Indian scientists have once again achieved a major success at the global level. India's first human gene therapy for hemophilia patients has been successful. When this therapy was used on patients suffering from severe hemophilia A, within a few weeks the rate of bleeding reached zero and there was no need to change the blood clotting factors repeatedly.
In this study published in the New England Journal of Medicine, researchers from the Central Government's Department of Biotechnology and the Stem Cell Research Center (CSCR) at CMC Vellore said that in patients suffering from severe hemophilia A, the amount of clotting protein called Factor 8 starts decreasing. Due to this, even a minor injury causes frequent and abnormal bleeding. People suffering from hemophilia A may bleed even when there is no injury. This puts their life at risk. To form clots in the blood of patients, they have to change Factor 8 repeatedly. To solve this problem, the researchers started research on five critical category patients aged 20 to 41 years. Under this, human gene therapy with lentiviral vector was started.
More than 1.36 lakh cases in the country
Actually, hemophilia is a serious bleeding disorder caused by a deficiency of clotting factors (ie, factor 8 and factor 9 proteins). More than 1.36 lakh cases are registered in India. India ranks second in the world with the highest number of patients. There are also some obstacles like cost in their treatment, frequent visits to the hospital, venous access especially in children.
Use of lentiviral vector instead of AAV vector
For this therapy, CSCR scientists found an alternative system to restore factor 8 expression by using lentiviral vector instead of AAV vector. Viral vectors are used in this. This gives the patient the ability to produce factor 8. Based on pre-clinical data, the first trial of this therapy was successful in five patients suffering from severe hemophilia A. In the trial, these patients received autologous hematopoietic stem cells (HSCs) which were transduced with lentiviral vector factor 8. Analysis found that autologous hematopoietic stem cells gave rise to blood cells producing factor VIII protein for a sufficient period of time in the patients, which eliminates the risk of recurrent infection.
Now the gene made in India will be available to the whole world
According to sources in the Center's Department of Biotechnology, the lentiviral vector discovered by scientists through this study will be produced in India and made available to the whole world. In February this year, Union Minister Dr. Jitendra Singh, while announcing this human trial at a program in Delhi, had said that if successful, the production of the vector will start in India itself and the country will move forward with further clinical trials.
(PC: ANI)
