We will have to get used to this type of initiative. In the past, the development of a drug was only possible thanks to the research of pharmaceutical companies which, in the capitalist system, looked only where the profit promised to be important; therefore against diseases such as hypertension, rheumatism or migraine; especially not against so-called orphan diseases because they are rare. The fashion for start-ups and fundraising on the internet has changed everything
In early 2017, François Besnier, Maïthé Tauber and Jean-Hubert Gallouët laid the foundations of OT4B, a start-up of a new kind. Based on the very promising results of clinical studies carried out by Professor Maïthé Tauber, in patients suffering from Prader-Wili syndrome and from patents held by 4 co-inventors, OT4B’s mission is to provide families and children with of this syndrome, a new therapeutic solution by 2020.
40 new patients per year
Prader Willi’s disease only affects a few thousand children who, due to poor functioning of a small gland located in the middle of the brain, the hypothalamus, suffer from generalized muscle weakness and poor management of their diet. For example, he does not seem to feel full when their needs are lower than those of other children. They become obese and put their lives in danger, especially since their muscle problems do not encourage them to be physically active.
The culprit is a chromosome that got damaged on its own. This means that this disease is not transmitted from parents. This makes its diagnosis even more delicate because nothing allows the family to suspect its occurrence.
A very gripping disease for the family
The doctor himself does not think about it in these children who are a little soft, eat little and badly but who, paradoxically, have a clear tendency to grow abnormally.
The only solution, while waiting for a treatment, is a constant presence to get these children moving while limiting their calorie intake. A constant, thankless and exhausting job for the families and the medical environment of these children.
This is why the prognosis of this disease is not homogeneous. It depends too much on the time and means of the parents. Some children particularly surrounded by people manage to follow an almost normal education, which proves that it is possible to limit the consequences, even if this is not the rule.
Oxytocin, attachment hormone
Unfortunately, there is no specific treatment for this disease. At least while waiting for a therapy which the company OT4B hopes will come from oxytocin, a natural hormone oxytocin which is an important hormone in the mechanism of the attachment of the mother to her child.
Take a virgin sheep. Put her in a herd. She is only interested in her. You inject her with a syringe of oxytocin, so she keeps rounding the lambs together to protect them. This miracle is caused by a hormone, oxytocin, which brain specialists also call the attachment hormone. It is especially secreted at the time of childbirth, in particular by the fetus, which, by this mechanism, warns that it wishes to go out: it is the child who triggers the childbirth and not a sort of calendar or a standard duration. Secretion continues throughout breastfeeding. We could deduce from this that the woman is attached “chemically” to her baby, unlike the father who does not have the same secretion capacities, thus explaining the differences between the “biological” motivation of the two parents. The excuse is imperfect because the man has his own production of oxytocin, certainly less important than the woman, but sufficient to understand the phenomenon of the exceptional duration of certain couples. Do not look for the secret of the oak wedding anniversary (80 years of marriage) elsewhere than in the regular production of this hormone, which is not the result of any sexual stimulation but of the tenderness of the caresses.
It is therefore an astonishing use in Prader Willi’s disease but not inexplicable:
“The first results of clinical studies have shown that 88% of newborns treated with oxytocin fully recover their sucking and swallowing abilities. Their behavior is also changing towards their mother, they are closer to her and their interactions are better ”, declare the start-up.
Rare diseases not so rare
We are always surprised that rare diseases actually affect a lot of people. Four million patients in France and undoubtedly many more French people because these diseases involve a lot the entourage.
Without really unity between those who suffer because the rarity concerns more than 7000 diseases which do not have much to do between them, we discover moreover 4 to 5 per week
.
