FINALLY! A Cure For Hemophilia
Scientists have created a new gene therapy for hemophilia, a deadly disease in which blood does not clot. This new therapy may allow most patients to live normal lives. They won’t need frequent infusions needed and they’ll say goodbye to the damage done to their joints caused by internal bleeding.
BioMarin, a California-based biotechnology company, presented some serious results at the World Federation of Hemophilia. BioMarin has annual sales of $890 million and has made the hemophilia treatment one of its most important experimental priorities.
Patients with hemophilia A, the most common form of the disease that is diagnosed in 800 American newborns each year, lack sufficient amounts of factor VIII, which is a clotting factor in the blood. According to Forbes, researchers at BioMarin had “hoped the gene therapy could raise plasma factor VIII levels to 5% on the scale usually used for measuring clotting factor levels. Instead, in 6 of 9 patients treated, levels are at 50%, which is virtually normal. The results look even better when two patients who got a lower dose and did not hit the 50% level are excluded.”
“I’m amazed, honestly,” says John Pasi, the professor at the London School of Medicine who ran BioMarin’s study. “It has hit the jackpot.”
“It’s miles better than I had ever expected or even dreamt of, Nathwani says. “The idea that you can get factor VIII expression in the normal range is outstanding. It’s really very impressive, and that with minimal toxicity.” – says Amit Nathwani, who led a team at University College London that originally developed the treatment.
The treatment that BioMarin developed is actually a virus known as an adeno-associated virus that has had its own DNA removed. It is a little edited-down version of the gene for factor VIII, and it is put into the virus’ shell, which is injected into the liver. This virus creates little pockets in the liver cells and takes over their molecular machinery to make factor VIII.