No More Hemophilia Inhibitors?

For those who suffer from the chronic bleeding disorder hemophilia and antibodies caused by it’s medication (clotting factor), new research outlined in the Penn Current  may put a smile on your face today.  As we know, pharmaceuticals called factor replacement therapies, reduce and stop bleeding in those with hemophilia.  Almost 25% of those who inject regularly, however, develop antibodies called inhibitors that destroy the clotting factor. There could be a new treatment to tolerize inhibitors just around the corner says Henry Daniell, a professor at University of Pennsylvania School if Dental Medicine.

While researching with his Penn colleagues, Daniell relied on his plant-based drug-delivery platform, which uses genetically engineered plants to produce biotherapeutic proteins. They were able to fuse genes that helped promote immune responses in order to tolerize the clotting factor, Factor VIII (8). The researchers introduced these fused genes into tobacco plant chloroplasts, grew the plants, ground them up, and suspended them in a solution. They fed the solutions with the modified plant material to a group of mice with hemophilia A over two months, and fed another group of mice a solution with normal plant material. After giving the mice infusions of FVIII, the rodents fed normal plant solution formed high levels of inhibitors, while the group that was fed the modified plant material formed much lower levels of inhibitors. The researchers found they could both prevent and reverse  inhibitor formation.

Daniell believes this new finding offers a promising alternative. “The only current treatments for inhibitor formation cost $1 million and are risky for patients,” Daniell says. “Our technique, which uses plant-based capsules, has the potential to be a cost-effective and safe alternative.”

2014 has so far been a year of surprises and breakthroughs for those suffering from bleeding disorders, especially hemophilia.  As new research continues to improve, so do new and improved treatments – truly an exciting time in medicine.

Photo Credit – University of Pennsylvania School of Dental Medicine

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