'Birth defect' drug thalidomide useful for lung disease

One of the biggest scandals in the history of medicine is that of thalidomide, or softenon as it is known in some countries. It was developed in the 50's as a drug for morning sickness, pain and to aid sleep, but it was later discovered that there are severe side effects. Pregnant women taking thalidomide gave birth to horribly deformed children, and the drug was consequently pulled from the market. However, the damage was already done, as thousands of babies were born with birth defects. Despite these horrible side effects, the drug has seen some renewed interest, as it has been shown useful in several conditions. A recent study shows that thalidomide might come in handy to treat a severe lung disease.

Lungs
Specifically, the researchers analysed the effect of thalidomide on the symptoms associated with idiopathic pulmonary fibrosis (IPF). This disease is characterized by tissue of the lung losing its function due to scarring. Idiopathic refers to the fact that this form of the disease has no known cause. Because there is no proper treatment available, and half of the patients die within three years of diagnosis, finding a cure is an important clinical need.

Thalidomide
Scientists from the Johns Hopkins University School of Medicine provided patients enrolled in the study with a placebo and thalidomide treatment, and analysed the effects it had on the symptoms of IPF. The rationale for providing patients with thalidomide is the fact that it is known to have an anti-inflammatory effect, while IPF is hypothesized to have an inflammatory origin. Because we are talking about a disease that is described as being idiopathic, this hypothesis is of course not confirmed.

Cough
While only twenty patients completed both the treatment and placebo phase of the study, the researchers did find a satisfactory result. Patients enrolled in the study reported that thalidomide treatment significantly decreased their level of coughing, which is a common symptom in IPF. While this does not tell us much about increased survival or other meaningful outcomes, the cough symptoms are annoying enough to warrant thalidomide treatment. Additionally, the results give rise to a larger study with more patients, in order to uncover its long term effects. Perhaps equally important is the result that showed patients had a higher self-reported quality of life during the periods that they were on the drug.

Outlook
It is interesting to see that a notorious drug such as thalidomide is gaining renewed clinical interest; it is not the first time the drug has been retested for specific clinical uses. It fits within the current clinical and regulatory context, by providing the right drug to the right patient, and dealing with the level of uncertainty and gaps in our knowledge. That means we are getting more careful with the applications of a drug. Obviously this is necessary with a drug such as thalidomide: prescribing it to pregnant patients is a bad idea, while the drug may, perhaps even due to its previously unrecognized side effects, be beneficial for another, well-defined group of patients. In addition, the effect of thalidomide on IPF patients may provide insights into the cause of the disease.