New approach to cure for haemophilia

A group of medical researchers from the Medical College of Wisconsin and BloodCenter Wisconsin in the United States have published an article on a new approach to cure haemophilia with genetic engineering.

The novelty in their approach is to "hide" the FVIII proteins inside blood platelets. By doing so, FVIII is shielded from attack by FVIII-antibodies (inhibitors), which occur in up to 30% of patients and which render normal factor concentrate treatment ineffective.

In experiments they have succesfully introduced a gene into blood stem cells that cause the platelets to produce and store FVIII proteins.

As usual, the results are from trials with mice and a treatment for humans is still far away. More controlled experiments will have to be conducted with larger animals and ultimately with humans, before the technique can be applied to a larger population.

Read the full story

Create reply »

physics

06 Jul 2006 17:18

Profile

Guestbook

Somebody correct me if I`m wrong, but by this method, it looks like FVIII transfusion will be no longer necessary for patients who will take part in this gene therapy. I will highlight these 2 key points:
1. FVIII will be PRODUCED and STORED in blood platelets. (does that mean the platelets will have it`s own ability to keep producing FVIII?)
2. "This process would last for the rest of the patient`s life and will work regardless of whether antibodies are present or absent," says Dr. Montgomery
What do you guys think?

Create reply »

cicero

06 Jul 2006 20:38

Profile

Guestbook

I think you are correct. The gene therapy aims to modify the platelets in the body so they continue to produce FVIII. However, at the world congress, reports from similar experiments showed that sometimes the effect wears off after a while, thus requiring additional treatments. The issue seems to be whether you can replace the existing genes so that new platelets will inherit the modified genes instead of the defective ones. In some experiments they succesfully introduce the new gene in the existing platelets, but fail to carry the new gene over into new platelets. Hence the effect would wear off in the same pace as modified platelets are destroyed. Even so, the effect will be much longer than current replacement therapy, since the half-life of platelets will be much longer than for FVIII proteins (as far as I know - I am no expert on this subject :-) ).

There is a lot of promising research on gene therapy being published, but even so we are still a long way away from a cure. Another interesting development is focused on increasing the half-life of FVIII in the blood. This research is much more advanced and could be available for mass treatment in a not so distant future.

Create reply »