Cured dogs offer hope to haemophiliacs
Published: August 9, 2005
There is new hope for haemophiliacs after American researchers managed to cure the condition in dogs.
The same approach has been trialed on a small number of humans, including two Australians.
“When we started to use the higher doses, one of the subjects in particular had a good elevation of his protein level to the point that he didn’t require treatment with outside protein,” Dr Katherine Manno, from the children’s hospital in Philadelphia, said.
Doctors take a gene that has been modified to encourage blood clotting.
They inject it directly into the liver of the patient with haemophilia.
The early studies showed the treatment worked well, but was only temporary - the liver rejected the drug after several weeks.
But researchers now think that giving patients a short course of drugs to suppress the immune system will allow the cells to take hold and start producing protein for the blood to clot.
“If the medicine does get into the liver and teach it how to make factor for a long period of time it could have a tremendous benefit for people with haemophilia,” Dr Manno said.
Long-term benefits
Australian haemophilia experts say the research could provide a long-term cure for the disease, though they say that still remains some years away.
“It needs to be safe for patients and it needs to be cost effective,” Sharon Caris, from the Australian Haemophilia Foundation, said.
Doctors say the dogs have remained disease-free five years after being treated.
The research was presented at the 20th Congress of the International Society on Thrombosis and Haemostasis at Darling Harbour in Sydney.
If you enjoyed this good news Subscribe to Good News Blog
Share this
To share this simply copy and paste one of the below URL's: