Medical breakthrough: Researchers permanently cure hemophilia B patients

0

British doctors make a breakthrough
“It’s really all a miracle”: Researchers permanently cure patients with a variant of the hemophilia

The team that conducted the hemophilia study now hopes that the majority of adults with hemophilia can be cured in the next three years (icon image)

© Motortion / Getty Images

07/21/2022, 14:27

2 min reading time

A new form of therapy in Britain has cured people suffering from the bleeding disease hemophilia B.

Hemophilia – also known as bleeding disorder – is an inherited disease. It mainly affects men. Due to a change on the X chromosome, too little coagulation factor is formed, which means that bleeding does not stop in the usual time. As a result, injuries such as bruises, i.e. hematomas, are often disproportionately large and general wound healing can be severely delayed, as summarized by the German Hemophilia Society (DHG).

Usually only one of about 13 different coagulation factors is affected in the narrower sense of the two known forms of hemophilia. For example, in hemophilia A it is coagulation factor VIII, in variant B it is coagulation factor IX.

A novel treatment now corrects the genetic defect in variant B that causes people’s blood to have trouble clotting and stopping bleeding. According to the BBC, patients who took part in the study in which the therapy was tested report that their lives now feel “completely normal”.

Anopheles mosquito bites humans

News from medicine

Neither vaccination nor infection provide permanent immunity to malaria. Researchers have now found why

People with hemophilia are missing a protein

Those affected by the hemophilia are usually unable to produce enough of the so-called coagulation factor IX. For example, if you cut yourself and the wound becomes crusted, clotting factor IX is one of the proteins that stops the bleeding. Patients are now being vaccinated with factor IX during the new therapy to prevent bleeding, which can be fatal in the worst case.

Through the injections, an artificial virus gets into the body, which is filled with the components of the missing factor IX. The virus then behaves like a microscopic transporter, delivering information about the added components to the liver, which then starts producing the clotting protein on its own.



Eckart von Hirschhausen presents his new issue "Live healthy" before

The results of the study, published in the New England Journal of Medicine, showed that nine out of ten patients given the therapy did not need additional injections afterwards. “I haven’t had any treatment since I had my therapy, it’s really all a miracle. Well, it’s science, but it feels pretty wonderful to me,” says Elliott Mason, one of the study participants in an interview with the ” BBC”.

cancer therapies

The main weapons of medicine against cancer


Surgery For most localized tumors, surgery is the most important treatment—and often the first step. The aim is to remove the cancerous tissue as completely as possible. Surgeons also remove adjacent, healthy tissue into which tumor cells may have already penetrated, as well as associated lymph nodes, because metastases often form here first. The doctors proceed as gently as possible and try to preserve the affected organ and its function as much as possible. Open surgery is not always necessary; sometimes a minimally invasive procedure, known as keyhole surgery, is sufficient today, for example for tumors in the stomach and intestines or cervical cancer. The doctor then makes just a small incision through which he can insert surgical equipment and a small camera into the body.

The operation
For most localized tumors, surgery is the most important treatment—and often the first step. The aim is to remove the cancerous tissue as completely as possible. Surgeons also remove adjacent, healthy tissue into which tumor cells may have already penetrated, as well as associated lymph nodes, because metastases often form here first. The doctors proceed as gently as possible and try to preserve the affected organ and its function as much as possible. Open surgery is not always necessary; sometimes a minimally invasive procedure, known as keyhole surgery, is sufficient today, for example for tumors in the stomach and intestines or cervical cancer. The doctor then makes just a small incision through which he can insert surgical equipment and a small camera into the body.

More


The research team hopes that the majority of adults with hemophilia could be cured in the next three years with the research results that have now been obtained, because there are “many young patients with excruciating torment” worldwide, says Prof. Pratima Chowdary of the Royal Free Hospital and University College London, who needed urgent help.

Sources: “New England Journal of Medicine”, BBC, DHG

km

#Subjects

source site