Drug company Baxter International, based in Deerfield, Illinois, sells recombinant clotting factors for
treating haemophilia and also has its sights on TFPI.
The results from the first human trials for a gene therapy to
treat haemophilia A have just been published.
Not exact matches
But many of the physicians who
treat patients with
haemophilia are not convinced.
Clotting - factor infusions
treat symptoms of
haemophilia, but gene therapy could provide a cure.
When
treated with replacement coagulation proteins, the dog naturally develops antibodies, or inhibitors, against the therapy — a problem that is also seen in some 5 % of humans with
haemophilia B.
After decades of frustrations, firms believe there are now major opportunities for gene therapy in
treating inherited conditions such as
haemophilia.
In Madrid last week, Amit Nathwani of the Royal Free NHS Trust in London announced that six people
treated for
haemophilia using AAV in early 2011 are still producing the blood clotting factor they previously lacked.
About 30 % of people with
haemophilia A develop inhibitors, and once they do,
treating their bleeding becomes much more difficult.
The hunt is on for ways to diagnose and
treat the joint problems that are now the main chronic problem in
haemophilia
Replacing the clotting ability lacking in
haemophilia has been the treatment since the 1840s, when attempts were made to
treat people with the disease by transfusion with whole blood from people with normal clotting.
Professor Pasi continued: «Incredibly exciting is the potential for a significant change in how
haemophilia is
treated globally.
The results, published in the New England Journal of Medicine, demonstrate the therapy being used to
treat 10 male
haemophilia patients without significant side effects.