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The Long, Bumpy Path Toward Effective Cystic Fibrosis Treatment

Danny Bessette in 2009 holding the magazine that featured him on the cover as a child. (Photo: Steve Barrett/Science)

By Karen Weintraub
Guest Contributor

When he was 4, Danny Bessette was featured on the cover of Science Magazine. His was the new face of cystic fibrosis. A disease, recently lethal to children his age was now on the verge of being beaten, the coverage suggested. After all, the gene had just been discovered.

Twenty years later, he was featured in Science again, symbolizing the painful scientific realization that discovering a gene is still a long way from developing a cure.

Danny Bessette is 27 now, and if he still represents something larger than himself it’s the patience and fortitude that he and others touched by disease have had to exhibit during the ups and downs of scientific discovery.

And yet levels of optimism today are probably as high as they’ve been since 1989. Drugs developed out of the understanding of how the gene works are just now reaching patients. They have brought dramatic improvements to some. It’s not yet clear whether they’ll be able to help Bessette. Continue reading

Report: Vertex Overstated Cystic Fibrosis Drugs Benefit

(BuzzNewsTrends/flickr)

The Boston Globe reports that Cambridge-based Vertex Pharmaceuticals overstated the benefits of a new drug combination to treat cystic fibrosis when it released interim clinical trial data earlier this month.

Rob Weisman writes:

Shares of Vertex Pharmaceuticals Inc. plunged more than 18 percent in the first hour of trading Tuesday after the company acknowledged interim clinical trial data it released earlier this month for an experimental drug combination treating cystic fibrosis was in error.

In a statement correcting its earlier report on an ongoing midterm study, Cambridge-based Vertex said the interim data — which sent Vertex’s stock up more than 55 percent on May 7 — showed relative improvements in breathing for patients taking the drug in a clinical trial, not absolute improvements as the company previously reported. Continue reading

Special Report: New Cystic Fibrosis Drug Brings Gift Of A Future

[Note: This is a “good news” story. It describes the scientific road to the first drug that successfully attacks the underlying defect in cystic fibrosis, bringing dramatic improvements. But the drug appears to work for only 4% of cystic fibrosis patients. CommonHealth plans to write next about the remaining majority, their lives and their prospects.]

The snowdrifts towered before her, taller than she was, dumped by yet another of last winter’s blizzards. The snowblower was broken. Her husband has a bad back. So Roe Van Epps picked up her shovel. When she had cleared her entire driveway, she turned to find her husband behind her, tears in his eyes.

Her first thought was that he was going to critique her shoveling. “What’s wrong?” she demanded.

Nothing was wrong. “Oh my God, you’re shoveling!” he said.

It sank in. “Oh my God!” she echoed.

In her entire 41 years, Roe had never been able to shovel snow. Or to run. Or to go a full winter without getting ill enough to need weeks of intravenous antibiotics.

She was born with cystic fibrosis, a genetic disease that affects 30,000 Americans, gumming up their lungs with dangerously thick mucus that tends to breed bacteria. At birth, doctors told her parents that her life expectancy was age five. Along with school and play, her youth consisted of hospital beds and piles of pills and hours each day of inhalation therapy.

Medical treatments that continually improved in small steps, from new antibiotics to improved enzymes, kept her alive. But she remained a person without a future, told at each life stage not to expect the next. Her husband planned for retirement. She did not.

Now, because of a still-experimental drug called VX-770, made by Cambridge-based Vertex Pharmaceuticals, that has changed.

Roe clearly has a spirit as sparkly-bright as a Roman candle. When she came to speak to the cystic fibrosis team at Children’s Hospital Boston recently, she wore zebra-print stilettos that made the same fun-wild statement as the many shades of red in her hair. But she is openly bewildered by the new possibility of living out a full lifespan.

“Now I’m like, holy cow, I might be 80!” she said. “Maybe I should start using some face cream!” It’s almost like she’s a different person: “I still have cystic fibrosis but I can do things, I can live my life.”

Roe does not use the word “cure.” No one does when they talk about VX-770. Dr. Greg Sawicki, a cystic fibrosis specialist at Children’s, says he would definitely call it a “major advance,” the first drug to come this far that attacks the basic defect in cystic fibrosis. More cause for optimism? Yes. But a cure? No.

Roe still takes antibiotics and does hours of inhalation therapy every day. She doesn’t dare stop. But “this is life-changing,” she said. “I’m very, very careful, I’m trying not to get excited, but I really am, at the same time.”

This is two stories, intertwined. One is Roe’s life with cystic fibrosis and then on VX-770. The other is a tale of amazing science — and a gamble of hundreds of millions of dollars that hit the medical jackpot. Researchers tell it with a hint of disbelieving awe in their voices: For once, nature played no tricks. For once, everything came together just as it was supposed to, from theory to test-tube to human patients. Continue reading

FDA Staffers: Vertex Hep C Drug More Effective Than Current Therapies

One step closer to market?

The Boston Globe reports that Cambridge-based Vertex Pharmaceuticals is one step closer to getting its first drug on the market:

A proposed drug developed by Vertex Pharmaceuticals Inc. cures more patients with the hepatitis C virus in less time than existing therapies, according to a briefing document filed today by Food and Drug Administration staffers who reviewed the new drug application.

The drug, called telaprevir, would be used in combination with two current therapies. Together, the drug “cocktail” proved more effective in late-stage clinical trials than the older drugs by themselves, the briefing paper said. The document was prepared in advance of a day-long hearing Thursday by an FDA advisory committee that will recommend whether the agency should approve the new drug.

Tomorrow the same FDA advisory committee will consider a rival hepatitis C treatment, boceprevir, developed by Merck & Co. Both drugs act to inhibit the protease enzyme that enables the virus to reproduce, and both target a market projected by some securities analysts to exceed $2 billion annually this year and in 2012.