A new breakthrough in gene therapy has reduced the risk of bleeding in people with the rare disease hemophilia Bhave said experts.
The researchers found that a single injection of the gene therapy called FLT180a eliminated the need for people to self-inject on a weekly basis coagulation factors.
The study was led by experts from University College London (UCL), the Royal Free Hospital in London and biotechnology company Freeline Therapeutics.
Usually when a person cuts himself, coagulation factors Mix with blood cells called platelets to stop bleeding.
But people with hemophilia Clotting factors are absent and there is a risk of severe bleeding.
Most people (about 85%) have Hemophilia Acaused by a deficiency in blood clotting factor VIII, while hemophilia B is caused by a deficiency in factor IX.
people with Hemophilia B need regular injections – usually weekly – to make up for their clotting factor IX deficiency, but may still experience debilitating joint damage.
What did the process reveal?
In the new 26-week study, published in the New England Journal of Medicine, experts found that a single treatment with FLT180a resulted in sustained production of the protein from the liver in nine out of 10 patients with severe or moderate disease hemophilia.
This eliminated her regular injections by correcting her genetic flaw and has long lasting effects.
Lead author Professor Pratima Chowdary from UCL said: “Eliminating the need for hemophilia patients Injecting the missing protein regularly is an important step towards improving the quality of life.
“The long-term follow-up study will monitor patients for duration of expression and monitoring for late effects.”
As part of the study, the patients had to take immunosuppressive drugs for between several weeks and several months so that their bodies did not reject the gene therapy.
Although the treatment was generally well tolerated, all patients experienced side effects with abnormal side effects blood clot in one that received the highest FLT180a dose and had the highest protein levels.
Freeline co-founder Professor Amit Nathwani from UCL, who co-authored the study, said: “Gene therapy is still a young field that is pushing the frontiers of science for people with serious genetic diseases.”
He said the new study adds to “the growing evidence that gene therapy has the potential to free patients from the challenges of having to adhere to lifelong therapy, or could provide treatment where none exists today.” ”
What are hemophilia symptoms?
Corresponding the NHS website Hemophilia symptoms can include:
- Nosebleeds that take a long time to stop
- Bleeding from wounds that last a long time
- bleeding gums
- Skin that bruises easily
- pain and stiffness around joints, such as elbows, due to bleeding in the body (internal bleeding)
https://www.theargus.co.uk/news/national/uk-today/20293953.gene-therapy-found-cut-risk-bleeding-haemophilia-b-patients/?ref=rss Gene therapy has been found to reduce the risk of bleeding in hemophilia B patients