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"There have been new studies where new molecules can be used, we have molecules like monoclonal antibodies (type of protein produced in the laboratory that can bind to a substance in the body) and we also have a Gene therapy They are making very interesting progress for hemophilia A and B, "said Miguel Antonio Escobar, medical director of the University of Texas Research Center, in an interview with Efe.
Escobar participated this week in the seventh edition of Roche Press Day, an informative and educational meeting that promotes the continuing education of journalists and where the latest developments in the health sector in Latin America are known.
There are about 500,000 people in the world who suffer from hemophilia, a disease that consists of the absence of a protein in the blood that allows clotting.
For hemophilia A, which accounts for 85% of cases, there is a lack of what is called factor VIII and for B (who suffers the remaining 15%), there are low or nonexistent percentages of factor IX.
To make blood clotting possible, a total of 13 factors must be involved together.
"You have to think about coagulation as if it was a symphony, if you miss a part, it does not work well," says Escobar, who works as a hematologist at the Center. Hemophilia and Thrombophilia of the Gulf States of the University of Texas. United)
"One of the molecules (Emicizumab) has recently been approved, clinical studies have shown that it is very safe and medically effective, it prevents bleeding, which is the goal we have in hemophiliac patients ".
Daniel Ciriano, Roche's medical director, the pharmaceutical company that developed the molecule, told Efe that it would be administered to patients who develop coagulation factor VIII inhibitors.
"Today we replace factor VIII with a factor VIII that the body does not produce.
Continuous administration means that 30% of patients develop antibodies that block it, inactivate it ( to factor VIII), "he added.
The new treatment realizes that patients with developed inhibitors work and that the annual rate of bleeding is controlled.
"In patients treated with the molecule, the annual bleeding rate is reduced to almost zero," explained Ciriano.
Patients' quality of life and adherence to treatment also improve since people, including children, should not inject two or three times a week to provide the postmenopausal factor. that they need.
"When the injections are done once a week, and studies show that today they can be done every two to four weeks, these are benefits that are appreciated by patients," he said. he says.
Escobar also pointed out that certain "gene therapies" are already in clinical trials: they consist in "choosing a vector, the associated adenoid 8, to which genetic modifications are made".
"Then it is injected into the individual suffering from hemophilia and the liver is able to produce factor VIII or IX," explained the specialist.
Hemophilia is a genetic disorder that affects the male population (99% of cases) and is transmitted from mother to child.
In Latin America, there are an estimated 58,000 cases, of which 52% have not yet been diagnosed.
Lowering this number is the goal of the World Federation of Hemophilia (WFH).
To combat the under-diagnosis, the federation works with countries in the region where they found disparities in terms of data.
"Guatemala, Bolivia and Paraguay have the largest number of subregisters and we have good results in Brazil, Argentina, Venezuela, Costa Rica, Chile and Panama," said César Garrido, member of the board of directors of the WFH. Profit established in 1963, formed by a worldwide network of patient organizations in 140 countries.
Until 1937, the life expectancy of a person with hemophilia was 13 years. Today, the average fluctuates between 65 and 70 years old.
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