New Delhi . Indian scientists have developed for the first time human gene therapy using lentiviral vector for severe hemophilia A, achieving a significant medical milestone.
This therapy, developed by the Center for Stem Cell Research (CSCR) at Christian Medical College (CMC) Vellore and supported by the Department of Biotechnology, has shown transformative results. It is a translation unit of BRICINSTEM.
In the beginning of this year, C.M.C. Vellore scientists successfully conducted the country's first human clinical trial of gene therapy for hemophilia A (FVIII deficiency).
Five participants aged between 22 to 41 years were included in this research, in which many transformative results emerged.
In a paper published in the New England Journal of Medicine, the scientists said the therapy successfully reduced annual bleeding rates to zero in all five enrolled participants, while Factor VIII production continued for a longer period of time, eliminating the need for repeated injections. Went.
The team said this effect was seen in an 81month cumulative followup study that established a relationship between factor VIII activity and vector copy numbers in peripheral blood.
Hemophilia A is a serious bleeding disorder caused by a deficiency of the clotting factor VIII. This significantly affects the quality of life of patients, causing bleeding. Although it is rare, there are approximately 136,000 cases of hemophilia in India, the second highest in the world.
Current treatments require frequent Factor VIII replacement therapy, which faces many challenges such as high cost, finding veins in children, and low patient acceptance.
Newer gene therapy approaches involve the use of lentiviral vectors to insert a normal copy of the factor VIII gene into autologous hematopoietic stem cells (HSCs).
These modified HSCs generate blood cells that are capable of producing functional Factor VIII for a long period of time.
“After gene therapy, the participants were monitored for six months,” the researchers said. The results showed a strong correlation between factor VIII activity levels and vector copy number in peripheral blood.''
“This achievement highlights the longterm efficacy and safety of the therapy, providing new hope for patients with severe hemophilia A,” he said.
This pioneering study is a transformative leap forward in making accessible and effective treatments accessible in resourcelimited settings, opening up new possibilities for the management of previously incurable diseases. It is expected that the second phase human trials of this therapy will be conducted soon.
This pioneering study represents a transformational leap in accessible and effective treatments for resourcelimited settings, opening up new possibilities for previously incurable diseases. It is expected that soon the second phase of this therapy will undergo human testing.
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Image Credit: KhasKhabar.