Haemophilia Patients Stop Bleeding With Gene Therapy
We’ve come across some ground breaking research straight out of Philadelphia. One injection of experimental gene therapy stopped spontaneous bleeding in nine haemophilia patients.
Researchers are cautiously optimistic that this treatment could last for years. They believe this despite the small group of participants and the short follow up time. This would mean that patients with haemophilia B would be:
- Free from painful internal bleeds in their joints
- And would no longer need to pay for costly clotting medication
So How Does This Haemophilia Treatment Work?
Researchers explain that this treatment consists of one injection. It holds non-replicating viruses that are designed to carry the genetic recipe for clotting factor nine. This factor is absent in people with haemophilia B. In addition to preventing bleeds, researchers say that the treatment did not cause problems for the patients’ immune system. A problem frequently occurring in earlier research. Instead this was safely managed with steroids.
This treatment has less viruses then the last treatment. But, it has a more potent for of the gene recipe.
Lindsey A. George is a hematologist at CHOP and the lead author of the study. She says:
“The idea here is you can have less protein, but have more activity.”
All nine patients were men with levels of clotting factor so low, they were at risk of spontaneous bleeds. After the treatment, all had levels high enough to avoid that complication and to allow normal physical activity.
Haemophilia B In A Nutshell
Hemophilia B, or Christmas disease, is an inherited rare disorder that results in deficiency of blood clotting factor nine. Spontaneous gene mutation and acquired immune difficulty can result in this disorder as well.
The hallmark of hemophilia is hemorrhage into the joints. This bleeding is painful and leads to long-term inflammation and deterioration of the joint. This results in:
- permanent deformities
- loss of mobility
- limbs of unequal length
Researchers presented the results at the annual meeting of the American Society of Hematology in San Diego.
If you enjoyed this article, read What Your Blood Type Actually Says About Your Health