As published in El Informador ,some researchers used gene therapy to successfully treat patients with severe hemophilia, a disorder in which blood does not clot.
The study was preliminary and involved only six patients, while other promising previous attempts to use gene therapy against hemophilia finally failed. However,a single infusion with the new treatment worked in some patients for more than one year, increasing its ability to clot significantly.
“I think this is a great advance in the field. It’s a good lesson in terms of not giving up when there are good ideas,” said Dr. Ronald Crystal, chairman of genetic medicine at Weill Cornell Medical College in the city of new York.
Hemophilia is an inherited disorder and threatening that primarily affects males.
In severe cases, people with the disease may spontaneously suffer internal bleeding, even in the brain. Bleeding into the joints generates debilitating movement problems and severe pain.
Experts said the new method needs to be tested in more patients to confirm its effectiveness and ensure that it is safe. Even if all goes well, there are still several years for most patients can access it.
Since late 1960, doctors have prescribed for hemophiliacs infused clotting proteins. This has been a success as it has increased its average life to 63 years.
However, for severe cases, treatment may involve two or three weekly infusions at a cost of over $ 250,000 a year.
In my opinion this development will help many people, because not everyone has such high figures as to acquire the current cure, which will be more accessible once the investigation finished.