New Delhi: Offering hope to around India’s 1,36,000 hemophilia A affected patients, the Department of Biotechnology and the Centre for Stem Cell research (CSCR), CMC, Vellore, have successfully executed a first in-Human Gene Therapy with lentiviral vector for Severe Hemophilia A.
In the groundbreaking study the researchers observed an annualised zero bleeding rate in all the five subjects enrolled with the production of Factor VIII for a prolonged period of time thus obviating the need for repeated infusions, DBT stated.
“The volunteers were followed up for 6 months after undergoing therapy. Factor VIII activity was observed to correlate with vector copy number in the peripheral blood. The annualised bleeding rate was observed to be zero for all five participants over a cumulative follow-up of 81 months”, it added.
The department also informed that, scientists from CSCR undertook development of an alternate system for restoring Factor VIII expression, using a lentiviral vector in place of AAV vector and based on promising preclinical data a first-in-human clinical trial was conducted to evaluate the safety and feasibility of this gene therapy technology for severe hemophilia A involving five participants who received autologous haematopoietic stem cells (HSCs) transduced with lentiviral vector expressing Factor-VIII gene.
Haemophilia is a serious bleeding disorder caused by the deficiency of clotting factors, (i.e., Factor VIII and Factor IX proteins), leads to spontaneous bleeding episodes, and a reduced quality of life for patients and their families. Severe Hemophilia A is managed with repeated factor VIII replacement or hemostatic products that stop or prevent bleeding. Prohibitive cost, venous access especially in children, patient acceptance etc are some of the bottlenecks with respect to standard of care Hemophilia therapy.
Gene therapy using viral vectors which introduces a normal copy of the gene has emerged as a paradigm-changing therapeutic option for Hemophilia. In the case of haemophilia, gene therapy provides the body with the ability to produce Factor VIII, so that the need for repeated infusions can be obviated.
