Times of India
11 May 2011
By Jayashree Nandi
Bangalore, India

Treating Thalassemia Could Be First Project
Altering genes to cure diseases has moved from sci–fi flicks to a major research investment in India. Bangalore is also looking at it with hope. The latest in labs here is gene therapy for thalassemia and haemophilia, which affect Indians in large numbers. Doctors and researchers are working to make it a reality.

Though treatment could be a long way off, the gene has been found and so too the vector to introduce it into the body. A team of doctors at the recently launched Mazumdar Shaw Centre for Therapeutics is exploring thalassemia treatment. Gene therapy became popular because of its potential in curing genetic blindness but could be a potential saviour in terminal conditions like cancer.

"Both haemophilia and thalessemia are single gene defects and congenital diseases which unfortunately have no cure. The only option is blood transfusion for life. The beta globin gene that has been identified for thalassemia can be transferred to the body through gene alteration. But when this transfer is being done, the gene doesn’t settle permanently but is transient.

At our centre, we’re collaborating with genomics companies and institutions to study how it can be done permanently and then applied to cure thalassemia," said consultant haemotoglogist Dr Sharat Damodar. Thalassemia is an inherited blood disorder in which the body makes an abnormal form of haemoglobin, while haemophilia is a bleeding disorder and blood takes a long time to clot. Initially, the team will start with animal tests.

Thalassemia was chosen for gene therapy because the number of patients is the highest in India and the Mediterranean but not so much in the West. About 10,000 children are born in India with thalessemia major every year. Almost 3,000 of them can be cured with bone marrow transplant but even if 10% of them were to get transplant, India has to do at least 300 transplants every year.

Medical director and vice–president, Mazumdar Shaw Cancer Center and Narayana Hrudayalaya Multi–Specialty Hospital, Dr Paul C Salins, told TOI: "There is no doubt that the future is in gene therapy. If we can alter the gene itself it’ll have far–reaching implications. Through our research, we’re trying to find out how we can deal with diseases that affect the common man."

Next–Generation Sequencing
Associated with gene therapy is exploring what in the gene sequence causes cancer. You either inherit or develop cancer as you grow. The incidence of the latter is higher and is caused by various genetic changes. Head of Institute of Bioinformatics and Applied biotechnology and Ganit Labs, Binay Panda, is collaborating with the advanced therapeutics centre to do genetic analysis and next generation sequencing.

"We want to dissect changes associated with cancer in the human genome – that’s the key to finding solutions. The human genome is 3 billion letters long. We have to read the letters and see how the building blocks of DNA change. An entire individual can be decoded in 10–15 days through this technology," he said. Binay is currently focusing on oral cancer genomics in India.

Biomarkers, an associated technology, is becoming popular. It helps in highlighting various markers in an individual for various diseases.

What’s it All About

• Experimental treatment involves introducing genetic material into a person’s cells to fight or prevent disease
• Genes are biological units of heredity; they determine traits like colour of hair and eye as well as ability of blood to carry oxygen
• Scientists need to identify more efficient ways to deliver genes to the body. To treat cancer and other diseases effectively with gene therapy, researchers must develop vectors that can be injected into patients and focus on target cells in body
• Similarly, genetic engineering is used in various crops to make them pest resistant or drought resistant. In India, there’s a major debate about how ethical and safe these technologies are. There’s a moratorium on Bt Brinjal, the first genetically manipulated food crop

Keep it at Bay
International Thalassemia Day was observed on May 8. The day highlighted the need to raise public awareness for prevention of thalassemia and the importance of clinical care for thalassemia patients across the world. Pregnant women are advised to take a simple blood test which can prevent more children from being born with thalassemia major.