2023-08-24 06:40:00
Every year some 400 children in Argentina are affected by the Hemolytic uremic syndrome (SUH), an endemic pathology for which we hold the global record of cases. Thus, HUS is today the first cause of pediatric acute renal failure and the second of chronic failure and 2.5 to 3% of patients with HUS. die for this cause. But if a team of Argentine scientists manages to confirm the results they have just obtained in Phase II trials, in a couple of years that situation could turn 180 degrees.
It is that if the researchers from the Immunova laboratory –formed by scientists from Conicet and private industry– finish developing the first medicine in the world that will be able to block the harmful action of Shiga toxin, responsible for HUS, it will be possible to treat these patients to save lives and significantly reduce the number of people on dialysis or who need to be transplanted, since it would be possible, for the first time, to treat the most serious forms of this Syndrome .
Argentine doctors will test a treatment against HUS
Just this week (08/19) was celebrated in our country the “national luch dayagainst Hemolytic Uremic Syndrome” which, in this 2023, came in the form of bad news: according to the data of the last National Epidemiological Bulletinso far this year, there was a increase of cases compared to the two previous years: so far 159 patients have been recorded, of which 126 were under 5 years of age. Another 122 cases had to be hospitalized, 57 were in intensive care and 3 deaths were noted. And the worst thing is that, until now, there are no treatments available, instead doctors can only stabilize the patient and give him “support” while the disease runs its course.
“Last July we completed Phase II testing of an innovative biologic drug that has the potential to become the first treatment capable of blocking the toxin Enter and avoid the severe forms of this pathology. Our drug contains different polyclonal antibody fragments and was developed locally by the Immunova team,” he explained to PROFILE the doctor Mariana Colonna, responsible for clinical research of this still young Argentine laboratory. And the expert added: “We tested it on 57 patients and the first big conclusion is that it showed be a tolerable and safe productwhich does not cause significant adverse effects.
In fact, they are still analyzing the results, “but we already know that they were encouraging and that is why we are going to ask the Anmat start Phase III in 2024 to medically verify the effectiveness of this medication to prevent deaths and reduce the severity of the disease, preventing many patients from ending up on dialysis and reducing the number of kidney transplant recipients motivated by its cause”.
Specifically, up to now the researchers have tested the serum in 16 hospitals, in a move that involved 300 professionals, who assisted around fifty HUS patients.
trials to come
“For the next stage of tests that we are already designing – in conjunction with Anmat technicians – we plan a mass rehearsal that will reach 200 patients, between one and 18 years of age and will be carried out simultaneously in about twenty medical centers throughout the country,” Colonna told this outlet.
HUS is a serious pathology and endemic which, only in Argentina, registers 400 cases per year, basically in boys. Of these, one in five will end up needing a kidney transplant, since it has become the first cause of pediatric acute renal failure and the second of chronic failure.
Recommendations to buy meat and prevent a disease that can be fatal
Finishing testing it will be, in addition to a medical challenge, also a highly complex test economicalsince completing Phase III requires an investment close to US$ 15 million. But when it is finally ready, it will be a medicine that will not only save Argentine lives, but that can also be exported since HUS does not have effective pharmacological options in any country on the planet.
In fact, the doctor Vanessa Zylbermanntechnical director of Inmunova told PROFILE that “until now there is no specific treatment in the world to prevent or cure this disease. And although Argentina has a record of cases, outbreaks are also usually registered in other countries. That is why we think that, if all goes well, in addition to taking advantage of it locally we could export“.
X-ray of the HES in Argentina
Every year there are about 400 cases a year, basically in boys. Of those, 1 in 5 in 5 will end up receiving a kidney transplant. HUS is the first leading cause of pediatric acute renal failure. It is the second second cause of chronic insufficiency. Its mortality amounts to between 2.5 and 3% of cases.
EG / ED
#Advances #treatment #HUS