The return of Thalidomide
The prohibited drug is returned through the vesselsAlexandra Borisova, "Newspaper.
Ru»Thalidomide, a drug that provoked the birth of tens of thousands of disabled children in the 1960s, has again become a news hero.
In addition to the treatment of leprosy, it can also be used for the successful therapy of rare severe diseases of the circulatory system.
From poison to medicine, as you know, one step. Firstly, it all depends on the concentration and amount of the agent used. Secondly, the initially useful remedy may subsequently have severe side effects that practically exclude its use for its "direct" purpose. So began the use of morphine, so began the history of thalidomide, which caused the birth of tens of thousands of defective children in the middle of the last century. The history of this drug in an artistic form was described in detail by Arthur Haley in his novel "The Potent Medicine".
Thalidomide, once completely banned, has recently begun to return to the market, proving to be an effective remedy for the treatment of very serious diseases (in these cases, unfortunately, the issue of childbirth for patients is no longer worth it). Currently, thalidomide is used to treat leprosy, multiple myeloma and other serious oncological diseases. Of course, the drug is not available for free sale, its use is regulated by the Pharmion Risk Management Program (PRMP)
But the potential of thalidomide has not yet been exhausted. According to a study published in Nature Medicine (Franck Lebrin et al., Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary hemorrhagic telangiectasia), it is also able to treat rare disorders of the circulatory system, such as Randu-Weber-Osler disease (or congenital hemorrhagic telangiectasia, hemorrhagic telangiectasia, HHT).
This disease occurs, according to statistics, in one in 10 thousand people and is accompanied by frequent severe bleeding, both nasal and internal. Sources of bleeding are telangiectasia or, simply put, vascular "asterisks", persistent uneven expansion of small vessels of the skin or mucous membranes in the form of red spots. At the moment there is no generally accepted strategy for the treatment of this disease. Most often, treatment is carried out surgically, but the prognosis of the disease is often unfavorable, and recovery is rather a rare exception.
French doctors were looking for a cure for NNT, testing various drugs on mice genetically predisposed to this disease (most often NNT is hereditary). They found that the use of thalidomide reduces the risk of severe bleeding and stabilizes blood vessels.
The mechanism of action of the drug is activation of the PDGF-B protein (platelet growth factor). It stimulates the growth of vascular wall cells and strengthens them, preventing bleeding.
"Treatment with thalidomide makes it possible to eliminate defects in the walls of blood vessels, which cause bleeding. A biopsy of the nasal mucosa tissue of experimental mice with NNT allowed us to assume that the same effect will be observed in people suffering from this disease," explained Frank Lebrin, the first author of the work, an employee of the College de France, Paris.
The story of thalidomide – an insufficiently tested drug, the use of which as a sleeping pill and antiemetic by pregnant women provoked the birth of tens of thousands of sick children – at one time stirred up the whole world. For chemists and pharmacists, this example has become a textbook to illustrate the fundamental difference in the effect of optical isomers of the same compound on the body.
The task of obtaining optically pure products has occupied its niche among the priorities of organic synthesis. However, in the case of thalidomide itself (in which, as they say, one isomer heals, and the other cripples), the optical purity of the drug does not give the necessary result, since in the human body isomers are able to pass into each other. Well, doctors were left with a testament about the need to very accurately check the drugs prescribed to pregnant women and be guided, first of all, not by the comfort of the mother during pregnancies, but by considerations of the health of the unborn child.
In the period from 1957 to 1961, 40,000 children with peripheral neuritis and from 8,000 to 12,000 newborns with physical deformities were born, of which only about 5,000 did not die at an early age, remaining disabled for life.
Some of them are now famous people: French DJ Pascal Kleiman, who was born without arms, German bass-baritone Thomas Kvasthoff, who was born with severe pathologies of the arms and legs, and British artist Alison Lapper.
(A heartwarming story about the wedding of two thalidomide survivors was published two years ago by MailOnline: A truly special love story: Two married thalidomide survivors living happily 50 years after drug's launch. For the faint of heart, we recommend disabling the display of drawings before reading – VM.)
Portal "Eternal youth" http://vechnayamolodost.ru05.04.2010