Experts call them sMALL. The acronym stands for Inflammatory Bowel Disease. I’m there Crohn’s disease and ulcerative colitis. Unfortunately, these pictures are increasingly manifesting themselves in children and adolescents. They must be recognized as early as possible and treated accordingly. But above all it is necessary to find specific treatments for this age group.
In this sense goes a research of a group of Italian pediatricians of the scientific society SIGENP, who went to explore the effect of an old drug, which became sadly known in the middle of the last century but is now carefully studied for its positive effects, thalidomide. The results of a study, published by the most prestigious international journal of the sector, Inflammatory Bowel Disease, and coordinated by a group of Italian researchers led by Dr. Matteo Bramuzzo, of the IRCCS Burlo Garofolo of Trieste, collected the data of 10 years of clinical experience of the treatment of these frameworks in eight Italian centres.
What emerges from the research
When the affected children are very young, under the age of six, these pathologies are often defined with the acronym VEO-IBD (Very Early Onset Inflammatory Bowel Disease), i.e. very early onset inflammatory bowel disease. “In 37 children with VEO-IBD – Bramuzzo – thalidomide therapy proved to be effective and well tolerated: side effects only in 36% of cases, much lower than what was observed in adolescent patients (77%). It may seem like a small number of cases but, since we are dealing with rare diseases and patients with highly selected characteristics, the size of this population is significant. Even more interesting is the fact that the drug enabled sustained remission of the disease in a significant proportion of children.”
The duration of treatment ranged from a minimum of one year to a maximum of 9 years. Unexpectedly, therefore, thalidomide, considered an outdated and harmful molecule, brings new hope to medicine. According to Claudio Romano, president of SIGENP and Director of the Pediatric Gastroenterology and Cystic Fibrosis Unit of the University of Messina “this study has made it possible to add a new drug in the treatment of these complex pathologies in children. Furthermore, the relative low cost of this therapy compared to other drugs should be considered. But, beyond the positive results, we must not forget that the indication for the use of thalidomide must be discussed carefully with the doctors of the center where the child is followed up.”
“It is not yet the panacea for these diseases – continues Bramuzzo – but the results observed with our study are among the best ever obtained. In fact, until now this pathology in younger children was treated with the same drugs – biological and otherwise – that are used in adults, with often unsatisfactory results: now, thanks to this study, we have ascertained that thalidomide is certainly a valid therapeutic option in children with very early-onset inflammatory bowel disease unresponsive to conventional therapies.
The history of thalidomide in the 1950s
“A solution to combat insomnia, morning sickness, anxiety and other ailments in pregnancy”. This was more or less the message that, towards the end of the 1950s, accompanied what was to become both a great commercial success and one of the most dramatic passages in the history of the pharmaceutical industry. Indeed, they promised to eliminate the nuisance thalidomide tabletsa substance that has created thousands of malformed children in the world, with cases of amelia and phocomelia, or the lack of arms with hands that in practice start directly from the shoulder, and/or legs.
The story begins in 1957, when a drug containing it is put on the market, after an experiment on 300 patients which had not given rise to particular side effects. Before, the medicine was on free sale and it was only in 1961 that a medical prescription became necessary.
In that year, a pediatrician named Wildukind Lenz hypothesized for the first time a correlation between the increase in neonatal malformations and drug intake. Then shortly after, the relationship with the medicine is recognized. The drug, taken in four years by about five million women worldwide, was taken off the market in Germany in 1961 and in Italy the following year.
Now science is studying, and not only for Crohn’s disease in children, or even has already identified, possible therapeutic indications for thalidomide. Stories like the one we have summarized cannot be repeated because the situation is profoundly different.
Drug controls they are continuous, right from the very first stages of research, so much so that in practice nine out of ten drugs “die” before being born. Since the first studies on a specific molecule, for example, also thanks to computer systems it is possible to see if that chemical compound can cause damage to some cells, for example the liver or another organ. And then there is pharmacovigilance, which today is no longer just passive, i.e. a behavior waiting for a response, but also active: patients are followed up and monitored and, as soon as an undesirable effect is detected, the doctor can report it immediately.