A new study by the University of Florida Health and the University of Pennsylvania has discovered that a new treatment to combat hemophilia lies in the cells of plants. Hemophilia is a genetic disease wherein the body is no longer able to coagulate blood due to a deficiency in clotting factor proteins. The severity of symptoms can vary, where the most general cases involve internal and external bleeding.
More serious cases of hemophilia can lead to deeper and more frequent internal bleeding, joint damage, and inter-cranial hemorrhage. The most common type of hemophilia is hemophilia A, also known as clotting factor VIII deficiency. Typical treatments involve blood transfusions and clotting factor infusions. While even the severest case of hemophilia is completely treatable, up to 30 percent of patients develop antibodies known as inhibitors that combat the infusions, rendering them ineffective.
Plant cell treatment
The researchers, led by Doctors Dheeraj Verma and Babak Moghimi, discovered that plant cells are able to direct the immune system to take in the infused clotting proteins instead of attacking them. They studied hemophilia A, wherein the clotting factor protein FVIII becomes mutated. FVIII has a heavy and light chain, with the lighter containing a C2 domain. They fused the heavy chain with a cholera toxin subunit protein and injected the material into the chloroplasts of a genetically modified tobacco plant, where both the DNA of the cholera toxin and the C2 domain were mixed together.
The plant leaves were ground and fed over a period of time to an experimental group of mice that had hemophilia A. A second group of mice was fed unaltered FVIII. After two months, the unaltered FVIII group produced high amounts of inhibitors in their bloodstream, while the altered FVIII group had considerably lower levels. The scientists discovered more cytokines in the experimental group, which suppressed the immune response and allowed the immune systems of the mice to tolerate the clotting protein. The experimental FVIII also greatly suppressed inhibitor formation, and when injected into the control group mice, reversed the formation and decreased activity by seven times as much.
Benefits of taking plant-based capsules
There are numerous benefits of this new and innovative hemophilia treatment. It is much less expensive, as many current treatments cost up to a million US dollars, and is also considerably less risky to take. The FVIII material is produced as plant capsules and is much easier and safer for a patient than blood transfusions. It does not come with any risk of adverse reactions or infection. The research into plant cells has also proven how effective this treatment is against inhibitors. It still does not serve as a permanent cure and patients would need to continue taking it to maintain low levels of inhibitory activity.
Work is now underway to produce this substance in lettuce plants for human consumption. With global funding and support from pharmaceutical companies, FVIII medication will soon be available to the public as an official hemophilia treatment.
Potential for more discoveries in hemophilia treatment
This study about hemophilia treatment has not only produced very promising results, it also gives hope that even more effective hemophilia treatments will be discovered in the future. While there is still much to learn about this plant-based protein technique, it has already opened the door to a whole new slew of possible non-invasive treatments. With further improvement and research, this treatment not only has the potential to benefit those with hemophilia, but also other patients suffering from adverse immune system responses.