Wednesday, September 19, 2012

'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.

2 comments:

  1. THALIDOMIDE, HAVING MAJOR PROBLEMS? C.O. P. D 2 YEAR LIFESPAN 6 YEARS AGO?

    Posted by booker4 25 minutes agoEdit | Delete
    HAVE BEEN TAKING THALIDOMIDE FOR OVER SIX YEARS,1 X 100 MIL TABLET EVERY NIGHT FOR 4 NIGHTS IN A ROW, TAKING PLENTY OF WATER, AFTER FOOD, TWO OR THREE HOURS BEFORE BEDTIME, EVERY SIX WEEKS I REPEAT THE SAME, MY LIFE CHANGED OVER NIGHT, FELT TEN TIMES BETTER, BREATHING EASIER, COUGHING STOPPED, I READ UP ON ALL ITS PAST AND KNOW MOST OF ITS SIDE EFFECTS, I DO NOT AND WILL NOT INDULGE IN SEX EVER WHILE TAKING THIS DRUG, I GET NO HELP FROM ANYONE REGARDING ITS COST, THE DOCTORS IN THIS COUNTRY[ENGLAND] WILL NOT PRESCRIBE IT, THE TABLETS COME IN STRIPS OF 30 AND COST ME £1,500, A TIME, MY SUPPLIER HAS OFFERED ME A DISCOUNT IF I PURCHASE MORE,WHICH I SUPPOSE IS NORMAL,
    SO, IF THERE'S ANYONE WHO FINDING IT HARD TO FINANCE THIS DRUG AS I DO, AND WOULD LIKE TO GET THE COSTS DOWN, THEN PLEASE CONTACT ME,
    EMAIL, SPACELEW@BTINTERNET.COM
    THANK-YOU,
    MICHAEL

    ReplyDelete