In a groundbreaking achievement, India’s first-in-human gene therapy for severe hemophilia A has shown remarkable outcomes, marking a significant leap in medical innovation. This pioneering therapy, developed using lentiviral vectors by the Centre for Stem Cell Research (CSCR) at Christian Medical College (CMC), Vellore, has successfully delivered sustained benefits to patients, eliminating the need for frequent clotting factor infusions.
Highlights of the Study:
- Supported by the Department of Biotechnology, the single-centre study enrolled five participants, all of whom achieved zero annualized bleeding rates over a cumulative follow-up period of 81 months.
- The gene therapy utilizes a lentiviral vector to insert a normal copy of the Factor VIII gene into the patient’s own hematopoietic stem cells (HSCs). These modified HSCs consistently produce functional Factor VIII, addressing the underlying deficiency that causes hemophilia A.
- The therapy eliminates the need for regular Factor VIII replacement, overcoming challenges such as high treatment costs, venous access in children, and low compliance.
Significance of the Results:
- Hemophilia A, caused by a deficiency of clotting Factor VIII, is a life-threatening disorder that affects quality of life due to spontaneous and recurrent bleeding episodes. India has the second-highest global burden of hemophilia, with around 136,000 cases.
- Current treatments require frequent and costly infusions, but this gene therapy offers a long-term solution with zero bleeding events observed in all participants.
- Published in the New England Journal of Medicine, the findings highlight the therapy’s safety and efficacy, showcasing a strong correlation between Factor VIII levels and vector copy numbers in peripheral blood.
A Hopeful Future: Dr. Alok Srivastava, lead researcher at CSCR, emphasized that this development marks a transformative step toward making advanced treatments accessible in resource-limited settings. With these promising results, the therapy unlocks new possibilities for managing severe genetic disorders, offering hope to thousands of patients.
This achievement positions India as a global leader in innovative gene therapy approaches, providing a beacon of hope for previously incurable diseases.
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