Thalidomide, the morning-sickness drug linked to birth defects in the 1960s and revived decades later to fight cancer, helped with a debilitating cough tied to a fatal lung condition in a small study.
Coughing was reduced by about 63 percent for patients with idiopathic pulmonary fibrosis who were given thalidomide, according to a 20-person trial reported today in the Annals of Internal Medicine. While patients on the medicine experienced side effects including constipation, dizziness and malaise, each opted to keep taking thalidomide after the trial ended.
Researchers suspected the drug’s effects on the immune system may make it a potential treatment for IPF, though a previous trial wasn’t successful in determining its efficacy. The study did suggest thalidomide helps against the dry, debilitating cough that afflicts as many as 80 percent of IPF patients, said Maureen Horton, at Johns Hopkins University School of Medicine.
“We noticed the people on thalidomide, their cough went away,” said Horton, an associate professor of medicine and environmental health sciences at the Baltimore school and a study author. “They came to us and said, ‘Hey, you cured my cough.’”
The disease affects 80,000 to 100,000 people in the U.S., Horton said in a telephone interview. Its cause is unknown.
In today’s report, adverse events were found in 74 percent of patients on thalidomide, compared with 22 percent on placebo. Thalidomide is sold by Summit, New Jersey-based drugmaker Celgene Corp. (CELG) as Thalomid for multiple myeloma.
The company provided funding and supplied the drug for the study. It wasn’t involved with the trial design.
Celgene sells the drug under strict regulations to prevent pregnancies while patients are on therapy, said Greg Geissman, a spokesman for the company. Thalomid had $339 million in revenue last year. Celgene also sells a next-generation therapy, Revlimid, which drew $3.2 billion in 2011 sales for multiple myeloma and myelodysplastic syndromes.
The results warrant a larger study to evaluate thalidomide or Revlimid’s effects on cough in IPF, and potentially to assess whether they may work to treat the disease itself, Horton said. There’s no approved treatment for IPF in the U.S.; InterMune Inc. (ITMN), a biotechnology company based in Brisbane, California, sells Esbriet in the European Union and is in late-stage trials in the U.S.
The results published today “will encourage patients and frustrate them because thalidomide is not FDA-approved for this indication and they’re not going to be able to get this,” Horton said. “My goal is to gain enough interest” to do further studies, she said.
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