Researchers at the Johns Hopkins University School of Medicine have published findings that indicate thalidomide can significantly reduce the disabling cough seen in people with idiopathic pulmonary fibrosis (IPF).
IPF is a progressive, fatal lung disease that causes a person's lungs to become stiff and scarred. This prevents oxygen from getting out of the lungs and into the rest of the body. The cause of the disease is unknown, and about 80,000 people in the U.S. have been diagnosed with it. Most people with IPF have a dry cough for which there is currently no treatment, other than a lung transplant.
“The constant cough caused by the disease can affect the quality of life in many ways,” said Dr. Maureen Horton, a pulmonary disease specialist and professor at Johns Hopkins University School of Medicine as well as the lead author of the study. “Some no longer go to church or to social gatherings because people think they are infectious. Other patients may have more pronounced urinary incontinence due to the cough, for example, so it has wide-ranging effects.”
Thalidomide is an anti-inflammatory drug that was used as a sleep aid in the 1950's. It was also used to treat morning sickness, but was pulled from pharmacy shelves in 1961 after it became clear the medication caused severe birth defects. Though the medication is not banned, it is now prescribed with strict controls. It is most commonly used to treat multiple myeloma and kidney cancer. Johns Hopkins states that the drug had not been studied for treating lung disease before.
The study, which has been published in the Annals of Internal Medicine, looked at 20 patients over the age of 50. Patients took either low doses of thalidomide or a placebo for three months, were off the medication for two weeks, and then the pills for thalidomide and placebo patients were swapped. "We performed a randomized, double-blind, placebo-controlled trial of thalidomide in patients with IPF to determine its effectiveness in suppressing cough,” said Horton
The small study found that patients on thalidomide reported their coughing had decreased about 63% and that their "respiratory-specific quality of life" had improved about 20%.
“Although the results were significant, this was a small study and we believe that a larger trial is warranted to confirm these promising results and also assess the drug’s impact on the disease itself,” said Horton. “We have some hope that this therapy may be able to slow the progression of IPF, but that would have to be tested in a larger study.”