Chinese scientists declare breakthrough that allows haemophilia patients ‘to live like normal people’
- Researchers designed a gene therapy drug to treat the disorder and found it to be safe and effective in preventing bleeding events a year after infusion
- Study achieved the best treatment results internationally and ‘takes curing incurable haemophilia B from a hope to a reality’, according to team
Chinese researchers have made what they claim is “a historic breakthrough” in the treatment of haemophilia, an inherited disorder that can cause unstoppable bleeding.
They designed gene therapy drug BBM-H901 to treat haemophilia B and have found it to be safe and effective in preventing bleeding events a year after infusion.
After 58 weeks of treatment, the average level of clotting factor activity of 10 patients included in the study increased from less than 2 per cent to 36.9 per cent, according to the study published last week in The Lancet Haematology.
In haemophilia, the level of clotting factor activity is closely linked to the severity of the disease. Normal clotting factor activity ranges from 50 to 120 per cent and a severe haemophilia B patient may have an activity of zero or less than 1 per cent. Individuals with factor levels between 20 and 40 per cent rarely have spontaneous bleeding, according to the researchers.
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“In our long-term follow-up, only one patient had received replacement therapy due to aggravation of a previous haematoma. No other patients had excess bleeding, which greatly reduced their pain,” the researchers said in a statement.