Indian scientists have achieved a significant breakthrough in the treatment of severe haemophilia A, a hereditary disorder characterized by a deficiency of Factor VIII, leading to frequent and severe bleeding. This novel gene therapy, tested on five patients in Tamil Nadu, aims to deliver a therapeutic gene to the body, potentially eliminating the need for frequent treatments.
This innovative therapy offers a potential one-time solution, teaching the body to produce enough Factor VIII to prevent haemorrhages. The patients involved in the trial reported no bleeding episodes during a follow-up period of 14 months, showcasing the therapy's effectiveness.
Traditional treatments for haemophilia require frequent injections of clotting factors. In contrast, this gene therapy uses an adenovirus as a vector to introduce the Factor VIII gene, making it a safer and potentially more effective alternative.
In India, the estimated cost of treating a haemophilia patient with this gene therapy is around ₹2.54 crore over a decade. While this cost is substantial, the long-term benefits may reduce the overall financial burden on patients and healthcare systems.
This Indian trial is in parallel with the U.S.-approved therapy, Roctavian, which has successfully reduced bleeding episodes from 5.4 to 2.6 times per year. The Indian version of the therapy could offer a more accessible and adaptable solution for local conditions.
The study demonstrates that gene therapy can be successfully implemented in resource-constrained settings like India. This advancement holds the potential to significantly improve the lives of thousands of haemophilia patients, estimated to be between 40,000 and 100,000 in the country.
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