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UCSF Researchers Drive Major Progress in Lung Disease

May 18, 2014
News Office: Pete Farley (415) 502-6397

Researchers from UC San Francisco played major roles in five significant multicenter studies of lung disease published May 18, 2014 in The New England Journal of Medicine (NEJM).

In one set of trials, three different drugs were tested in separate studies for their effectiveness in idiopathic pulmonary fibrosis (IPF), a progressive scarring disease of the lungs primarily affecting the elderly that has a 3-year survival rate of about 50 percent, a worse prognosis than that for many cancers. Two of these drugs were found to significantly improve IPF patients' symptoms, and one, pirfenidone, was shown to reduce the number of deaths by 68 percent in the group of patients who received the drug during the year-long trial.

Two additional trials assessed the effectiveness of statin drugs in acute respiratory distress syndrome (ARDS) and chronic pulmonary obstructive disease (COPD). Neither statin was found to provide benefit for patients with these conditions.

The findings of all five studies are being reported at the annual meeting of the American Thoracic Society, being held this year in San Diego, Calif., from May 16 to May 21.

Dr. Talmadge King

Talmadge E. King, Jr., M.D.

The pirfenidone trial was led by Dr. Talmadge E. King, Jr., professor and chair of UCSF's Department of Medicine, in his role as member of a research consortium known as the ASCEND Study Group. Though this drug, manufactured by Brisbane, Calif.-based InterMune, has been approved for IPF in other countries, it has not yet received approval from the U.S. Food and Drug Administration (FDA).

Dr. Harold R. Collard, associate professor of medicine and director of UCSF's Interstitial Lung Disease Program was a member of the INPULSIS Trial Investigators, a research group that tested the effectiveness in IPF of nintedanib, a drug that tamps down the formation of new blood vessels. This trial also reported a significant improvement in symptoms in patients receiving the drug.

Dr. Hal Collard

Harold R. Collard, M.D.

King also took part in a study of a compound known as acetylcysteine, conducted by the Idiopathic Pulmonary Fibrosis Clinical Research Network. That trial showed no significant improvement in symptoms among patients taking the drug.

There have been virtually no effective treatments for IPF, and the pirfenidone and nintedanib trials were hailed in an accompanying NEJM editorial as "a major breakthrough for patients," though it remains to be seen how well these drugs will work in patients who are more severely ill than those who qualified for the trials. But the success of pirfenidone and nintedanib, and the failure of acetylcysteine, which targets inflammatory responses, "mean that it is now clear that idiopathic pulmonary fibrosis is a disease perpetuated by aberrant wound healing, rather than primarily by chronic inflammation," writes editorial author Dr. Gary M. Hunninghake, of Brigham and Women's Hospital in Boston, and this new knowledge should lead to more new treatments.

In a fourth trial published in this week's NEJM, UCSF's Dr. Michael A. Matthay, professor of medicine and anesthesia, and Dr. Kathleen D. Liu, associate professor of medicine, took part in a study of the effectiveness of rosuvastatin (trade name Crestor) in ARDS. The trial, conducted by The National Heart, Lung, and Blood Institute ARDS Clinical Trials Network, failed to find any benefit from this treatment.

Finally, in a study of the effectiveness of simvastatin (trade name Zocor) in COPD, Dr. Stephen C. Lazarus, professor of medicine, Dr. Prescott G. Woodruff, associate professor of medicine, and members the COPD Clinical Research Network and the Canadian Institutes of Health Research did not establish any clinical benefit for the drug.

Though the findings of both statin trials were negative, these results are important, said an NEJM editorial entitled "Statin Strikeout." Both drugs, which have been approved by the FDA to reduce inflammatory responses in other conditions, have been given to patients on the theory that inflammatory mechanisms underlie both ARDS and COPD. Although the theory behind these "off-label" uses was reasonable, and clinical observations seemed to back up that theory, write Dr. Jeffrey M. Drazen and Annetine C. Gelijns, of the Icahn School of Medicine at Mt. Sinai in New York, double-blind trials were essential to establish whether these drugs really are effective in these lung diseases.

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