Thalidomide is the drug behind one of the largest medicine linked disasters of the twentieth century. Back in the late 1950’s/early 1960’s, it was used to treat severe morning sickness in pregnant women in Europe, resulting in 10 thousand children being born with missing limbs.

Apparently, one of the side effects of the medicine was to stop new blood vessels from growing; in the developing fetus, this meant that the limbs being formed at the time the medicine was taken resulted in the limb not growing.

But thalidomide is also being used for other diseases. The most common use is to treat painful nodules that are related to an immune response during treatment for leprosy (aka Hansen’s disease).

These nodules, a form of Erythema Nodosum, can occur with other diseases (I’ve seen a couple of cases, usually from a common strep infections, but in the US it is also seen with Tuberculosis and sarcoidosis), but in patients with Hansen’s disease, they can be common, and if not treated, result in amyloidosis, arthritis, nerve damage, eye problems and other complications.

An 8-year case-control study of 649 patients noted a 19% overall prevalence of ENL in patients with leprosy treated with multidrug therapy, with 45% of patients experiencing recurrence after therapy was stopped. About 90% of ENL reactions occurred within the first 2 years of multidrug therapy.[10] It is important that the reaction be treated specifically on diagnosis, as it could progress to neuritis, arthralgias, leukocytosis, lymphadenitis, iridocyclitis, orchitis, or nephritis. Secondary amyloidosis is also a complication of chronic ENL.[5] Up to 2 million people worldwide are permanently disabled by leprosy even after the disease has been treated.[7]

So thalidomide is a valuable medicine, but since some of those treated are young women who can get pregnant, there is a worry about a new epidemic of limbless children in those using the medicine.

Hanson’s disease is rare in the United States, but Thalidomide is being used to stop “graft versus host” reactions.  This is when a person gets a bone marrow transplant, but the new bone marrow starts attacking the person’s mucus membrane, causing sores and other problems.

Both these diseases are usually seen by specialists (even in Africa, Hansen’s disease treatment is often supervised by specialists who travel from village to village to check on patients).

But there is another disease where Thalidomide can be used, and that is a nasty form of cancer called Multiple Myeloma. 

Multiple Myeloma is cancer of the plasma cells in the bone marrow, and as it spreads, it starts growing into the bones, causing fractures and severe bone pain. It’s a painful cancer, and people can develop other problems from the disease: problems with calcium, and since plasma cells produce antibody protein,  complication is too much protein in the blood causing clotting problems, or even kidney failure from too much protein in the urine (Myeloma kidney).

So how does it work?

The antiangiogenic effect of thalidomide was hypothesized to account for much of its benefit in multiple myeloma, a disease associated with elevated plasma levels of vascular endothelial growth factor and fibroblast growth factor.

Okay, got that? Think of it as stopping the roots from growing so the plant (fast growing plasma cells of the myeloma) stops getting larger.

When I was still in practice, one of our elderly diabetics developed multiple myeloma, and was placed on Thalidomide. His oncologist had to special order it, and he had to sign a lot of papers to guarantee that he wouldn’t get pregnant, and that he would use a condom so that if his elderly wife got pregnant she wouldn’t be exposed to the drugs in his “bodily fluids”.

Alas, he developed problems from the Thalidomide: the mild sleepiness he could handle, but he developed the rash side effect of the medicine that we had to treat. But it seemed to help: his bone pain improved, and he was able to remain living at home with his wife and family.

When I retired, his myeloma and bone pain was in remission, but he had just started dialysis for diabetic kidney failure, while his wife’s heart disease was slowly deteriorating.

When I discussed his wife’s tests with her cardiologist, I mentioned her husband’s health problems, and we both sighed, because they were the nicest couple, and we all knew that all we could offer was to try to keep them comfortable for the limited time that they had.

When I left to come to the Philippines, they gave me a figurine of an angel, so that I would remember them, which I do. I can’t help looking at it and be reminded that, even if we can’t cure our patients, nevertheless they thank us for just trying to help.

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Nancy Reyes  is a retired physician living in the rural Philippines. Her website is Finest Kind Clinic and Fishmarket, and she writes medical essays at HeyDoc Xanga Blog.

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