintain the proper level of hydration, which allows mucus to move freely in the lungs and other organs.
Currently, the only test available to predict how a patient will respond to CFTR drugs requires a rectal biopsy, followed by complicated laboratory manipulation of the sample to determine the drug’s likely effectiveness.
After noticing balls forming from cells on the inside of the nose, the researchers realized that the balls–called nasospheroids–were filled with fluid. Fluid is at the heart of cystic fibrosis, since hydration of nasal and lung surfaces is what determines whether mucus is slippery or sticky.
“When CFTR is turned on, the balls shrink down—mediated by the transport of salt and water–and we can quantify that by measuring the size of the balls,” Guimbellot said.
The results suggest that growing nasospheroids from nasal samples could provide a quick screening method to determine how a patient’s cells react to different CFTR drugs.
“It is a relatively simple procedure that doesn’t require any anesthesia and uses a brush that costs a few dollars,” Martina Gentzsch, Ph.D., an associate professor of cell biology and physiology at UNC, said in a statement.
Guimbellot said the next step will be to validate the approach for clinical application so that they can discriminate between small changes and further study to see if it will predict for individual patients what their actual clinical outcomes would be.
