In a significant medical feat, Indian scientists have developed the first-in-human gene therapy using lentiviral vectors for severe haemophilia A
The innovative therapy, developed by the Centre for Stem
Cell Research (CSCR) at Christian Medical College (CMC), Vellore -- a
translational unit of BRIC-inStem, and supported by the Department of
Biotechnology, has demonstrated transformational results.
Early this year, scientists at the CMC-Vellore successfully conducted the
country's first human clinical trial of gene therapy for haemophilia A (FVIII
deficiency).
The single-centre study, which enrolled five participants
aged between 22 and 41 years, showed transformational results.
“The therapy successfully produced zero annualised bleeding
rates in all five enrolled participants while enabling prolonged production of
Factor VIII, eliminating the need for repeated infusions,” said the scientists,
in the paper published in the peer-reviewed New England Journal of Medicine.
This effect was observed “over a cumulative follow-up of 81
months, correlating Factor VIII activity with vector copy numbers in the
peripheral blood,” the team said.
Haemophilia A is a severe bleeding disorder caused by the
deficiency of clotting Factor VIII. It significantly affects patients' quality
of life, leading to spontaneous bleeding episodes. Although rare, India bears
the world’s second-largest burden of haemophilia, with approximately 136,000
cases.
Current treatments require frequent Factor VIII replacement
therapy, which faces challenges such as high costs, venous access in children,
and low patient acceptance.
The new gene therapy approach involves the use of a
lentiviral vector to introduce a normal copy of the Factor VIII gene into
autologous haematopoietic stem cells (HSCs).
These modified HSCs generate blood cells capable of
producing functional Factor VIII over extended periods.
“Participants were monitored for six months following the
gene therapy. Results showed a strong correlation between Factor VIII activity
levels and the vector copy number in peripheral blood,” the researchers said.
“This achievement underscores the long-term efficacy and safety of the therapy,
offering renewed hope for patients with severe Haemophilia A,” they added.
This pioneering study marks a transformative leap in
accessible and effective treatments for resource-limited settings, unlocking
new possibilities for managing previously incurable diseases. The therapy is
expected to soon undergo a second phase human trial.
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