NINS and Novartis make breakthrough by applying gene therapy

For the first time in Bangladesh, gene therapy has been applied to treat the chronic neurological disease Spinal Muscular Atrophy (SMA). The National Institute of Neurosciences & Hospital (NINS) has reached a new milestone by applying gene therapy to a child for the first time in Bangladesh to treat this congenital disease.

The gene therapy, curable and United States Food and Drug Administration (US FDA) approved, costs around BDT 22 crore for a single dose and has been provided free of charge by the multinational pharmaceutical company Novartis Bangladesh.

Spinal Muscular Atrophy (SMA) is a rare and complex congenital disease of the nervous system caused by genetic disorder. The muscles of children suffering from this disease continue to weaken. As a result, the children cannot sit or stand. But they possess normal intelligence. Later, the affected children die due to respiratory complications. Every year, many children die in the world because of the lack of treatment for this disease.

This disease usually destroys the nerve cells of the spinal cord of children. As the nerve cells that control the muscles are damaged, the muscles become weak. The newborns suffering from this disease become very weak and gradually suffer from congenital heart disease.

The Global Managed Access Programme (GMAP) of Novartis is designed to provide a feasible pathway for patients. Under this, patients who fulfill the required eligibility are selected. Treatment is then completed in accordance with applicable local laws and regulations.

A Bangladeshi child has been selected for this treatment under the afore-mentioned programme. As the drug is prepared by taking into account each patient's genetic characteristics and various other physical factors, which is a very complicated process, the treatment is very expensive. The medical cost of this gene therapy is unaffordable for the common people.

Through this humanitarian initiative of Novartis, new doors have opened up for treating patients of SMA in Bangladesh. So far, this medicine has been administered to 2,300 patients worldwide.

Novartis has been ensuring not only the treatment for the patients, but also making sure that the doctors of this country can use this gene therapy for their patients through proper training.