Back to Top

Hemophilia A Gene Therapy Success seen in Dogs

November 20, 2013; posted by: webleed staff

An experimental gene therapy trial (documented in Nature Communications) in the United Kingdom has seen success in dogs that have a disease similar to hemophilia A.  The three dogs that received the normal human factor VIII gene delivered to the bone marrow for expression via platelets resulted in the production of normal factor VIII by platelets when blood vessels experienced injury (and needed to clot).

More than two years after the gene therapy, two of the three animals show no signs of any serious internal bleeding events; the third had received a lower dose of gene therapy and continued to need synthetic factor VIII to counter its severe bleeds.

When asked about the implications of this research, the UK’s Haemophilia Society is optimistic that a long-term solution could be realized for human patients within 10 years for Hemophilia B and eventually for Hemophilia A.  In 2011, six patients with haemophilia B received gene therapy that allowed four of them to go without medication, raising hope for the future.

“We see gene therapy as a potential long-term solution for people with bleeding disorders; given the practicalities and the research already done, we would see this on a 10-year horizon as most likely being relevant to haemophilia B and then haemophilia A”, a spokesperson for the Haemophilia Society told BBC News.

The advocacy group warns that there is a long way to go from a small, but successful, trial in dogs to clinical introduction of a safe and effective therapy for humans.

“Nevertheless, we are mindful that no therapy can be used until it has passed every hurdle including clinical testing, and we are very mindful of the dangers of raising false hopes amongst those affected by the various bleeding disorder conditions.”

Comment Here

Your email address will not be published.

/>

Translate »