Christian Medical College at Vellore conducted the first human clinical trial of gene therapy for haemophilia – a rare blood disorder that impairs body’s ability to clot the blood and exposes the patient to excessive bleeding from small injury or surgery which can even be fatal.
This was disclosed by Union Minister of Science & Technology Dr Jitendra Singh at an event organised on National Science Day at Vigyan Bhawan on 28 February 2024.
The scientists conducting the trials deployed a new technology of using lentivirus to inject a gene into patient’s stem cell. Lentiviruses are a family of viruses that cause diseases like AIDS by inserting DNA into their host cell’s genome. In this trial, scientists used lentivirus to inject a gene into stem cell linked to blood production. This is artificially introduced gene (called transgene) is primed to start the repair process.
This is called lentiviral vector gene therapy which is a method by which genes can be inserted, modified, or deleted in organisms using lentiviruses. This particular therapy is targeted at Haemophilia Type-A which the commonest among types A, B, and C.
Dr Singh further informed that the programme is supported by the Department of Biotechnology, the Centre for Stem Cell Research – a unit of InStem Bengaluru, in collaboration with Emory University, USA at Christian Medical College, Vellore. The minister hoped that manufacturing of this vector will commence soon in India and proceed with further clinical trials.
India has second largest number of patients suffering from haemophila. The existing therapy is periodic replacement of blood and the outcome depends on the severity of the diseased. It can cost anywhere between Rs 2 to 6 lakh and for a large population this is out of reach. Scientists have been long working on finding a cure applying novel therapeutics for the treatment of genetic diseases like thalassemia, duchenne muscular dystrophy, haemophilia.
Dr Singh said the National Science Day commemorates the discovery of “Raman Effect” by Nobel Laureate Sir CV Raman. He recalled C.V. Raman’s words that India could progress only through science, more science and still more science. He said, “After Prime Minister Narendra Modi took over, India is truly under the ‘Raman Effect’ as the PM Modi accords very high priority to science and keeps reiterating that science and technology are imperative to achieve the goal of Viksit Bharat.”
Highlighting India’s giant strides in the domain of science & technology, the minister said India’s bio-economy has grown thirteen folds in the last 10 years from $10 billion in 2014 to over $130 billion in 2024.
Dr. Jitendra Singh also expressed his confidence in the Anusandhan National Research Foundation that it will prove to be a boon for research and development as the Government has taken efforts to make it a comprehensive forum not only of scientists and industry but also researchers from social sciences and humanities.
During the event Dr. Jitendra Singh Acknowledging India progress in robust vaccine development capacity, which has been proved during Covid pandemic the Dr. Jitendra Singh said India is hailed as a global leader in preventive healthcare.
