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.

 

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  1. frank frank
    Reply

    NearestToilet app is a big help. In case you’ve an Android phone, it’s helpful to stay connected with nearest toilets: http://bit.ly/K6yFbF. It’s great to have access to 3 toilet maps this app offers when traveling. It rings when near a recommended/ thumbs up/ clean toilet. See more at http://NearestToilet.com

  2. Greg L.
    Reply

    This is another example of how every bit of research matters. While the side effects are a major concern, studying thalidomide as a way to treat Crohn’s disease could bring researchers closer to finding help for patients. A very interesting study.

  3. BP
    Reply

    You say that women and girls who take Thalidomide should never become pregnant. Does that mean even when they are no longer taking the drug? Does it have lasting consequences?

  4. JB
    Reply

    Does People’s Pharmacy have access to the additional data not just the abstract? I’m interested if the children were or were not screened for vitamin D status before the study, as D3 deficiency is very common in adults with Crohn’s. I know of at least one particular study showed a decrease in crohn’s attack with D3 supplementation over a 12 mos time period.
    People’s Pharmacy response: They were not screened for vitamin D.

  5. SJ
    Reply

    Number of children in study was 56. By providing the number in the study we are better able to judge the strength of the study.
    PEOPLE’S PHARMACY RESPONSE:
    This was a small study, but the the statistics were quite fascinating. The risk ratio was 4.0 for clinical remission with thalidomide. What that means is 4 times as many children taking thalidomide had a remission of symptoms compared to those on placebo. Those are pretty startling stats.
    Even more interesting was the NNT (number needed to treat). These researchers calculated that to be 2.86. In other words, for one child to get benefit you would need to treat almost 3.
    That might not seem impressive, but a recent analysis of statin-type cholesterol-lowering drugs found that “140 low risk people must be treated with statins for five years to prevent one major coronary event or stroke, without any reduction in all-cause mortality.” (published in BMJ Oct, 22, 2013).
    http://www.bmj.com/content/347/bmj.f6123?view=long&pmid=24149819
    We bring this to your attention only because in the BMJ article the number needed to treat is 140 to get one positive outcome over five years. A NNT of 2.86, on the other hand, is quite impressive.
    That said, we will need much more research into the effects of thalidomide for Crohn’s disease. The peripheral neuropathy complication is a real problem. Hopefully, this study will spur more research and may also open the door to drug development of better compounds for this hard-to-treat condition.

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