Biological and Health Sciences

Scientists advance in the approval of a serum to prevent hemolytic-uremic syndrome

CONICET researcher Fernando Goldbaum is one of the leaders of this medication that will be on clinical stage in patients.

Fernando Goldbaum, CONICET senior researcher and scientific director of Inmunova during the conferece held at the Hospital Italiano in which the advances were presented. Photo: CONICET Photography.

The hemolytic-uremic syndrome (SUH) is a serious disease that occurs when a person ingests food contaminated with the Escherichia coli bacterium, which can be present in meat, fruit, vegetables, unpasteurized milk or even in water. This bacterium produces the Shiga toxin (STEC), which generates SUH.

Its major incidence is in the pediatric population. According to the World Health Organization, Argentina has the highest rate in the world in children under five years old (8,5 cases per each 100.000 children). In the country, there are 5 thousand STEC infections in one-year-old children and around 500 develop the syndrome. SUH is the first cause of acute kidney failure in children and accounts for pediatric kidney transplants. It can leave lifelong effects like hypertension and neurological alterations, and cause death between 3 and 5 per cent of the cases.

From the urgent need to have specific therapies for this disease, CONICET researchers, the laboratorio Inmunova and the Hospital Italiano developed a biological drug that could become the first one capable of avoiding the progression of hemolytic-uremic syndrome. This medication is an antibody similar to the one used against venom of snakes or scorpions, which means that it acts by neutralizing the toxin in circulation, responsible for triggering the syndrome.

Recently, the Administración Nacional de Medicamentos, Alimentos y Tecnología Médica (ANMAT), approved the data on the first study in healthy adult volunteers. In this first stage, the participants were 14 people of the Healthy Volunteers Record of the Hospital Italiano. They received the intravenous serum.

The test conducted by the area of Clinical Pharmacology of the Hospital Italiano showed an excellent safety profile and appropriate pharmacokinetics (distribution of the drug in the body) for its later application.

One of the leaders of the project is Fernando Goldbaum, CONICET senior researcher at the Institute of biochemical Research of Buenos Aires (IIBBA, CONICET-Fundación Instituto Leloir) and scientific director of Inmunova, where he is its cofounder. Yesterday, at a conference organized at the office of the Hospital Italiano of Buenos Aires, the developments were presented and he said: “It’s a very strong antiserum that can neutralize toxin in pre-clinical models. We conducted the study at the Centro de Medicina Comparada (CMC) of the Universidad Nacional del Litoral (UNL) and CONICET, in a public-private linkage with INMUNOVA.”

During this year, we are going to start the clinical study Phase 2/3, which will be carried out with pediatricians and pediatric nephrologists of the Hospital Italiano and from other centers in all the country. The children infected with bacteria E. Coli -which produces Shiga toxin- and in risk to develop SUH will be invited to participate in the clinical study as volunteers under the regulations of ANMAT. The aim is to prove the efficiency and safety of the product compared to the administration of placebo added to the standard treatment.

“We’re very glad to be the first to conduct basic research to take it to product that can cure a disease and very proud to be creating the first biotechnological product totally developed in Argentina and with global reach because we think it will be the first medicine capable of stopping SUTH”, the researcher concluded.