Cure for cystic fibrosis may be found in ocean

by Symptom Advice on October 31, 2011

By SEAN TEEHAN October 19, 2011 12:00 AM

Scientists in Woods Hole and at a Charlestown laboratory are looking under water to treat a disease that robs sufferers of the ability to breathe.

Woods Hole Oceanographic Institution and Flatley Discovery Lab have struck a $1.18 million deal to collaborate on a three-year project to find out if microbes from the ocean are the key to finding a drug to treat the underlying disorder behind cystic fibrosis.

In the ocean, which has a vast number of predators, minuscule microbes have little means to protect themselves from being devoured by creatures that are a link or two higher on the food chain.

“They have to use chemistry to defend themselves,” said Tracy Mincer, a WHOI microbiologist and chemist working on the project. “And they produce potent chemicals to do this.”

These chemicals could also work to correct hereditary defects in chronic illnesses such as cystic fibrosis.

Based on that possibility, the Flatley lab, a nonprofit cystic fibrosis research laboratory, is paying WHOI to extract chemicals from oceanic microbes for the experiments.

“It’s just become known over the past 50 years that microbes are a great source of chemical diversity,” Mincer said.

Afflicting about 30,000 people in the United States, cystic fibrosis is caused by a defective gene that creates a thick mucus that clogs the lungs and obstructs the pancreas, preventing it from absorbing food, according to the Cystic Fibrosis Foundation in Bethesda, Md. Those who suffer from the disorder are unable to efficiently clear their lungs of mucus, so it piles on and thickens like oatmeal left on the stove too long, said Laurie Fink, a spokeswoman for the foundation.

“People have a lot of trouble breathing, and they’re getting these terrible lung infections all the time,” Fink said. Those who suffer from more severe cases feel as if they are holding their noses and trying to breathe through a drinking-straw, she said.

Sufferers lose approximately 2 to 3 percent of their lung function each year and their median life expectancy is in the mid-30s, according to foundation data.

The Flatley Discovery Lab opened about two years ago with the goal of developing a drug that treats the underlying disorder of the disease rather than just symptoms, said Richard Fitzpatrick, Flatley’s chief operating officer and chief scientific officer.

Chemical samples from WHOI will be sent to the Flatley lab where scientists will study how cells afflicted with the disorder react to them, Fitzpatrick said. As tests go on, scientists plan to mix and match chemicals and hope to eventually develop a drug that could succeed in treating the cell deficiency.

Similar processes have led to discoveries of antibiotics such as streptomycin and Augmentin, said Mincer, who discovered a chemical with the potential to treat multiple myeloma, a type of cancer, while he was in graduate school at Scripps Institution of Oceanography at the University of California in San Diego. The chemical is currently in clinical trials.

“We’re on the verge of some breakthrough therapy,” Fink said, noting that there is currently a drug called VX-770 that recently completed its clinical trials and might be able to treat the disorder (rather than just its symptoms) for about 4 percent of cystic fibrosis sufferers. Vertex Pharmaceuticals, maker of the drug, plans to submit a new drug application to the FDA later this month.

Mincer said he believes if the ocean microbe project does not result in a cystic fibrosis breakthrough, it will at least yield good leads as to how to advance treatment.

“It’s really a new frontier for drug discovery,” Mincer said.

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