TUESDAY, Nov. 26, 2013 (HealthDay News) -- Thalidomide, a drug made infamous after it caused devastating birth defects in the 1950s, may help treat children with Crohn's disease who haven't responded to other medications, new research suggests.
After eight weeks of treatment, more than 46 percent of children taking thalidomide had reached remission, compared to about 11 percent of those given an inactive placebo. And remission lasted longer for the children on thalidomide -- an average of 181 weeks compared to just 6.3 weeks for those who took the placebo.
The Italian researchers published their results in the Nov. 27 issue of the Journal of the American Medical Association. Funding for the study was provided by the Italian Medicines Agency, while the drug's manufacturer, Celgene, provided the drug for the study.
Crohn's disease is an inflammatory bowel disease. It's estimated that about 500,000 people in the United States have Crohn's disease, and for about one-quarter of those patients, symptoms started in childhood, according to background information in the study. Pediatric cases of Crohn's disease tend to be more severe, and nearly one in five children with the disease will require surgery within five years of when the disease starts, the study noted.
Thalidomide was first used in the late 1950s as a treatment for insomnia, anxiety and morning sickness, according to the March of Dimes. It was approved for use in Europe, Japan, Canada and Australia, but never in the United States. By the early 1960s, it was apparent that the drug caused significant birth defects, including severely malformed or missing arms and legs. The medication was quickly withdrawn from the market.
It remained off the market for a significant time until researchers realized that the drug had other uses, particularly for dampening an overactive immune system. And if care was taken to ensure that no one who might be pregnant or become pregnant took the drug, its other side effects were less serious, according to the March of Dimes.
The U.S. Food and Drug Administration approved the use of thalidomide to treat a complication of leprosy, and the drug was approved in 2006 to treat multiple myeloma, a cancer of the bone marrow.
Thalidomide is currently being studied as a treatment for a number of inflammatory diseases, but in all cases where the drug is used, any woman of reproductive age must be counseled on the potential for serious birth defects should she become pregnant while on thalidomide. The March of Dimes reports that even one dose of thalidomide can cause birth defects. Males taking the drug should use condoms because thalidomide can also affect the sperm quality.
The current study looked at what effect, if any, thalidomide could have on Crohn's disease in children and adolescents. A total of 54 children with moderate to severe Crohn's disease were included in the study. All of the children had received other treatments for Crohn's that had failed to control their disease.
Twenty-eight children received thalidomide and 26 were given a placebo for eight weeks. Those who hadn't shown improvement while on placebo for eight weeks were switched to thalidomide for an additional eight weeks. Any of the children who responded to thalidomide therapy remained on it for at least 52 weeks, according to the study.
Children or teens taking thalidomide were more likely to achieve a remission of their disease, and the remission was likely to last far longer than a remission achieved while on placebo, the study found.
Side effects were infrequent. The most severe frequent side effect was a condition that causes strange sensations in the arms or legs. When someone stops taking thalidomide, the condition goes away. Thalidomide also can have a strong sedative effect, which can have less of an impact if given at night.
"As it turns out, the mechanism of thalidomide is very effective for exactly the targets we're trying to treat in Crohn's, and it might be helpful for sparing kids from high steroid use," said Dr. Jonathan Ramprasad, a pediatric gastroenterologist at McLane Children's Scott &White Hospital in Temple, Texas.
"But, it won't be a wonder drug. This wouldn't be a first-line treatment, or a treatment on its own. It would be used as an [additional] therapy," noted Ramprasad, who was not involved with the study. "The research is still early, and we need to see longer term follow-up."
Another expert, Dr. Michael Pettei, chief of pediatric gastroenterology at Cohen Children's Medical Center of New York in New Hyde Park, N.Y., agreed.
"While it's good to have another agent, more studies need to be done on this," he said.
"There are a large number of agents currently available for the treatment of pediatric Crohn's disease, and all have different benefits and side effect profiles. If additional studies confirm these findings, thalidomide might be an additional choice," Pettei added.
Both experts said that because the drug has been around for so long, treatment with thalidomide would likely be very affordable.
Children and teens could probably continue using the drug into adulthood, although when they reached reproductive age, they would need to be counseled on the very serious risks of birth defects should a pregnancy occur. And, counseling and the use of birth control methods are currently required for a prescription for thalidomide.
Learn more about the treatment of Crohn's disease in children from the Crohn's and Colitis Foundation of America.