Thalidomide May Help Patients With Deadly Lung Disease

Thalidomide, known for its birth defect risk, may improve life for some.

September 17, 2012, 3:26 PM

Sept. 17, 2012— -- For Diane Gwartney, every waking minute was a battle against the urge to cough.

"Just try to imagine coughing all day long until it hurts your ribs," said Gwartney, a 72-year-old retired English professor. "Trying to conduct a class while you're coughing is difficult and embarrassing."

William Chambers, a 62-year-old businessman, also had a severe cough. His was so troubling, he recalled, "that I would arrange my schedule so I wouldn't have to talk so much anymore... even dinner conversation would be embarrassing."

Gwartney and Chambers both thought they had a lingering cases of bronchitis before extensive testing traced their coughs to idiopathic pulmonary fibrosis (IPF). It's a disease that kills an estimated 40,000 people every year -- about as many people as breast cancer does.

Now, a new study out of Johns Hopkins University School of Medicine suggests that the key to improving the lives of patients with IPF may be a drug with an infamous history -- thalidomide.

Marketed in the 1950s by a German-based company, Gruenenthal, for the treatment of morning sickness in pregnancy, thalidomide was linked to more than 10,000 often-horrific cases of birth defects worldwide. Many children were born with improperly formed limbs, bones and internal organs.

The drug was finally withdrawn in 1961, and Gruenenthal recently issued an apology for its harmful effects in late August.

But since it was withdrawn from medical practice more than half a century ago, thalidomide has been attracting attention from doctors as a possible treatment for a broad range of disorders, such as multiple myeloma, leprosy, and Crohn's disease.

The small trial from Johns Hopkins, published Monday in the Annals of Internal Medicine, hints that thalidomide may be effective in improving the lives of IPF patients as well -- a group for whom good news has been in relatively short supply.

IPF is a progressive, often fatal lung disease affecting people over the age of 40, in which normal lungs begin slowly to become stiff and scarred over time. It currently has no known cause and no cure short of a lung transplant. The average survival after diagnosis is 3 to 5 years, and up to 80 percent of patients experience a debilitating cough.

Thalidomide does not appear to treat the disease itself, but it may make it much more bearable.

Gwartney and Chambers enrolled in the Johns Hopkins study hoping for some sort of relief. They were among 20 IPF patients who were given thalidomide or a placebo for three months and asked to report their symptoms and quality of life.

All of the patients were then switched from thalidomide to placebo or vice versa for another three months, and asked to report how they did.

The researchers found that in patients taking the drug, frequency of coughing decreased by about 63 percent on average. The patients' quality of life -- in other words, their ability to do daily activities -- improved by about 20 percent.

"What is exciting about the trial for me is that, the data are so dramatic and this is the first [drug] trial in IPF to show any successful outcome," said lead study author Dr. Maureen Horton, an associate professor of medicine at Johns Hopkins University School of Medicine. "Given the [lack] of treatments we have for any aspect of this disease, it's good to have anything that can make their lives better."

Horton said the patients involved in the trial "were originally in shock" when they heard they would be taking the notorious drug. But afterward, both Gwartney and Chambers described their improvement as remarkable.

"[Thalidomide] stopped the cough in its tracks," said Gwartney. "As far as [treating] the symptoms go, it's been a lifesaver."

Chambers echoed these sentiments; for him, the drug reduced his coughing up to 90 percent.

"I have a lot more confidence and ability to live up to my professional expertise," he said. "[Thalidomide] has been a godsend for me, and something that I have been blessed with."

However, thalidomide does not come without risks. Aside from relatively minor side effects like constipation, malaise, and dizziness, the drug is also associated with tingling in the extremities. More importantly, the drug still poses a threat for birth defects when used in women of childbearing age.

All of the patients in this trial were over the age of 50, and none were going to have children. Still, Chambers said that the drug company conducted a monthly interview with him to ensure that he did not expose any pregnant or child-bearing women to the drug.

"There's a lot of reasons for people to stay away from this drug," Chambers said.

Dr. Micah Bhatti, a fellow in infectious disease at the University of Chicago Medical Center, said he thought patients "would be open to the use of thalidomide if they've exhausted other treatment options and there is a frank and open discussion about the known side effects."

All of the patients in the study -- Gwartney and Chambers included -- requested to stay on the drug after the trial was over, despite its potential risks. They have both been taking it now for nearly two years.

Gwartney said that she feels that new treatments designed actually to treat her disease will probably come too late to help her. The disease, she said, continues to progress.

"But that's not the fault of the thalidomide," she said. "That, at least, makes each day livable."

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