Ege University administers Turkey's first gene therapy for hemophilia A patients

İzmir's Ege University administered the first gene therapy for hemophilia A patients in the country, the school said on Sep. 2, possibly saving eight patients from life-long treatments with a one-time cure.

Thursday September 02 2021 11:12 am

Hemophilia is usually an inherited bleeding disorder in which the blood does not clot properly.

Duvar English

İzmir's Ege University administered gene therapy on patients with hemophilia A for the first time in Turkey, the school reported on Sep. 2.

Aged 18 to 25, the eight patients were treated as part of phase three of an international clinical trial run by Hemophilia Federation Chair Dr. Kaan Kavaklı.

Kavaklı had also performed gene therapy on four patients of type hemophilia B in 2020.

"The gene therapy administered on four patients last year was conducted on eight patients this year, and they have regained their health," University Rector Dr. Necdet Budak said.

Gene therapy saves patients from having to take medication for their whole lives, Budak said, noting that the practice aims to completely cure the disease.

Ege University has a 30-year-old center focusing on blood clotting, Kavaklı noted, adding that they've been working on hemophilia treatments for the past two decades.

Six to seven thousand people in Turkey suffer from hemophilia, Kavaklı said.

Hemophilia is usually an inherited bleeding disorder in which the blood does not clot properly. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery.

There are two major types of hemophilia, type A and type B.

In hemophilia A, there is a lack of clotting factor VIII. This accounts for about 80 percent of hemophilia cases.

In hemophilia B, the person lacks clotting factor IX.

Both A and B can be mild, moderate, or severe, depending on the amount of clotting factor that is in the blood.