No drug in the world that has been more vilified than thalidomide. That is understandable, since it caused so much misery. In the 1950s thousands of babies were born with severely deformed limbs and other malformations after their mothers used thalidomide during pregnancy. Thalidomide represented one of the worst drug scandals in history.
Thalidomide (Thalomid) has been resurrected from the ashes. During the 1990s the FDA approved Thalomid for a rare form of leprosy. At most, 100 patients with this condition might have benefited from thalidomide in the U.S.
But the company that helped to revive thalidomide had another (much bigger) target in mind. Multiple myeloma is a life-threatening blood cancer. It wasn’t long before cancer doctors began prescribing the drug off-label. Eventually the FDA approved Thalomid for this hard-to-treat cancer. The drug revolutionized the treatment of multiple myeloma and set the stage for even better anticancer medications.
At the time thalidomide was under investigation for these new uses, we talked to the person who was spearheading this innovative research. Sol Barer, PhD, was Chairman and Chief Executive Officer of Celgene, the company behind the resurrection of thalidomide. He told us that this unique drug affected the immune system and might be beneficial for conditions other than leprosy and multiple myeloma. In addition to rheumatoid arthritis, Sol told us that he thought thalidomide might work surprisingly well against other hard-to-treat conditions such as Crohn’s disease.
We were open to such concepts but wanted to see data before becoming too enthusiastic. Crohn’s is an inflammatory bowel disease that can be life threatening. Current treatments have unpleasant side effects and are not always that effective. Sometimes people have to have their colons removed surgically to survive.
Enter thalidomide, 2013! It turns out that Dr. Barer was right. It took a long time, but a study in JAMA (Nov. 26, 2013) demonstrates that thalidomide can be surprisingly effective against Crohn’s disease in children and adolescents.
The Italian kids in this trial were not responding to traditional treatment. They were randomized to receive either placebo or thalidomide. 46.4% of the kids who got thalidomide had remission of their symptoms after four weeks, compared to 11.5% of the kids on placebo. After eight weeks, improvement was even greater. The children on placebo who did not improve were switched to thalidomide and over half of them also achieved remission within two months. Nearly two-thirds of the children who received thalidomide in the study experienced great improvement in their condition. Those are impressive numbers.
According to the researchers who did this study, “As many as 1.2 million people in Europe and more than half a million in the United States are estimated to have Crohn disease.” They point out that the incidence of this inflammatory bowel disease is increasing and that there are serious consequences, especially in children. They include impaired growth, osteoporosis, sexual and psychological problems.
Thalidomide is not without side effects, and some of them are serious. Some of the children in the Italian study developed peripheral neuropathy, known as a possible response to the drug. Thalidomide can also result in an unusually slow heart rate (bradycardia) and may interfere with a normal menstrual cycle. The scientists did not find any dangerous blood clots occurring in the study children, but this too has been reported from other studies. Of course the most horrific side effect is the possibility of birth defects. No woman who takes thalidomide should ever become pregnant.
The bottom line is that this study should open the door for further research into the potential immune modulating affects of thalidomide for inflammatory bowel disease and possibly other autoimmune conditions.