Five hospitals from different provinces began the phase II clinical trial of the world’s first treatment for Hemolytic Uremic Syndrome (HUS)a development of Argentine scientists against this disease that each year causes the hospitalization of between 400 and 500 boys and girls in the country, of which half may be left with sequelae for the rest of their lives.
The centers that started with the study are the Pedro de Elizalde General Children’s Hospital, the Zabala Clinic and the Güemes Sanatorium, in the city of Buenos Aires; the Children’s Sanatorium of Rosario (Santa Fe); and the Sanatorio Allende, in the city of Córdoba, and in the coming weeks institutions will continue to be added in the provinces of Buenos Aires, Córdoba, Mendoza, Neuquén and La Pampa until reaching a total of 16.
“In 2018 we successfully passed phase 1 that we carried out at the Italian Hospital. It was a study in adult volunteers; after this Anmat (National Administration of Medicines, Food and Medical Technology) authorized the clinical trial in girls and boys, who are the most affected by the disease, but we had to interrupt it due to the pandemic and now we are resuming it,” Linus Spatz, biologist and director of Inmunova, the biotechnological company that developed the drug, told Télam.
“In 2018 we successfully passed phase 1 that we carried out at the Italian Hospital. It was a study in adult volunteers; after this Anmat authorized the clinical trial in girls and boys, who are the most affected by the disease”Linus Spatz, biologist and director of Inmunova
For its part, Alejandro Balestracci, pediatrician at the Nephrology Unit of Hospital Elizaldewho has already treated the first patient without any adverse effects being recorded, stated that “we are very excited to participate in this study to evaluate the efficacy of a drug that makes it possible to make the clinical picture of HUS milder”.
And he recalled that “if the effectiveness is proven, it will be the first treatment in the world for this disease.”
As Spatz explained, the trial seeks to evaluate the efficacy and safety of the treatment developed by Inmunova in at least 100 girls and boys between 8 and 12 years old who are hospitalized with a recent diagnosis of HUS.
At this stage of the investigation, all trial participants will receive the drug, which is administered intravenously through a serum, along with the usual care within the standard of hospital care for this disease.
“The treatment is a serum that contains polyclonal antibodies against the Shiga toxin produced by the bacteria Escherichia coli that is the cause of the disease,” Spatz said.
“If the effectiveness is proven, it will be the first treatment in the world for this disease”Alejandro Balestracci, pediatrician at the Nephrology Unit of Hospital Elizalde
And he continued: “For the development of this serum we investigated the toxin for many years until we managed to produce, together with Conicet researchers, a molecule that can be injected into animals without causing damage, just like any vaccine. In response, the animals generated a huge amount of neutralizing antibodies.
These antibodies were fractionated using biotechnological processes until only the fragments that are directed against the toxin were obtained.which are then purified and formulated in the laboratory to obtain the medicine.
“If phase 2 is successfully passed, it would go on to phase 3, in which the aim is to test the efficacy of the treatment in more patients. As it is a worldwide problem and there are no specific treatments, in addition to working with Anmat in the regulatory framework of the study protocols, we also dialogue with the regulatory entities of the United States and Europe (FDA and EMA) so that, once the results are obtained, they can be presented to these organisms for their approval”, Spatz described.
How is it spread and what are the symptoms?
Escherichia coli bacteria can be present in meat but also in fruits, vegetables, unpasteurized milk and even in water.
According to the Ministry of Health, the symptoms of HUS are vomiting, irritability and, in some cases, seizures (as part of a neurological compromise of varying degrees); always requires hospitalization can lead to death.
This box is preceded by digestive symptoms that occur during the previous week: Diarrhea or bloody diarrhoea, so in the face of these symptoms you should always go to a medical center.
According to the World Health Organization (WHO), Argentina has the highest global incidence of HUS in children under 5 years of age: this disease affects between 400 and 500 boys and girls annually in the country; half of them can suffer more severe forms, with rapid deterioration of kidney function.
HUS is the first cause of acute renal failure in pediatrics, and the origin of approximately 20% of kidney transplants in children and adolescents.
These cases require long hospital stays and may require dialysis to help the damaged kidney filter waste and fluids.
It is estimated that each year approximately 3% of children who developed hemolytic uremic syndrome die in the country and many others are left with lifelong sequelae, such as kidney damage or neurological injuries.
Also HUS is the leading cause of acute renal failure in pediatricsand the origin of approximately 20% of kidney transplants in children and adolescents.
How to prevent it
In its prevention, it is essential to incorporate hygiene and care habits when handling food, such as wash hands with soap and water before and after using the bathroom or changing diapers, and after handling raw food, and when returning from the street.
Also cook meat thoroughly, especially ground meat and products made from it; consume drinking water, boiled or with at least two drops of bleach per liter, and pasteurized milk.
Add to that, wash fruits and vegetables thoroughly with drinking water, avoid contact of raw meat with other foods, and keep meats refrigerated and separate from vegetables or other foods.
Another measure is washing kitchen utensils and surfaces that come into contact with raw foodespecially if there will be contact with those who are ready to consume, as well as properly wash the sponges and rags used in the kitchen.
Finally It is recommended to use authorized swimming pools and avoid bathing in contaminated water.
